0001628280-26-012399 | 10-K | XBRL Viewer | Rhythm Pharmaceuticals INC

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Asset Acquisitions - Xinvento B.V. (Details) - Xinvento B.V. - USD ($) $ in Millions		12 Months Ended
	Feb. 27, 2023	Dec. 31, 2023
Asset Acquisitions
Purchase consideration	$ 5.7
Aggregate payment upon achievement of development and commercial milestones	206.0
Clinical development milestone payments	6.0
Regulatory approval and commercial milestones milestone payments	125.0
Sales milestone payments	75.0
Cash consideration	4.5
Holdback, payable on the one-year anniversary of the acquisition	0.5	$ 0.5
Acquisition related costs	$ 0.6
Research and development
Asset Acquisitions
Purchase consideration		$ 5.7

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)	12 Months Ended
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Operating activities
Net loss	$ (196,539,000)	$ (260,602,000)	$ (184,678,000)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense	66,818,000	39,682,000	32,553,000
Depreciation and amortization	1,338,000	1,563,000	1,758,000
Non-cash interest expense	20,584,000	20,603,000	13,360,000
Non-cash accretion & amortization of short-term investments	(6,516,000)	(8,165,000)	(9,835,000)
Non-cash rent expense	428,000	400,000	401,000
Change in fair value of embedded derivative liability	(910,000)	(1,420,000)	(190,000)
Gain on settlement of forward contract	0	(8,900,000)	0
Acquired IPR&D assets classified as investing activities	0	92,385,000	5,667,000
Other non-cash items	90,000	0	0
Changes in operating assets and liabilities:
Accounts receivable	(7,570,000)	(3,645,000)	(8,643,000)
Inventory	(7,474,000)	(10,117,000)	(5,707,000)
Prepaid expenses and other current assets	(6,327,000)	(5,524,000)	2,876,000
Deferred revenue	(1,092,000)	0	(148,000)
Other long-term assets, net	(397,000)	7,943,000	1,656,000
Accounts payable, accrued expenses and other liabilities	21,892,000	21,918,000	14,773,000
Net cash used in operating activities	(115,675,000)	(113,879,000)	(136,157,000)
Investing activities
Purchases of short-term investments	(348,669,000)	(268,313,000)	(354,918,000)
Maturities of short-term investments	252,469,000	260,640,000	354,967,000
Acquisition of IPR&D assets	(40,000,000)	(40,500,000)	(5,667,000)
Purchases of property and equipment	(953,000)	0	(47,000)
Net cash used in investing activities	(137,153,000)	(48,173,000)	(5,665,000)
Financing activities
Repayment of deferred royalty obligation	(19,917,000)	(12,900,000)	(7,398,000)
Proceeds from issuance of common stock upon completion of public offering, net of offering costs	188,657,000	0	0
Proceeds from the exercise of stock options	13,604,000	15,976,000	7,468,000
Proceeds from issuance of common stock from ESPP	1,584,000	1,270,000	1,053,000
Net proceeds from issuance of common stock	34,035,000	39,146,000	48,875,000
Proceeds from royalty financing agreement, net of issuance costs	0	0	24,370,000
Gain on settlement of forward contract	0	8,900,000	0
Proceeds from Series A Preferred Stock, net of issuance costs	0	138,850,000	0
Net cash provided by financing activities	217,963,000	191,242,000	74,368,000
Effect of exchange rates on cash	87,000	2,000	(142,000)
Net increase (decrease) in cash, cash equivalents and restricted cash	(34,778,000)	29,192,000	(67,596,000)
Cash, cash equivalents and restricted cash at beginning of period	89,601,000	60,409,000	128,005,000
Cash, cash equivalents and restricted cash at end of period	54,823,000	89,601,000	60,409,000
Supplemental disclosure of non-cash investing and financing activities:
Non-current liability issued in exchange for the acquisition of IPR&D	0	33,669,000	0
Issuance of common stock in exchange for IPR&D	0	18,716,000	0
Accretion of preferred stock dividends	$ 5,378,000	$ 3,970,000	$ 0

Significant Agreements -RareStone Group Ltd. (Details) $ in Thousands		1 Months Ended	3 Months Ended		12 Months Ended
	Mar. 14, 2025 USD ($)	Dec. 31, 2021 USD ($) item shares	Sep. 30, 2022 USD ($)	Jun. 30, 2022 USD ($)	Dec. 31, 2025 USD ($)	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)	Mar. 13, 2025 USD ($)	Mar. 31, 2022 USD ($)
Significant Agreements
Total revenues					$ 189,757	$ 130,126	$ 77,428
Additional consideration for share repurchase	$ 0
Deferred revenue					194	1,286			$ 1,300
License revenue
Significant Agreements
Total revenues					$ (5,014)	$ 0	$ 0
Termination Agreement
Significant Agreements
Repayment of upfront fees on termination	6,300
Reversal of license revenue recognized on termination of contract	6,300
Reduction in revenue	$ 5,000
RareStone Group Ltd.
Significant Agreements
Upfront payment		$ 7,000
RareStone Group Ltd. \| License agreement
Significant Agreements
Upfront payment		$ 7,000
Issuance of common stock as consideration for LGC license (in shares) \| shares		1,077,586
Equity recorded at fair value		$ 1,000		$ 1,000						$ 2,400
Rarestone valuation realized gain (loss)			$ (1,000)	(1,000)
Gain (loss) on contract termination				$ 300
Upfront consideration		$ 8,000
Number of performance obligations \| item		2
RareStone Group Ltd. \| License agreement \| License revenue
Significant Agreements
Total revenues								$ 6,800

Income Taxes - Components of income taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Components of loss before income taxes
United States	$ (196,054)	$ (258,927)	$ (178,669)
Foreign	12	(1,329)	(5,445)
Loss before income taxes	$ (196,042)	$ (260,256)	$ (184,114)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

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+ Details

Fair Value of Financial Assets and Liabilities

12 Months Ended

Dec. 31, 2025

Fair Value Disclosures [Abstract]

Fair Value of Financial Assets and Liabilities

The following tables present information about the Company's financial assets and liabilities measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values (in thousands):


	Fair Value Measurements as of December 31, 2025 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market funds	$	33,949	$	—	$	—	$	33,949
Marketable securities:
US treasury securities	71,877		—		—		71,877
Corporate debt securities and commercial paper	—		262,771		—		262,771
Derivative asset	—		—		1,180		1,180
Total	$	105,826	$	262,771	$	1,180	$	369,777


	Fair Value Measurements as of December 31, 2024 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Commercial Paper	$	—	$	2,984	$	—	$	2,984
Money market funds	71,334		—		—		71,334
Marketable securities:							—
US treasury securities	65,118		—		—		65,118
Corporate debt securities and commercial paper	—		166,310		—		166,310
Derivative asset	—		—		270		270
Total	$	136,452	$	169,294	$	270	$	306,016

As of December 31, 2025 and 2024 the carrying amount of cash and cash equivalents and short-term investments was $388.9 million and $320.6 million, respectively, which approximates fair value. Cash and cash equivalents and short-term investments includes investments in U.S. treasury securities and money market funds that invest in U.S. government securities that are valued using quoted market prices. Accordingly, money market funds and government funds are categorized as Level 1. The financial assets valued based on Level 2 inputs consist of corporate debt securities and commercial paper, which consist of investments in highly-rated investment-grade corporations.

The embedded derivative asset associated with our deferred royalty obligation, as discussed further in Note 11, Long-Term Obligations, is measured at fair value using an option pricing Monte Carlo simulation model and is included as a component of the deferred royalty obligation. The embedded derivative (asset) or liability is subject to remeasurement at the end of each reporting period, with changes in fair value recognized as a component of other (expense) income, net. The assumptions used in the option pricing Monte Carlo simulation model include: (1) our estimates of the probability and timing of related events; (2) the probability-weighted net sales of IMCIVREE, including worldwide net product sales, upfront payments, milestones and royalties; (3) our risk-adjusted discount rate that includes a company specific risk premium; (4) our cost of debt; (5) volatility; and (6) the probability of a change in control occurring during the term of the instrument.

The forward contract associated with our Series A Convertible Preferred Stock, as discussed further in Note 9, “Series A Preferred Stock”, is measured at fair value. In order to value the forward contract, a binomial lattice model was used to determine the fair value of the Series A Preferred Stock. The fair value of the forward contract was measured as the difference between the consideration payable of $150.0 million and the fair value of the Series A Preferred Stock. The fair value of the forward contract was determined to be $0 at initial issuance and the change in the fair value from initial issuance to settlement of $8.9 million was recognized as other income in the consolidated statements of operation for the year ended December 31, 2024. The significant assumptions used in the binomial lattice model include: (1) the Company’s common stock price on the issuance and settlement dates; (2) the Conversion Price as of $48.00 as per the Agreement; (3) a 20-year term to maturity; (4) risk free rates (4.6% - 5%); and (5) volatility (68% and 67%).

The following tables set forth a summary of the changes in the estimated fair value of our embedded derivative liability (asset) (in thousands):


	Year ended December 31,
	2025		2024
Beginning aggregate estimated fair value of Level 3 liability (asset)	$	(270)	$	1,150
Change in fair value of embedded derivative	(910)		(1,420)
Fair value of forward contract - Series A Convertible Preferred Stock	—		8,900
Settlement of forward contract	—		(8,900)
Ending aggregate estimated fair value of Level 3 liability (asset)	$	(1,180)	$	(270)

The estimated fair value of the derivative (asset) or liability related to our Royalty Interest Financing Agreement (RIFA) with HealthCare Royalty was determined using Level 3 inputs. The fair value measurement of the derivative (asset) or liability is sensitive to changes in the unobservable inputs used to value the financial instrument. Changes in the inputs could result in changes to the fair value of each financial instrument.

Marketable Securities

The following tables summarize the Company's marketable securities (in thousands):


	December 31, 2025
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	262,412	$	370	$	(11)	$	262,771
U.S. Treasury Securities	71,634		243		—		71,877
	$	334,046	$	613	$	(11)	$	334,648


	December 31, 2024
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	166,255	$	147	$	(92)	$	166,310
U.S. Treasury Securities	65,074		79		(35)		65,118
	$	231,329	$	226	$	(127)	$	231,428

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

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+ Details

Stock-based Compensation - Black Scholes option pricing (Details)	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Weighted average assumptions
Risk‑free interest rate	4.32%	4.29%	2.35%
Expected term (in years)	6 years 3 months	6 years 1 month 17 days	6 years 1 month 9 days
Expected volatility	71.67%	74.59%	76.11%
Expected dividend yield	0.00%	0.00%	0.00%

Income Taxes

12 Months Ended

Dec. 31, 2025

Income Tax Disclosure [Abstract]

Income Taxes

The components of loss before income taxes are as follows (in thousands):


	As of December 31,
	2025		2024		2023
United States	$	(196,054)	$	(258,927)	$	(178,669)
Foreign	12		(1,329)		(5,445)
Loss before income taxes	$	(196,042)	$	(260,256)	$	(184,114)

Components of provision for income taxes are as follows (in thousands):


	As of December 31,
Current:	2025		2024		2023
U.S. Federal	$	—	$	—	$	—
U.S. State and Local	—		—		1
Foreign	497		346		563
Total Current Expense	$	497	$	346	$	564

Deferred:
U.S. Federal	$	—	$	—	$	—
U.S. State and Local	—		—		—
Foreign	—		—		—
Total Deferred Expense	$	—	$	—	$	—

The following table presents reconciliation of the income tax expense (benefit) at the federal statutory tax rate to the Company's effective income tax rate for the year ended December 31, 2025 after the adoption of ASU 2023-09:


	December 31, 2025
	Amount		Percentage
	(In thousands)
U.S. Federal Statutory Tax Rate	$	(41,169)	21.00	%
State and Local Income Tax, Net of Federal (National) Income Tax Effect	—		—	%
Foreign Tax Effects:
Other Foreign Jurisdictions	529		(0.27)	%
Tax Credits:
Research & Development Credits	(13,073)		6.67	%
Changes in Valuation Allowances	51,008		(26.02)	%
Nontaxable or Nondeductible Items:
Executive Compensation Limit	8,661		(4.42)	%
Stock-Based Compensation	(2,283)		1.16	%
LG Chem	(3,930)		2.00	%
Deferred royalty financing	913		(0.46)	%
Other	207		(0.10)	%
Other Adjustments	(366)		0.19	%
Income tax expense / Effective Tax Rate	$	497	(0.25)	%

The following table presents a reconciliation of the income tax expense (benefit) at the federal statutory tax rate to the Company's effective income tax rate for the years ended December 31, 2024 and 2023, before the adoption of ASU 2023-09 is as follows:


	As of December 31,
		2024		2023
Statutory tax rate		21.00	%	21.00	%
State tax, net of federal benefit		5.61		10.24
Research and development credits		3.75		4.02

Stock-based compensation		(0.57)		(1.37)
Other		(1.61)		(0.16)
Rate changes		(2.04)		(6.52)
Executive Compensation		(2.26)		(0.32)
License Agreement		(1.51)		—
Change in valuation allowance		(22.50)		(27.10)
Effective tax rate		(0.13)	%	(0.21)	%

The principal components of the Company's deferred tax assets and liabilities are as follows (in thousands):


	As of December 31,
	2025		2024
Deferred tax assets:
Net operating loss carryforwards	$	236,991	$	167,375
Research and development credits	19,853		17,604
Orphan drug credit	46,211		34,603
Capitalized license fee	23,418		21,661
Stock-based compensation	8,573		5,113
Section 174 Costs	33,718		67,624
Deferred revenue	—		336
Section 163(j) Interest Limitation	5,589		4,417
Accrued Expenses & Other	7,811		7,971
Total deferred tax assets	382,164		326,704
Valuation allowance	(381,166)		(325,503)
Net deferred tax assets	998		1,201
Deferred tax liabilities:
Operating lease right-of-use asset and other	(998)		(1,201)
Net deferred tax liabilities	(998)		(1,201)

Total deferred tax assets / liabilities	$	—	$	—

ASC 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of available evidence, it is more likely than not that some portion or all of the deferred tax assets will not be realized. After consideration of all the evidence, both positive and negative, the Company has recorded a full valuation allowance against its deferred tax assets at December 31, 2025 and 2024, because the Company's management has determined that is it more likely than not that these assets will not be realized. The increase in the valuation allowance of $55.7 million in 2025 and $58.3 million in 2024 primarily relates to the net loss incurred by the Company during each period.

As of December 31, 2025, the Company had federal and state net operating loss carryforwards of approximately $917.3 million and $807.9 million, respectively, which are available to reduce future taxable income. The net operating loss carryforwards expire at various times beginning in 2033 for federal and state purposes. Of the federal net operating loss carryforwards at December 31, 2025, $844.1 million can be carried forward indefinitely. As of December 31, 2025, the Company had gross foreign net operating loss carryforwards of approximately $4.5 million which have an indefinite carryforward period.

As of December 31, 2025, the Company had federal and state research tax credits of approximately $15.3 million and $4.6 million, respectively, which may be used to offset future tax liabilities. Additionally, as of 2025, the Company had a federal orphan drug credit related to qualifying research of $46.2 million. These tax credit carryforwards will begin to expire at various times beginning in 2033 for federal purposes and 2028 for state purposes.

The net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, respectively, as well as similar state provisions and other provisions within the Internal Revenue Code. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years.

The Company has not provided U.S. deferred income taxes or foreign withholding taxes on unremitted earnings of foreign subsidiaries as such amounts are considered to be indefinitely reinvested in these jurisdictions. The accumulated earnings in the foreign subsidiaries are primarily utilized to fund working capital requirements as its subsidiaries continue to expand their operations. The amount of any unrecognized deferred tax liability related to undistributed foreign earnings is immaterial.

The Company has not recorded any reserves for uncertain tax positions as of December 31, 2025 and 2024. The Company has not, as yet, conducted a study of research and development credit carryforwards. This study may result in an adjustment to the Company's research and development credit carryforwards; however, until a study is completed and any adjustment known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company's research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance. Thus, there would be no impact to the balance sheets or statements of operations and comprehensive loss if an adjustment were required.

Interest and penalty charges, if any, related to unrecognized tax benefits will be classified as provision for income taxes in the accompanying statements of operations and comprehensive loss. As of December 31, 2025 and 2024, the Company had no accrued interest or penalties related to uncertain tax positions.

The Company is subject to examination by the U.S. federal, state, local, and foreign income tax authorities for tax years 2013 forward. The Company is not currently under examination by the Internal Revenue Service or any other jurisdictions for any tax years.

- Definition

The entire disclosure for income tax.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 740
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477617/942-740-50-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
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-Name Accounting Standards Codification
-Section S50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480738/235-10-S50-1

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 944
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478822/944-740-50-1

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12

Reference 5: http://www.xbrl.org/2003/role/exampleRef
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-Section 55
-Paragraph 231
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482663/740-10-55-231

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
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-Paragraph 12C
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-12C

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
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-Name Accounting Standards Codification
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 270
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477891/740-270-50-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 6.I.5.Q1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479360/740-10-S99-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-13

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org/740/tableOfContent

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
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-Section 50
-Paragraph 14
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-14

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482685/740-10-50-21

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
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-Subparagraph (b)
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482603/740-30-50-2

+ Details

Stock-based Compensation - Stock-based compensation expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Stock-based Compensation
Total	$ 66,818	$ 39,682	$ 32,553
Stock options
Stock-based Compensation
Total	22,225	21,788	17,671
Employees stock purchase plan
Stock-based Compensation
Total	685	538	581
Restricted stock units
Stock-based Compensation
Total	39,256	16,886	5,998
Performance stock units
Stock-based Compensation
Total	4,652	470	8,303
Research and development
Stock-based Compensation
Total	19,802	10,514	8,449
Selling, general, and administrative
Stock-based Compensation
Total	$ 47,016	$ 29,168	$ 24,104

Summary of Significant Accounting Policies - Inventory (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Accounting Policies [Abstract]
Raw Materials	$ 6,745	$ 6,776
WIP	1,631	1,250
Finished Goods	17,377	10,715
Total Inventory	$ 25,753	$ 18,741

Summary of Significant Accounting Policies - Patent Costs (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Indefinite-Lived Intangible Assets [Line Items]
Selling, general, and administrative	$ 194,941	$ 144,304	$ 117,532
Patents
Indefinite-Lived Intangible Assets [Line Items]
Selling, general, and administrative	$ 2,000	$ 1,300	$ 600

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 805
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-Name Accounting Standards Codification
-Section 55
-Paragraph 38
-Subparagraph (e)
-Publisher FASB
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Reference 2: http://www.xbrl.org/2003/role/exampleRef
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Reference 4: http://www.xbrl.org/2003/role/exampleRef
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Reference 5: http://www.xbrl.org/2003/role/exampleRef
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-Section 55
-Paragraph 14
-Subparagraph (a)
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-URI https://asc.fasb.org/1943274/2147479876/805-20-55-14

Reference 6: http://www.xbrl.org/2003/role/exampleRef
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-URI https://asc.fasb.org/1943274/2147479876/805-20-55-14

Reference 7: http://www.xbrl.org/2003/role/exampleRef
-Name Accounting Standards Codification
-Section 55
-Paragraph 40
-SubTopic 30
-Topic 350
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482640/350-30-55-40

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 30
-Name Accounting Standards Codification
-Section 45
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482686/350-30-45-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 350
-SubTopic 30
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482665/350-30-50-3

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482665/350-30-50-2

+ Details

- Definition

The aggregate total costs related to selling a firm's product and services, as well as all other general and administrative expenses. Direct selling expenses (for example, credit, warranty, and advertising) are expenses that can be directly linked to the sale of specific products. Indirect selling expenses are expenses that cannot be directly linked to the sale of specific products, for example telephone expenses, Internet, and postal charges. General and administrative expenses include salaries of non-sales personnel, rent, utilities, communication, etc.

+ References + Details

Fair Value of Financial Assets and Liabilities - Marketable Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Fair Value of Financial Assets and Liabilities
Amortized Cost	$ 334,046	$ 231,329
Gross Unrealized Gains	613	226
Gross Unrealized Losses	(11)	(127)
Fair Value	334,648	231,428
Corporate debt securities and commercial paper (due within 1 year)
Fair Value of Financial Assets and Liabilities
Amortized cost, current	262,412	166,255
Gross unrealized gains, current	370	147
Gross Unrealized Losses	(11)	(92)
Fair value, current	262,771	166,310
Fair Value	262,771	166,310
U.S. Treasury Securities
Fair Value of Financial Assets and Liabilities
Amortized cost, current	71,634	65,074
Gross unrealized gains, current	243	79
Gross Unrealized Losses	0	(35)
Fair value, current	71,877	65,118
Fair Value	$ 71,877	$ 65,118

Asset Acquisitions	12 Months Ended
Asset Acquisitions	Dec. 31, 2025
Asset Acquisition [Abstract]
Asset Acquisitions	Asset Acquisitions LG Chem, Ltd. On January 4, 2024, the Company entered into a license agreement and share issuance agreement with LG Chem, Ltd. (“LGC”). Under the terms of the license agreement, the Company obtained worldwide rights to LGC’s proprietary compound bivamelagon. The total purchase consideration of $92.4 million was composed of $40.0 million of cash paid at closing and issued shares of the Company’s common stock with an aggregate value of $20.0 million. The shares were issued at a per share price equal to the ten-day volume weighted average closing price for our common stock, calculated as of the trading day immediately prior to January 4, 2024. As of January 4, 2024, the fair value of common stock issued was $18.7 million. The total purchase consideration also included an additional $40.0 million license fee payable in 18 months, which had a present value at closing of $33.7 million, which was accreted to its full value through interest expense, and $0.8 million of transaction costs which are recorded as selling, general and administrative expenses. On July 1, 2025, the Company made this additional payment of $40.0 million to LGC. In addition, under the terms of the license agreement, we agreed to pay LGC up to $205 million in cash upon achieving various regulatory and sales milestones based on net sales of bivamelagon. In addition and subject to the completion of Phase 2 development of bivamelagon, the Company has agreed to pay LGC royalties of between low-to-mid single digit percent of net revenues from its MC4R portfolio, including bivamelagon, commencing in 2029 and dependent upon achievement of various regulatory and indication approvals, and subject to customary deductions and anti-stacking. Royalties may further increase to a low double digit percent royalty, though such royalty would only be applicable on net sales of bivamelagon in a region if bivamelagon is covered by a composition of matter or method of use patent controlled by LGC in such region and the Company’s MC4R portfolio is not covered by any composition of matter or method of use patents controlled by the Company in such region. Such increased rate would only apply on net sales of bivamelagon for the limited remainder of the royalty term in the relevant region. The assets acquired were In-Process Research and Development (“IPR&D”) assets. However, since the IPR&D assets were determined to have no alternative future use, the Company recognized the $92.4 million of purchase consideration as research and development expense in the year ended December 31, 2024. The Company determined that the additional contingent consideration did not meet the definition of a derivative as of the acquisition date. Therefore, the Company did not record a contingent consideration liability on the acquisition date. The Company will recognize any future contingent consideration payments related to the LGC transaction in the period in which the achievement of the underlying milestones becomes probable. Xinvento B.V. On February 27, 2023, the Company, through its wholly-owned Dutch subsidiary, Rhythm Pharmaceuticals Netherlands B.V., a Dutch private limited liability company (“Rhythm BV”), entered into a Share Purchase Agreement (the “Purchase Agreement”) with Xinvento B.V., a Dutch private limited liability company based in the Netherlands (“Xinvento”), and the other parties named therein, pursuant to which, and concurrently with the execution thereof, Rhythm BV acquired all of the issued and outstanding shares of Xinvento. The aggregate consideration at closing was approximately $5.7 million, inclusive of transaction costs, as adjusted pursuant to the terms of the Purchase Agreement and subject to the distribution and payment terms set forth therein (the “Closing Purchase Price”). In addition to the Closing Purchase Price, the Purchase Agreement provides for the payment of additional contingent consideration totaling up to $206 million upon achievement of certain development, regulatory and commercial milestones by Xinvento, as follows: (i) up to an aggregate of $6 million in clinical development milestones; (ii) up to an aggregate of $125 million in regulatory approval and commercial milestones; and (iii) up to an aggregate of $75 million in sales milestones in the event a second molecule is selected, developed and approved. The total purchase consideration of $5.7 million was composed of $4.5 million of cash paid at closing, a $0.5 million holdback, payable on the one-year anniversary of the acquisition, and $0.6 million of acquisition-related costs. The $0.5 million holdback was paid in the quarter ended March 31, 2024, and is reflected in investing activities in the consolidated statement of cash flow for the year ended December 31, 2024. The Company determined that substantially all of the value as of acquisition date related to Xinvento’s In-Process Research and Development. As a result, the Company determined this transaction should be accounted for as an asset acquisition. The assets acquired were In-Process Research and Development (IPR&D) assets. However, since the IPR&D assets were determined to have no alternative future use, the Company recognized the $5.7 million of purchase consideration as research and development expense in the year ended December 31, 2023. The Company determined that the additional contingent consideration did not meet the definition of a derivative as of the acquisition date. Therefore, the Company did not record a contingent consideration liability on the acquisition date. The Company will recognize any future contingent consideration payments related to the Xinvento transaction in the period in which the achievement of the underlying milestones becomes probable. Xinvento's results of operations are included in the consolidated financial statements from the date of acquisition. For the years ended December 31, 2025 and 2024, the net loss associated with the operations of Xinvento was de minimis in the Company’s consolidated statements of operations.

Segment and Geographic Information (Tables)

12 Months Ended

Dec. 31, 2025

Segment Reporting [Abstract]

Schedule of segment profit or loss, including significant segment expenses

The table below is a summary of the segment profit or loss, including significant segment expenses (in thousands):

Segment Analysis


	2025		2024		2023

Net product revenue - U.S.	$	133,551	$	96,108	$	62,425
Net product revenue - International	61,220		34,018		15,002
Total net product revenue	194,771		130,126		77,428
License revenue	(5,014)		-		—
Total net revenue	189,757		130,126		77,428

Cost of sales	19,492		13,368		9,302
Global headcount expense	172,648		119,084		96,413
Preclinical, clinical and development expense	87,322		82,180		74,465
Commercial & medical affairs	66,963		55,767		47,905
Corporate, general & administrative	35,348		32,135		27,154
Other segment expenses	—		93,094		6,571
Other (income) expense, net	(1,264)		(11,139)		317
Interest (income) expense, net	5,290		5,893		(585)
Income taxes	497		346		564
Net loss	$	(196,539)	$	(260,602)	$	(184,678)

Schedule of revenue by geographic area

Total revenue by geographic area was as follows (in thousands):


	Year ended December 31,
	2025		2024		2023
US	$	133,551	$	96,108	$	62,425
International	61,220		34,018		15,003
Total net product revenue	$	194,771	$	130,126	$	77,428

Summary of Significant Accounting Policies - Other Long-Term Assets (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Accounting Policies [Abstract]
Long-term research and development costs	$ 1,660	$ 6,209
Other long-term assets	1,626	1,117
Other long-term assets	$ 3,286	$ 7,326

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2025

Income Tax Disclosure [Abstract]

Components of loss before income taxes

The components of loss before income taxes are as follows (in thousands):


	As of December 31,
	2025		2024		2023
United States	$	(196,054)	$	(258,927)	$	(178,669)
Foreign	12		(1,329)		(5,445)
Loss before income taxes	$	(196,042)	$	(260,256)	$	(184,114)

Components of provision for income taxes

Components of provision for income taxes are as follows (in thousands):


	As of December 31,
Current:	2025		2024		2023
U.S. Federal	$	—	$	—	$	—
U.S. State and Local	—		—		1
Foreign	497		346		563
Total Current Expense	$	497	$	346	$	564

Deferred:
U.S. Federal	$	—	$	—	$	—
U.S. State and Local	—		—		—
Foreign	—		—		—
Total Deferred Expense	$	—	$	—	$	—

Schedule of reconciliation of the income tax benefit at the federal statutory tax rate to effective income tax rate


	December 31, 2025
	Amount		Percentage
	(In thousands)
U.S. Federal Statutory Tax Rate	$	(41,169)	21.00	%
State and Local Income Tax, Net of Federal (National) Income Tax Effect	—		—	%
Foreign Tax Effects:
Other Foreign Jurisdictions	529		(0.27)	%
Tax Credits:
Research & Development Credits	(13,073)		6.67	%
Changes in Valuation Allowances	51,008		(26.02)	%
Nontaxable or Nondeductible Items:
Executive Compensation Limit	8,661		(4.42)	%
Stock-Based Compensation	(2,283)		1.16	%
LG Chem	(3,930)		2.00	%
Deferred royalty financing	913		(0.46)	%
Other	207		(0.10)	%
Other Adjustments	(366)		0.19	%
Income tax expense / Effective Tax Rate	$	497	(0.25)	%


	As of December 31,
		2024		2023
Statutory tax rate		21.00	%	21.00	%
State tax, net of federal benefit		5.61		10.24
Research and development credits		3.75		4.02

Stock-based compensation		(0.57)		(1.37)
Other		(1.61)		(0.16)
Rate changes		(2.04)		(6.52)
Executive Compensation		(2.26)		(0.32)
License Agreement		(1.51)		—
Change in valuation allowance		(22.50)		(27.10)
Effective tax rate		(0.13)	%	(0.21)	%

Schedule of principal components of deferred tax assets

The principal components of the Company's deferred tax assets and liabilities are as follows (in thousands):


	As of December 31,
	2025		2024
Deferred tax assets:
Net operating loss carryforwards	$	236,991	$	167,375
Research and development credits	19,853		17,604
Orphan drug credit	46,211		34,603
Capitalized license fee	23,418		21,661
Stock-based compensation	8,573		5,113
Section 174 Costs	33,718		67,624
Deferred revenue	—		336
Section 163(j) Interest Limitation	5,589		4,417
Accrued Expenses & Other	7,811		7,971
Total deferred tax assets	382,164		326,704
Valuation allowance	(381,166)		(325,503)
Net deferred tax assets	998		1,201
Deferred tax liabilities:
Operating lease right-of-use asset and other	(998)		(1,201)
Net deferred tax liabilities	(998)		(1,201)

Total deferred tax assets / liabilities	$	—	$	—

Income Taxes - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
Income Taxes - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Operating Loss Carryforwards [Line Items]
Increase in valuation allowance	$ 55,700	$ 58,300
Federal net operating loss carryforwards	917,300
State net operating loss carryforwards	807,900
Net operating loss indefinite carryforwards	844,100
Foreign net operating loss carryforwards	4,500
Federal orphan drug credit related to qualifying research	46,200
Accrued interest or penalties related to uncertain tax positions	0	$ 0
Federal \| Research tax credits
Operating Loss Carryforwards [Line Items]
Research tax credits	15,300
State \| Research tax credits
Operating Loss Carryforwards [Line Items]
Research tax credits	$ 4,600

Intangible Assets, net - Amortization expense (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Definite-lived intangible assets
2026	$ 855
2027	855
2028	855
2029	855
2030	855
Thereafter	1,044
Total	$ 5,319	$ 6,174

CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK & STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
At-the-market offering
Net offering costs	$ 700	$ 1,100
IPO
Net offering costs	$ 12,600
Common stock reserved for the conversion of Series A convertible preferred stock
Issuance costs		$ 2,300

Fair Value of Financial Assets and Liabilities (Tables)

12 Months Ended

Dec. 31, 2025

Fair Value Disclosures [Abstract]

Schedule of financial assets and liabilities measured at fair value on a recurring basis


	Fair Value Measurements as of December 31, 2025 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market funds	$	33,949	$	—	$	—	$	33,949
Marketable securities:
US treasury securities	71,877		—		—		71,877
Corporate debt securities and commercial paper	—		262,771		—		262,771
Derivative asset	—		—		1,180		1,180
Total	$	105,826	$	262,771	$	1,180	$	369,777


	Fair Value Measurements as of December 31, 2024 using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Commercial Paper	$	—	$	2,984	$	—	$	2,984
Money market funds	71,334		—		—		71,334
Marketable securities:							—
US treasury securities	65,118		—		—		65,118
Corporate debt securities and commercial paper	—		166,310		—		166,310
Derivative asset	—		—		270		270
Total	$	136,452	$	169,294	$	270	$	306,016

Schedule of estimated fair value of embedded derivative liabilities (assets)

The following tables set forth a summary of the changes in the estimated fair value of our embedded derivative liability (asset) (in thousands):


	Year ended December 31,
	2025		2024
Beginning aggregate estimated fair value of Level 3 liability (asset)	$	(270)	$	1,150
Change in fair value of embedded derivative	(910)		(1,420)
Fair value of forward contract - Series A Convertible Preferred Stock	—		8,900
Settlement of forward contract	—		(8,900)
Ending aggregate estimated fair value of Level 3 liability (asset)	$	(1,180)	$	(270)

Schedule of marketable securities

The following tables summarize the Company's marketable securities (in thousands):


	December 31, 2025
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	262,412	$	370	$	(11)	$	262,771
U.S. Treasury Securities	71,634		243		—		71,877
	$	334,046	$	613	$	(11)	$	334,648


	December 31, 2024
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Value
Assets
Corporate debt securities and commercial paper (due within 1 year)	$	166,255	$	147	$	(92)	$	166,310
U.S. Treasury Securities	65,074		79		(35)		65,118
	$	231,329	$	226	$	(127)	$	231,428

Nature of Business (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Accumulated deficit	$ 1,351,877	$ 1,155,338
Carrying amount of cash and cash equivalents and short term investments	$ 388,900	$ 320,600

- Definition

Amount of accumulated undistributed earnings (deficit).

+ References + Details

Segment and Geographic Information - Revenues (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Segment and Geographic Information
Total revenues	$ 189,757	$ 130,126	$ 77,428
Product revenue, net
Segment and Geographic Information
Total revenues	194,771	130,126	77,428
US \| Product revenue, net
Segment and Geographic Information
Total revenues	133,551	96,108	62,425
International \| Product revenue, net
Segment and Geographic Information
Total revenues	$ 61,220	$ 34,018	$ 15,003

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

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+ Details

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Total revenues	$ 189,757	$ 130,126	$ 77,428
Costs and expenses:
Cost of sales	19,492	13,368	9,302
Research and development	167,340	237,957	134,951
Selling, general, and administrative	194,941	144,304	117,532
Total costs and expenses	381,773	395,629	261,785
Loss from operations	(192,016)	(265,503)	(184,357)
Other income (expense):
Other income, net	1,264	2,239	190
Gain on settlement of forward contract	0	8,900	0
Interest expense	(20,583)	(20,603)	(13,892)
Interest income	15,293	14,711	13,945
Total other income (expense), net	(4,026)	5,247	243
Loss before income taxes	(196,042)	(260,256)	(184,114)
Provision for income taxes	497	346	564
Net loss	(196,539)	(260,602)	(184,678)
Accrued dividends on convertible preferred stock	(5,378)	(3,970)	0
Net loss attributable to common stockholders	$ (201,917)	$ (264,572)	$ (184,678)
Net loss per share attributable to common stockholders, basic (in dollars per share)	$ (3.11)	$ (4.34)	$ (3.20)
Net loss per share attributable to common stockholders, diluted (in dollars per share)	$ (3.11)	$ (4.34)	$ (3.20)
Weighted-average common shares outstanding, basic (in shares)	64,984,361	60,995,204	57,673,128
Weighted-average common shares outstanding, diluted (in shares)	64,984,361	60,995,204	57,673,128
Other comprehensive loss:
Net loss attributable to common stockholders	$ (201,917)	$ (264,572)	$ (184,678)
Foreign currency translation adjustment	(1,261)	2	(140)
Unrealized (loss) gain, net on marketable securities, net of tax	504	(175)	366
Comprehensive loss	(202,674)	(264,746)	(184,452)
Product revenue, net
Total revenues	194,771	130,126	77,428
License revenue
Total revenues	$ (5,014)	$ 0	$ 0

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2025

Payables and Accruals [Abstract]

Schedule of accrued expenses

Accrued expenses consists of the following (in thousands):


	December 31, 2025		December 31, 2024
Research and development costs	$	17,084	$	17,871
Professional fees	5,827		4,280
Payroll related	22,433		18,216
Royalties	2,870		2,091
Sales allowances	31,564		15,850
Other	4,078		4,350
Accrued expenses and other current liabilities	$	83,855	$	62,658

Series A Convertible Preferred Stock	12 Months Ended
Series A Convertible Preferred Stock	Dec. 31, 2025
Temporary Equity Disclosure [Abstract]
Series A Convertible Preferred Stock	Series A Convertible Preferred Stock On April 1, 2024, the Company entered into the Investment Agreement with certain affiliates of Perceptive Advisors LLC (“Perceptive”) and certain other investors (each, an “Investor” and collectively, the “Investors”), relating to the issuance and sale of 150,000 shares of a new series of the Company’s Series A Convertible Preferred Stock, par value $0.001 per share, titled the “Series A Convertible Preferred Stock” (the “Convertible Preferred Stock”), for an aggregate purchase price of $147.8 million, net of $2.3 million of issuance costs, or $1,000 per share (the “Issuance”). The Issuance closed on April 15, 2024. The Company determined the obligation to issue 150,000 shares of Convertible Preferred Stock to Perceptive and Investors in the future at a set price represented a forward contract which was required to be accounted for at fair value. The fair value of the forward contract was measured as the difference between the fair value of the Convertible Preferred Stock, as determined using a binomial lattice valuation model, and the consideration payable to the Company. The assumptions used in the binomial lattice model include: (1) the Company’s common stock price on the issuance and settlement dates; (2) the Conversion Price as of $48.00 as per the Agreement; (3) a 20-year term to maturity; (4) risk-free rates; and (5) volatility. The fair value of the forward contract upon issuance was determined to be $0. Upon closing, the value of the forward contract was determined to be $8.9 million and the fair value of the Convertible Preferred Stock was determined to be $141.1 million. The Convertible Preferred Stock was recorded at its fair value on the Issuance and the change in fair value of the forward contract was recorded as other income in its consolidated statement of operations for the year ended December 31, 2024. Issuance costs of $2.3 million were incurred and recorded as a reduction in the carrying value of the Convertible Preferred Stock in the year ended December 31, 2024. The Company classifies its Convertible Preferred Stock outside of stockholders’ equity as the redemption of such shares is outside the Company’s control. The Company did not adjust the carrying values of the Convertible Preferred Stock to redemption value as the shares are not probable of becoming redeemable as of December 31, 2025. On December 4, 2025, and in accordance with the terms described below, a holder of the Company's Series A Convertible Preferred Stock exercised their right to convert 17,500 shares of Series A Convertible Preferred Stock to common shares. Each of the 17,500 shares of Series A Convertible Preferred Stock converted to 20.8333 shares of common stock, resulting in a total of 364,582 shares of common stock. The Company reclassified the carrying value of the converted shares of $17.2 million from Series A convertible Preferred Stock to common stock and additional paid in capital, for this non-cash financing event. The Company accrued dividends of $5.4 million for the year ended December 31, 2025, as a reduction to additional paid-in capital and an increase to the carrying value of Series A Convertible Preferred Stock. After conversion of the 17,500 shares of Series A Convertible Preferred Stock, and the accretion of preferred stock dividends, the carrying value of Series A Convertible Preferred Stock as of December 31, 2025 was $131.0 million. The Convertible Preferred Stock has the following rights and privileges: Liquidation: The Series A Preferred Stock will rank senior to the Company’s common stock with respect to the distribution of assets upon the Company’s liquidation, dissolution or winding up. Upon any liquidation, dissolution or winding-up of the Company, whether voluntary or involuntary (“Liquidation”), each holder of Convertible Preferred Stock shall be entitled to receive payment for the greater of (i) 1.75 multiplied by the sum of the Liquidation Preference (i.e., Initial Liquidation Preference of $1,000 per share plus Paid-in-Kind (“PIK”) Dividends) plus unpaid Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference) or (ii) the amount such holder would have received if the Convertible Preferred Stock were fully converted to common stock. If the assets available for distribution are not sufficient to pay the holders of the Convertible Preferred Stock pursuant to the preceding sentence, the assets will be distributed ratably to the holders of the Convertible Preferred Stock. Voting: Holders of the Convertible Preferred Stock have the right to vote with the holders of common stock on each matter submitted for a vote on an as-converted basis, subject to the terms of the Convertible Preferred Stock as specified in the Amended and Restated Certificate of Designations. The holders of the Convertible Preferred Stock shall also have certain protective voting rights. Specifically, as long as the Convertible Preferred Stock are outstanding, each of the following events require at least a two thirds affirmative vote of the Convertible Preferred Stock holders: (a) any amendment or modification of the Certificate of Incorporation to authorize or create, or to increase the authorized number of shares of, any class or series of Dividend Parity Stock, Liquidation Parity Stock, Dividend Senior Stock or Liquidation Senior Stock, (b) any amendment, modification, repeal or waiver of any provision of the Certificate of Incorporation or the Amended and Restated Certificate of Designations that adversely affects the rights, preferences, privileges or powers of the Convertible Preferred Stock, (c) increase or decrease the number of authorized shares of Convertible Preferred Stock or issue additional shares of Convertible Preferred Stock, (d) the Company’s consolidation or combination with, or merger with or into, another Person, or any binding or statutory share exchange or involving the Convertible Preferred Stock, in each case unless: (i) the Convertible Preferred Stock either (x) remains outstanding after such consolidation, combination, merger, share exchange or reclassification; or (y) is converted or reclassified into, or is exchanged for, or represents solely the right to receive, preference securities of the continuing, resulting or surviving Person of such consolidation, combination, merger, share exchange or reclassification, or the parent thereof; (ii) the Convertible Preferred Stock that remains outstanding or such preference securities, as applicable, have rights, preferences and voting powers that, taken as a whole, are not materially less favorable to the Holders or the holders thereof, as applicable, than the rights, preferences and voting powers, taken as a whole, of the Convertible Preferred Stock immediately before the consummation of such consolidation, combination, merger, share exchange or reclassification; and (iii) the issuer of the Convertible Preferred Stock that remains outstanding or such preference securities, as applicable, is a corporation duly organized and existing under the laws of the United States of America, any State thereof or the District of Columbia that, if not the Company, will succeed to the Company under the Amended and Restated Certificate of Designations and the Convertible Preferred Stock. Redemption: The Company has the right to redeem all Convertible Preferred Stock after the Redemption Trigger Date, which is the fifth anniversary of the Initial Issue Date of April 15, 2024. The amount payable on the redemption date is equal to the Liquidation Preference (i.e., Initial Liquidation Preference of $1,000 per share plus PIK Dividends) plus any unpaid Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference). If a change of control occurs, each holder shall have the right to require the Company to repurchase all, or any whole number of shares that is less than all, of the holder’s Convertible Preferred Stock at an amount equal to 1.75 multiplied by the sum of the Liquidation Preference (i.e., Initial Liquidation Preference of $1,000 per share plus PIK Dividends) plus any unpaid Regular Dividends (to the extent such accumulated and unpaid Regular Dividends are not included in such Liquidation Preference). As of December 31, 2025, the Company does not consider a change of control to be probable and therefore the Convertible Preferred Stock is not considered redeemable. Dividends: After the second anniversary, dividends on the Convertible Preferred Stock accrue quarterly, at a 6% annual rate, and if not paid out in cash before the quarter end, will become PIK Dividends and added to the liquidation preference, or original issue price plus PIK Dividends. Since dividends do not commence until the second anniversary of the Issuance, the Convertible Preferred Stock is considered increasing rate preferred stock. Accordingly, the Company accretes the dividends, using the effective interest method, from Issuance to the first contractual call date, April 15, 2029. Conversions: Holders of Convertible Preferred Stock have the option to convert any number of whole shares at any time. The conversion is based on the sum of the Liquidation Preference plus unpaid Dividends divided by the $48.00 Conversion Price. Given the Initial Liquidation Preference of $1,000, each share of Convertible Preferred Stock would be convertible into 20.8333 shares of common stock, prior to any adjustments such as PIK Dividends, unpaid Dividends, stock splits, or voluntary conversion rate increases. Upon conversion, cash will be paid in lieu of any fractional share of common stock. However, based on certain restrictions on the conversion of the Convertible Preferred Stock specified in the Amended and Restated Certificate of Designations, a holder of Convertible Preferred Stock is not entitled to effect a conversion of any portion of its shares of Convertible Preferred Stock, or to vote in its capacity as a holder of shares of Convertible Preferred Stock with respect to matters submitted to holders of the common stock if, after giving effect to such conversion, that holder would beneficially own in excess of 4.99%, in the case of one holder, or 9.99%, in the case of the other holder, of the number of shares of common stock outstanding immediately after giving effect to such exercise. On May 7, 2024, the Company filed an Amended and Restated Certificate of Designations in respect of the Convertible Preferred Stock containing certain technical amendments to the terms of the Convertible Preferred Stock. The amendments contained in the Amended and Restated Certificate of Designations (x) limited the voting rights of the Convertible Preferred Stock to 24.9438 shares of the Company’s common stock per $1,000 liquidation preference of Convertible Preferred Stock and (y) eliminated a 1% step up in the interest rate that otherwise would have applied in the unlikely event that the Company was required to obtain and failed to obtain stockholder approval for certain conversion shares underlying the Convertible Preferred Stock. On July 10, 2024, the Company filed with the SEC a prospectus supplement to the prospectus included in the Company’s registration statement on Form S-3ASR filed with the SEC on March 2, 2023, covering the resale from time to time by the Investors of up to an aggregate of 3,124,995 shares of common stock, to satisfy registration rights that the Company granted to such stockholders in connection with the Issuance.

Series A Convertible Preferred Stock (Details) $ / shares in Units, $ in Thousands						12 Months Ended
	Dec. 04, 2025 shares	May 07, 2024 $ / shares shares	Apr. 15, 2024 USD ($) $ / shares shares	Apr. 01, 2024 USD ($) $ / shares shares	Mar. 02, 2023 shares	Dec. 31, 2025 USD ($) $ / shares shares	Dec. 31, 2024 USD ($) $ / shares shares	Dec. 31, 2023 USD ($)	Oct. 18, 2022 $ / shares	Sep. 19, 2022 $ / shares
Series A Convertible Preferred Stock
Share price (in USD per share) \| $ / shares									$ 26.00	$ 26.00
Derivative liability						$ 0	$ 0
Conversion of Series A preferred stock redeemable to common stock						17,242
Convertible preferred stock dividends						5,378	3,970	$ 0
Carrying value of convertible preferred stock						130,957	$ 142,820
Additional Paid-In Capital
Series A Convertible Preferred Stock
Conversion of Series A preferred stock redeemable to common stock						$ 17,241
Common stock reserved for the conversion of Series A convertible preferred stock
Series A Convertible Preferred Stock
Issuance of Series A Preferred Stock, net of issuance costs (in shares) \| shares				150,000	3,124,995		150,000
Convertible preferred stock, par value (in USD per share) \| $ / shares				$ 0.001		$ 0.001	$ 0.001
Aggregate purchase price				$ 147,800
Issuance costs				$ 2,300			$ 2,300
Share price (in USD per share) \| $ / shares				$ 1,000
Shares issued upon conversion (in shares) \| shares			20.8333
Conversion of Series A preferred stock redeemable to common stock (in shares) \| shares	17,500					17,500
Conversion of Series A preferred stock redeemable to common stock (in shares) \| shares	364,582
Carrying value of convertible preferred stock						$ 130,957	142,820	$ 0
Multiplier factor to liquidation preference per share			1.75
Initial liquidation preference (in USD per share) \| $ / shares		$ 1,000	$ 1,000
Annual rate of convertible preferred stock			6.00%
Conversion price (in USD per share) \| $ / shares			$ 48.00
Limited voting rights of convertible preferred stock to common stock shares \| shares		24.9438
Step up interest rate		1.00%
Common stock reserved for the conversion of Series A convertible preferred stock \| One holder of Series A convertible preferred stock
Series A Convertible Preferred Stock
Percentage ownership after conversion of preferred stock			4.99%
Common stock reserved for the conversion of Series A convertible preferred stock \| Separate holder of Series A convertible preferred stock
Series A Convertible Preferred Stock
Percentage ownership after conversion of preferred stock			9.99%
Common stock reserved for the conversion of Series A convertible preferred stock \| Forward Contracts
Series A Convertible Preferred Stock
Issuance of Series A Preferred Stock, net of issuance costs (in shares) \| shares			150,000
Issuance costs							$ 2,300
Per share or per unit amount of temporary equity issued (in USD per share) \| $ / shares			$ 48.00
Temporary equity, contract terms			20 years
Derivative liability			$ 8,900	$ 0
Fair value of convertible preferred stock			$ 141,100

Asset Acquisitions - LG Chem, Ltd. (Details) - USD ($) $ in Thousands			12 Months Ended
	Jul. 01, 2025	Jan. 04, 2024	Dec. 31, 2024	Dec. 31, 2023
Asset Acquisitions
Issuance of common stock, net of offering costs				$ 48,875
Issuance of common stock as consideration for LGC license			$ 18,716
LG Chem, LTD.
Asset Acquisitions
Aggregate payment upon achievement of development and commercial milestones		$ 205,000
Purchase consideration		92,400
Aggregate consideration at closing		40,000
Issuance of common stock, net of offering costs		20,000
Issuance of common stock as consideration for LGC license		18,700
License agreement consideration payment received in cash		$ 40,000
Lock up period for subsequent cash payment		18 months
License fee payable		$ 33,700
Additional consideration on asset acquisition	$ 40,000
LG Chem, LTD. \| Selling, general, and administrative
Asset Acquisitions
Acquisition related costs		$ 800
LG Chem, LTD. \| Research and development
Asset Acquisitions
Purchase consideration			$ 92,400

Summary of Significant Accounting Policies - Application of New or Revised Accounting Standards (Details)	12 Months Ended
	Dec. 31, 2025 segment
Accounting Policies [Abstract]
Number of reporting segments	1

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Current assets:
Cash and cash equivalents	$ 54,301	$ 89,137
Short-term investments	334,648	231,428
Accounts receivable, net	26,081	18,512
Inventory	25,753	18,741
Prepaid expenses and other current assets	26,133	16,382
Total current assets	466,916	374,200
Property and equipment, net	1,104	632
Right-of-use asset	3,049	3,477
Intangible assets, net	5,319	6,174
Restricted cash	522	464
Other long-term assets	3,286	7,326
Total assets	480,196	392,273
Current liabilities:
Accounts payable	13,947	12,328
Accrued expenses and other current liabilities	83,855	62,658
Other current liability - LG Chem	0	37,704
Deferred revenue	194	1,286
Deferred royalty obligation, current	7,296	1,541
Lease liability	650	0
Total current liabilities	105,942	115,517
Long-term liabilities:
Deferred royalty obligation	100,886	108,269
Lease liability, non-current	3,342	3,938
Derivative liability	0	0
Total liabilities	210,170	227,724
Commitments and contingencies (Note 12)
Series A convertible preferred stock, $0.001 par value: 150,000 shares authorized; 132,500 and 150,000 shares issued and outstanding at December 31, 2025 and December 31, 2024, respectively. Liquidation preference of $132,500 and $150,000 as of December 31, 2025, and December 31, 2024, respectively.	130,957	142,820
Stockholders’ equity:
Common stock, $0.001 par value: 120,000,000 shares authorized; 67,205,321 and 62,390,654 shares issued and outstanding at December 31, 2025 and December 31, 2024, respectively	67	61
Additional paid-in capital	1,491,675	1,177,045
Accumulated other comprehensive (loss) income	(796)	(39)
Accumulated deficit	(1,351,877)	(1,155,338)
Total stockholders’ equity	139,069	21,729
Total liabilities, convertible preferred stock and stockholders’ equity	$ 480,196	$ 392,273

Significant Agreements	12 Months Ended
Significant Agreements	Dec. 31, 2025
Significant Agreements [Abstract]
Significant Agreements	Significant Agreements License Agreements RareStone Group Ltd. In December 2021, the Company entered into an Exclusive License Agreement with RareStone Group Ltd., or the RareStone License. Pursuant to the RareStone License, we granted to RareStone an exclusive, sublicensable, royalty-bearing license under certain patent rights and know-how to develop, manufacture, commercialize and otherwise exploit any pharmaceutical product that contains setmelanotide in the diagnosis, treatment or prevention of conditions and diseases in humans in China, including mainland China, Hong Kong and Macao. In accordance with the terms of the RareStone License, RareStone made an upfront payment to Rhythm of $7.0 million and issued Rhythm 1,077,586 ordinary shares. The Company was eligible to receive development and commercialization milestones, as well as tiered royalty payments on annual net sales of IMCIVREE. The Company initially estimated the fair value of the RareStone equity to be $2.4 million based on a preliminary valuation during the first quarter of 2022. Upon completion of the valuation procedures during the second quarter of 2022, the Company concluded the initial fair value of the RareStone equity to be $1.0 million. During the third quarter of 2022, the Company estimated the fair value of the RareStone equity to be de minimis based upon the results of an updated valuation and recorded an other-than-temporary impairment of $1.0 million related to the decline in fair value as a component of other expense in our consolidated statements of operations and other comprehensive loss for the year ended December 31, 2022. The other-than-temporary impairment of $1.0 million included the reclassification of a $0.3 million unrealized loss previously recorded as a component of accumulated other comprehensive income (loss) in our consolidated statement of stockholders’ equity during the second quarter of 2022. The Company received total upfront consideration of $8.0 million comprised of an upfront payment of $7.0 million, and the estimated fair value of the RareStone equity of $1.0 million. The Company determined that the RareStone License contains two performance obligations, the delivery of the license and the supply of clinical and commercial product. Based on a relative fair-value allocation between the license and the manufacture of clinical and commercial product, the Company recognized $6.8 million of license revenue in the consolidated statements of operations and comprehensive loss during the year ended December 31, 2022. The discount related to commercial manufacturing supply will be deferred and recognized over the commercial supply period or upon termination of the agreement. On March 14, 2025, we entered into a termination agreement (the “Termination Agreement”) with RareStone Group Ltd. (“RareStone”) and RareStone Medicine (Shenzhen) Co., Ltd. (“RareStone Shenzhen”), pursuant to which the Company, RareStone and RareStone Shenzhen have mutually agreed to terminate (i) the Exclusive License Agreement between the Company and RareStone, dated December 3, 2021 (the “License Agreement”); and (ii) the Share Purchase Agreement between the Company and RareStone, dated December 3, 2021 (the “Share Purchase Agreement”, and with the License Agreement the “RareStone Agreements”). Under the Termination Agreement, the Company agreed to pay $6.3 million as a repayment of a portion of the upfront payment made pursuant to Section 7.1 of the License Agreement. In connection with the Termination Agreement, the Company and RareStone also entered into a Share Repurchase Agreement dated March 14, 2025, pursuant to which the Company has agreed to convey all of the shares acquired under the original Share Purchase Agreement back to RareStone, for no additional consideration. The Company had previously written off the value of the shares in the year ended December 31, 2022. Prior to executing the termination agreement, the Company had recorded $1.3 million of deferred revenue related to this arrangement for unsatisfied performance obligations. As a result of the Termination Agreement, the Company recognized the $6.3 million paid to RareStone as a reduction in previously-recognized license revenue as it represented consideration paid to a customer, and recognized all existing deferred revenue resulting in a net reduction in license revenue of $5.0 million. Ipsen Pharma S.A.S. Pursuant to our March 21, 2013 license agreement with Ipsen Pharma, S.A.S., or Ipsen, the Company has an exclusive, sublicensable, worldwide license to certain patents and other intellectual property rights to research, develop, and commercialize compounds that were discovered or researched by Ipsen in the course of conducting its MC4R program or that otherwise were covered by the licensed patents. Under the terms of the setmelanotide Ipsen license agreement, assuming that setmelanotide is successfully developed, receives regulatory approval and is commercialized, Ipsen may receive aggregate payments of up to $40.0 million upon the achievement of certain development and commercial milestones and royalties on future product sales in the mid-single digits. Substantially all of such aggregate payments of up to $40.0 million are for milestones that may be achieved no earlier than first commercial sale of setmelanotide. In the event that the Company executes a sublicense agreement, it shall make payments to Ipsen, depending on the date of such sublicense agreement, ranging from 10% to 20% of all revenues actually received under such sublicense agreement. The Company capitalized a $5.0 million and $4.0 million commercial milestone as a finite-lived intangible asset, as a result of the first commercial sales of IMCIVREE in the U.S. and Europe during March 2021 and March 2022, respectively. There were no research and development expenses related to milestones recorded in each of the years ended December 31, 2025, 2024 and 2023. Camurus In January 2016, the Company entered into a license agreement with Camurus AB, or Camurus, for the use of Camurus' drug delivery technology. The contract includes a non-refundable and non-creditable signing fee of $0.5 million. The Camurus agreement also includes up to $7.8 million in one-time, non-refundable development milestones achievable upon certain regulatory successes. The Company is also required to pay to Camurus, mid to mid-high single digit royalties, on a product-by-product and country-by-country basis of annual net sales, until the later of (i) 10 years after the date of first commercial sale of such product in such country; or (ii) the expiration of the last to expire valid claim of all licensed patent rights in such country covering such product. The Company is also required to pay one-time, non-refundable, non-creditable sales milestones upon the achievement of certain sales levels for such product that cannot be in excess of $57.0 million. The Company recorded development milestone expenses related to this license agreement of $1.0 million during the year ended December 31, 2022. The expenses were recorded as research and development expenses when the milestone criteria were met in full during 2023. There are no research and development expenses related to milestones recorded in 2025 or 2024.

Cybersecurity Risk Management and Strategy Disclosure	12 Months Ended
Cybersecurity Risk Management and Strategy Disclosure	Dec. 31, 2025
Cybersecurity Risk Management, Strategy, and Governance [Line Items]
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]	Cybersecurity Risk Management and Strategy We have developed and implemented a cybersecurity risk management program intended to protect the confidentiality, integrity, and availability of our critical systems and information. We design and assess our cybersecurity program based on the NIST Cybersecurity Framework (CSF). This framework provides us with a common language and structure for identifying, assessing, and managing cybersecurity risks across our organization. We do not claim to comply with the standards or specifications by using this framework. It is a guide to help us manage the cybersecurity risks that are relevant to our business. Our cybersecurity program is integrated into our overall enterprise risk management program, and shares common methodologies, reporting channels and governance processes that apply across the enterprise risk management program to other legal, compliance, strategic, operational, and financial risk areas. To this end, we have implemented a cybersecurity program that includes, but is not limited to, the following key elements: •Governance: The Board of Directors oversees our cybersecurity risk management strategy, regularly reviewing threats, risks, and mitigation measures. These reviews include both internal and independent evaluations of risks, controls, and effectiveness. •Vigilance: Our global cybersecurity operations are designed to detect, prevent, contain, and respond to cyber threats and incidents, aiming to minimize business disruptions. •Safeguards: We use administrative, technical, and physical controls designed to secure our personnel, information systems, operations, and sensitive data from cyber threats, such as cybersecurity policies and procedures, risk assessments, cybersecurity training, secure facility access, surveillance systems, firewalls, intrusion detection and prevention systems, disaster recovery capabilities, malware and ransomware defenses, access controls, and data protection. •Education: Personnel receive periodic cybersecurity training. Our policies require employees to report any actual or suspected cybersecurity events. •Third Party Risk Management: We strive to apply our cybersecurity standards to our third-party ecosystem, identifying and managing risks posed by third parties. •Incident Response: We have established an incident response plan that guides how we handle cybersecurity events and incidents. This plan includes procedures to detect, analyze, contain, and eradicate threats, and communicate significant incidents to executive management, the Board of Directors, regulators, and shareholders when appropriate. •Enterprise-Wide Coordination: Relevant stakeholders across the company—such as R&D, commercial, technology, legal, compliance, and other departments—work together to identify emerging risks and respond to cybersecurity threats through a cross-functional approach. •External Service Providers: We use external service providers, where appropriate, to assess, test, or otherwise assist with aspects of our cybersecurity processes. We have not identified any cybersecurity incidents that have materially affected our operations, business strategy, results of operations, or financial condition. We face risks from cybersecurity threats that, if realized, are reasonably likely to materially affect us, including our operations, business strategy, results of operations, or financial condition. For more information, see the section titled “Risk Factors— Our information technology systems, or those of our third-party CROs, CMOs or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of setmelanotide development programs, regulatory investigations, enforcement actions and lawsuits.”
Cybersecurity Risk Management Processes Integrated [Flag]	true
Cybersecurity Risk Management Processes Integrated [Text Block]	We design and assess our cybersecurity program based on the NIST Cybersecurity Framework (CSF). This framework provides us with a common language and structure for identifying, assessing, and managing cybersecurity risks across our organization. We do not claim to comply with the standards or specifications by using this framework. It is a guide to help us manage the cybersecurity risks that are relevant to our business. Our cybersecurity program is integrated into our overall enterprise risk management program, and shares common methodologies, reporting channels and governance processes that apply across the enterprise risk management program to other legal, compliance, strategic, operational, and financial risk areas. To this end, we have implemented a cybersecurity program that includes, but is not limited to, the following key elements: •Governance: The Board of Directors oversees our cybersecurity risk management strategy, regularly reviewing threats, risks, and mitigation measures. These reviews include both internal and independent evaluations of risks, controls, and effectiveness. •Vigilance: Our global cybersecurity operations are designed to detect, prevent, contain, and respond to cyber threats and incidents, aiming to minimize business disruptions. •Safeguards: We use administrative, technical, and physical controls designed to secure our personnel, information systems, operations, and sensitive data from cyber threats, such as cybersecurity policies and procedures, risk assessments, cybersecurity training, secure facility access, surveillance systems, firewalls, intrusion detection and prevention systems, disaster recovery capabilities, malware and ransomware defenses, access controls, and data protection. •Education: Personnel receive periodic cybersecurity training. Our policies require employees to report any actual or suspected cybersecurity events. •Third Party Risk Management: We strive to apply our cybersecurity standards to our third-party ecosystem, identifying and managing risks posed by third parties. •Incident Response: We have established an incident response plan that guides how we handle cybersecurity events and incidents. This plan includes procedures to detect, analyze, contain, and eradicate threats, and communicate significant incidents to executive management, the Board of Directors, regulators, and shareholders when appropriate. •Enterprise-Wide Coordination: Relevant stakeholders across the company—such as R&D, commercial, technology, legal, compliance, and other departments—work together to identify emerging risks and respond to cybersecurity threats through a cross-functional approach. •External Service Providers: We use external service providers, where appropriate, to assess, test, or otherwise assist with aspects of our cybersecurity processes.
Cybersecurity Risk Management Third Party Engaged [Flag]	true
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag]	true
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag]	false
Cybersecurity Risk Board of Directors Oversight [Text Block]	Our Board considers oversight of cybersecurity risk to be an essential component of its enterprise risk oversight responsibility and directly oversees management’s implementation of our cybersecurity program. The Board receives periodic reports from management on our cybersecurity program and risks. In addition, management updates the Board, where it deems appropriate, regarding any cybersecurity incidents it considers to be significant. Board members receive presentations on cybersecurity risk and strategy from our Cybersecurity Director, as part of the Board’s continuing education on topics that impact public companies. The Cybersecurity Director, with the help of our IT and Legal team is responsible for assessing and managing our material risks from cybersecurity threats. This position has the primary responsibility for our overall cybersecurity risk management program and supervises both our internal personnel and our retained external cybersecurity consultants. The current Cybersecurity Director has extensive information security and program management experience and has held past positions as the head of cybersecurity for a wide range of organizations. Our management team supervises efforts to prevent, detect, mitigate, and remediate cybersecurity risks and incidents through various means, which may include briefings from internal security personnel and other information obtained from governmental, public, or private sources, including external consultants engaged by us, and alerts and reports produced by security tools deployed in the IT environment.
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block]	Board
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]	Our Board considers oversight of cybersecurity risk to be an essential component of its enterprise risk oversight responsibility and directly oversees management’s implementation of our cybersecurity program. The Board receives periodic reports from management on our cybersecurity program and risks. In addition, management updates the Board, where it deems appropriate, regarding any cybersecurity incidents it considers to be significant. Board members receive presentations on cybersecurity risk and strategy from our Cybersecurity Director, as part of the Board’s continuing education on topics that impact public companies.
Cybersecurity Risk Role of Management [Text Block]	The Cybersecurity Director, with the help of our IT and Legal team is responsible for assessing and managing our material risks from cybersecurity threats. This position has the primary responsibility for our overall cybersecurity risk management program and supervises both our internal personnel and our retained external cybersecurity consultants. The current Cybersecurity Director has extensive information security and program management experience and has held past positions as the head of cybersecurity for a wide range of organizations. Our management team supervises efforts to prevent, detect, mitigate, and remediate cybersecurity risks and incidents through various means, which may include briefings from internal security personnel and other information obtained from governmental, public, or private sources, including external consultants engaged by us, and alerts and reports produced by security tools deployed in the IT environment.
Cybersecurity Risk Management Positions or Committees Responsible [Flag]	true
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]	Cybersecurity Director, with the help of our IT and Legal team
Cybersecurity Risk Management Expertise of Management Responsible [Text Block]	The current Cybersecurity Director has extensive information security and program management experience and has held past positions as the head of cybersecurity for a wide range of organizations.
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block]	The Board receives periodic reports from management on our cybersecurity program and risks. In addition, management updates the Board, where it deems appropriate, regarding any cybersecurity incidents it considers to be significant. Board members receive presentations on cybersecurity risk and strategy from our Cybersecurity Director, as part of the Board’s continuing education on topics that impact public companies.
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag]	true

Accrued Expenses

12 Months Ended

Dec. 31, 2025

Payables and Accruals [Abstract]

Accrued Expenses

Accrued expenses consists of the following (in thousands):


	December 31, 2025		December 31, 2024
Research and development costs	$	17,084	$	17,871
Professional fees	5,827		4,280
Payroll related	22,433		18,216
Royalties	2,870		2,091
Sales allowances	31,564		15,850
Other	4,078		4,350
Accrued expenses and other current liabilities	$	83,855	$	62,658

Related-Party Transactions	12 Months Ended
Related-Party Transactions	Dec. 31, 2025
Related Party Transactions [Abstract]
Related-Party Transactions	Related-Party Transactions Expenses paid directly to related parties for the years ended December 31, 2025 and 2024, were immaterial. Outstanding payments due to related parties as of December 31, 2025 and December 31, 2024 were immaterial.

- Definition

The entire disclosure for related party transactions. Examples of related party transactions include transactions between (a) a parent company and its subsidiary; (b) subsidiaries of a common parent; (c) and entity and its principal owners; and (d) affiliates.

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Intangible Assets, net - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Finite-Lived Intangible Assets [Line Items]
Intangible assets, net	$ 5,319	$ 6,174
Remaining amortization period	11 years
Cost of sales
Finite-Lived Intangible Assets [Line Items]
Amortization expense	$ 900	$ 900	$ 900

- Definition

The aggregate expense charged against earnings to allocate the cost of intangible assets (nonphysical assets not used in production) in a systematic and rational manner to the periods expected to benefit from such assets.

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+ Details

Common Stock	12 Months Ended
Common Stock	Dec. 31, 2025
Equity [Abstract]
Common Stock	Common Stock Common Stock On December 4, 2025, one of the Company's Series A Convertible Preferred Stock holders exercised their conversion right and converted 17,500 Convertible Preferred A Stock into 364,582 shares of common stock. Refer to Note 9, Series A Convertible Preferred Stock, for further information. On July 9, 2025, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with Morgan Stanley & Co. LLC and BofA Securities, Inc., as the representatives of the several underwriters named in the Underwriting Agreement (collectively, the “Underwriters”), in connection with a follow-on offering, issuance and sale by the Company of 2,058,824 shares of the Company’s common stock. The offering price of the shares of common stock to the public was $85.00 per share. In addition, under the terms of the Underwriting Agreement, the Company granted the Underwriters a 30-day option to purchase up to 308,823 additional shares of Common Stock, at the public offering price per share, less underwriting discounts and commissions. On July 10, 2025, the Underwriters exercised the option in full. The closing of the sale of the shares pursuant to the offering, including the shares sold pursuant to the exercise in full of the option, took place on July 11, 2025, resulting in net proceeds of approximately $188.7 million, net of $12.6 million of underwriting discounts and commissions, and other offering expenses incurred by the Company, for a total share issuance of 2,367,647. On February 29, 2024, the Company and Cowen entered into Amendment No. 1 to Sales Agreement (the “Amendment”) to increase the aggregate offering price of the shares of common stock that may be issued and sold pursuant to the Sales Agreement to $200.0 million (excluding the aggregate offering price of shares of common stock issued and sold pursuant to the Sales Agreement prior to February 29, 2024). In connection with the Amendment, on February 29, 2024, the Company filed with the Securities Exchange Commission a prospectus supplement, dated February 29, 2024, which, combined with the Base Prospectus (together, the “New Prospectus”), amended the Prior Prospectus in its entirety. The issuances and sales under the Sales Agreement, as amended by the Amendment, will be made pursuant to the Registration Statement and the New Prospectus. In the quarter ended December 31, 2024 the Company sold 744,595 shares of common stock in the ATM program for net proceeds of $41.2 million. The Company also sold an additional 587,510 shares of common stock in the ATM program through January 21, 2025 for net proceeds of approximately $32.1 million. On September 19, 2022, the Company completed a public offering of 4,800,000 shares of common stock at a price to the public of $26.00 per share. The Company received $116,887 in net proceeds after deducting underwriting discounts, commissions and offering expenses. In addition, the Company granted the underwriters a 30-day option to purchase up to an additional 720,000 shares of its common stock at the price to the public, less underwriting discounts and commissions. On October 18, 2022, the Company completed the sale of an additional 580,000 shares of common stock at a price to the public of 26.00 per share pursuant to the partial exercise of the underwriters’ option to purchase additional shares, for aggregate net proceeds of approximately $14.2 million, after deducting underwriting discounts, commissions and offering expenses. On November 2, 2021, the Company entered into a Sales Agreement with Cowen and Company, LLC (“Cowen”), pursuant to which the Company may issue and sell shares of its common stock, having an aggregate offering price of up to $100.0 million, from time to time through an “at the market” equity offering program under which Cowen acts as sales agent (the “ATM Program”). Between August 10, 2023 and August 21, 2023, the Company sold approximately two million shares of its common stock in the ATM Program for net proceeds of approximately $48.9 million.

- Definition

The entire disclosure for equity.

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+ Details

Significant Agreements - Camurus (Details) - Camurus - License agreement - USD ($) $ in Millions	1 Months Ended	12 Months Ended
	Jan. 31, 2016	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2022
Significant Agreements
Non-refundable and non-creditable signing fee	$ 0.5
Royalty payment period	10 years
Milestone expenses		$ 0.0	$ 0.0	$ 1.0
Maximum
Significant Agreements
One-time non-refundable development milestone payment	$ 7.8
One-time non-refundable non-creditable sales milestone payment	$ 57.0

Fair Value of Financial Assets and Liabilities - Changes in Embedded Derivative Liability (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
Roll forward of the fair value of financial liabilities and assets
Beginning aggregate estimated fair value of Level 3 liability (asset)	$ (270)	$ 1,150
Ending aggregate estimated fair value of Level 3 liability (asset)	(1,180)	(270)
Level 3 \| Embedded Derivative Financial Instruments
Roll forward of the fair value of financial liabilities and assets
Change in fair value of embedded derivative	(910)	(1,420)
Fair value of forward contract - Series A Convertible Preferred Stock	0	8,900
Settlement of forward contract	$ 0	$ (8,900)

Stock-based Compensation

12 Months Ended

Dec. 31, 2025

Share-Based Payment Arrangement [Abstract]

Stock-based Compensation

2017 Equity Incentive Plan

The Rhythm Pharmaceuticals, Inc. 2017 Equity Incentive Plan (the “2017 Plan”) provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, performance units, restricted stock awards, restricted stock units and stock grants to employees, consultants, advisors and directors of us or our affiliates, as determined by the board of directors. The number of shares authorized under the 2017 Plan increases on the first day of each calendar year, commencing on January 1, 2018 and ending on (and including) January 1, 2027, by an amount equal to 4% of the outstanding shares of stock outstanding as of the end of the immediately preceding fiscal year. On January 1, 2026, 2025, 2024 and 2023, 2,688,212, 2,495,626, 2,377,062, and 2,264,497 shares, respectively, were added to the 2017 Plan. Notwithstanding the foregoing, the board of directors may act prior to January 1 for a given year to provide that there will be no such January 1 increase in the number of shares authorized under the 2017 Plan for such year, or that the increase in the number of shares authorized under the 2017 Plan for such year will be a lesser number than would otherwise occur pursuant to the preceding sentence.

Shares of common stock issued upon the exercise of stock options are generally issued from new shares of the Company. The 2017 Plan provides that the exercise price of incentive stock options cannot be less than 100% of the fair market value of the common stock on the date of the award for participants who own less than 10% of the total combined voting power of stock of the Company, and not less than 110% for participants who own more than 10% of the Company's voting power. Awards granted under the 2017 Plan will vest over periods as determined by the Company's board of directors. For options granted to date, the exercise price equaled the fair value of the common stock as determined by the board of directors on the date of grant.

As of December 31, 2025, an aggregate of 13,717,040 shares of common stock were authorized for issuance under the 2017 Plan, of which a total of approximately 4,636,850 shares of common stock remained available for future awards. In addition, a total of 9,080,190 shares of common stock reserved for issuance were subject to currently outstanding stock options, performance share units and restricted stock units granted under the Plan.

2022 Inducement Plan

On February 9, 2022, the Company’s board of directors adopted the Rhythm Pharmaceuticals, Inc. 2022 Employment Inducement Plan (the “2022 Inducement Plan”), which became effective on such date without stockholder approval pursuant to Rule 5635(c)(4) of the Nasdaq Stock Market LLC listing rules (“Rule 5635(c)(4)”). The 2022 Inducement Plan provides for the grant of non-qualified stock options, stock appreciation rights, performance units, restricted stock awards, restricted stock units and stock grants. In accordance with Rule 5635(c)(4), awards under the 2022 Inducement Plan may only be made to a newly hired employee who has not previously been a member of the Company’s board of directors, or an employee who is being rehired following a bona fide period of non-employment by the Company or a subsidiary, as a material inducement to the employee’s entering into employment with the Company or its subsidiary. An aggregate of 1,000,000 shares of the Company’s common stock have been reserved for issuance under the 2022 Inducement Plan.

The exercise price of stock options granted under the 2022 Inducement Plan will not be less than the fair market value of a share of the Company’s common stock on the grant date. Other terms of awards, including vesting requirements, are determined by the Company’s board of directors and are subject to the provisions of the 2022 Inducement Plan. Stock options granted to employees generally vest over a four-year period but may be granted with different vesting terms. Certain options may provide for accelerated vesting in the event of a change in control. Stock options granted under the 2022 Inducement Plan expire no more than 10 years from the date of grant. As of December 31, 2025, there were 371,148 stock option awards outstanding, 194,419 restricted stock unit awards outstanding and 59,067 shares of common stock available for future grant under the 2022 Inducement Plan.

2017 Employee Stock Purchase Plan

The Company maintains the Rhythm Pharmaceuticals, Inc. 2017 Employee Stock Purchase Plan, (the “2017 ESPP”), which became effective in connection with the completion of the Company’s IPO in October 2017. As of December 31, 2025, a total of 1,240,958 shares of common stock were reserved for issuance under the 2017 ESPP. In addition, the number of shares authorized under the 2017 ESPP increases on the first day of each calendar year, commencing on January 1, 2019 and ending on (and including) January 1, 2027, by an amount equal to the lesser of 1% of outstanding shares as of the end of the immediately preceding fiscal year. On January 1, 2025, 2024 and 2023, no shares were added to the 2017 ESPP. Notwithstanding the foregoing, the board of directors may act prior to January 1 of a given year to provide that there will be no such January 1 increase in the number of shares authorized under the 2017 ESPP for such year, or that the increase in the number of shares authorized under the 2017 ESPP for such year will be a lesser number than would otherwise occur pursuant to the preceding sentence. During the years ended December 31, 2025, 2024 and 2023, shares of 37,514, 44,554, and 49,819 were issued under the 2017 ESPP.

The purchase price of common stock under our ESPP is equal to 85.0% of the lower of (i) the market value per share of the common stock on the first business day of an offering period or (ii) the market value per share of the common stock on the purchase date. The fair value of the discounted purchases made under our ESPP is calculated using the Black-Scholes model. The fair value of the look-back provision plus the 15.0% discount is recognized as compensation expense over the 6 months purchase period.

Stock Options

The Company estimates the fair value of stock option awards to employees and non-employees using the Black-Scholes option-pricing model, which requires the input of subjective assumptions, including (a) the expected volatility of the underlying common stock, (b) the expected term of the award, (c) the risk-free interest rate, and (d) expected dividends. The Company bases its estimate of expected volatility using a blend of its stock price history for the length of time it has market data for its stock and using the historical volatility of a group of companies in the pharmaceutical and biotechnology industries in a similar stage of development as the Company that are publicly traded. For these analyses, the Company selected companies with comparable characteristics to its own including enterprise value, risk profiles and with historical share price information sufficient to meet the expected life of the stock-based awards. The Company computes the historical volatility data using the daily closing prices for the selected companies' shares during the equivalent period of the calculated expected term of its stock-based awards. The Company will continue to apply this process until a sufficient amount of historical information regarding the volatility of its own stock price becomes available.

The Company estimated the expected life of its employee stock options using the “simplified” method, whereby, the expected life equals the average of the vesting term and the original contractual term of the option. The risk-free interest rates for periods within the expected life of the option are based on the U.S. Treasury yield curve in effect during the period the options were granted. We have elected to account for forfeitures as they occur.

The grant date fair value of awards subject to service-based vesting is recognized ratably over the requisite service period, which is generally the vesting period of the respective awards. The Company's stock option awards typically vest over a service period that ranges from one to four years and includes awards with one year cliff vesting followed by ratable monthly and quarterly vesting thereafter and ratable monthly and quarterly vesting beginning on the grant date.

During the years ended December 31, 2025, 2024 and 2023, the Company granted 740,919, 926,543, and 842,528 stock option awards pursuant to the 2017 Plan to certain directors, employees and non-employees, respectively. Using the Black-Scholes option pricing model, the weighted-average grant date fair value relating to outstanding stock options granted under the 2017 Plan during the years ended December 31, 2025, 2024, and 2023 was $57.70, $32.69, and $17.72, respectively.

During the years ended December 31, 2025, 2024 and 2023, the Company granted 0, 57,100, and 229,360 stock option awards pursuant to the 2022 Inducement Plan. Using the Black-Scholes option pricing model, the weighted-average grant date fair value relating to outstanding stock options granted under the Company’s stock option plan during the years ended December 31, 2024 and 2023 were $29.16 and $15.30, respectively.

The fair value of stock options granted to employees and directors was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions:


	Year ended December 31,
	2025		2024		2023
Risk‑free interest rate	4.32	%	4.29	%	2.35	%
Expected term (in years)	6.25		6.13		6.11
Expected volatility	71.67	%	74.59	%	76.11	%
Expected dividend yield	—		—		—

A summary of the Company's stock option activity for the year ended December 31, 2025 is as follows:


	Number of Options	Weighted- Average Exercise Price		Weighted‑ Average Remaining Contractual Term	Aggregate Intrinsic Value
Outstanding as of December 31, 2024	6,611,391	$	23.29	6.66	$	216,413
Granted	740,919	$	57.70	—	$	—
Exercised	(843,525)	$	16.13	—	$	52,980
Cancelled	(42,607)	$	26.03	—	$	—
Outstanding as of December 31, 2025	6,466,178	$	28.15	6.12	$	510,138
Options exercisable at December 31, 2025	4,940,330	$	23.09	5.48	$	414,757

Restricted Stock Units

The Company may grant restricted stock units (“RSUs”) to employees and nonemployee directors under the 2017 Plan and to employees under the 2022 Inducement Plan. Each RSU represents a right to receive one share of the Company's common stock upon the completion of a specific period of continued service. RSU awards granted are valued at the market price of the Company's common stock on the date of grant. The Company recognizes stock-based compensation expense for the fair values of these RSUs on a straight-line basis over the requisite service period of these awards.

A summary of the Company's restricted stock unit activity for the year ended December 31, 2025 is as follows:


	Number of RSUs	Weighted- Average Grant Date Fair Value
Unvested as of December 31, 2024	1,826,709	$	36.40
Granted	1,771,564	65.24
Vested	(635,186)	33.36
Cancelled	(78,152)	47.84
Unvested as of December 31, 2025	2,884,935	$	54.42

As of December 31, 2025, the aggregate intrinsic value of unvested RSUs was $308.8 million.

Performance Stock Units

In November 2021, the Company granted a maximum of 956,145 performance stock units (“PSUs”) to employees under the 2017 Plan. Each PSU represents a right to receive one share of the Company's common stock upon vesting. The performance-based stock units granted in 2021 vested on December 31, 2024 based upon i) continued service through the vesting date and (ii) the achievement of specific clinical development and regulatory performance events, as approved by the compensation committee. PSU awards are valued at the market price of the Company's common stock on the date of grant. The Company recognizes stock-based compensation expense for the fair value of these PSUs for the awards that are probable of vesting over the service period. During each financial period, management estimates the probable number of PSU’s that would vest until the ultimate achievement of the performance goal is known. In the year ended December 31, 2024, 581,346 of the PSUs granted in 2021 vested.

In April 2024, the Company approved a maximum of 340,000 PSUs to employees under the 2017 Plan. Each PSU represents a right to receive one share of the Company's common stock upon vesting. The performance-based stock units approved in 2024 will vest based the achievement of the following milestones i) continued service through the the date on which the Compensation Committee determines that the performance metrics have been achieved, ii) the achievement of annual net product revenue amounts, and iii) the achievement of specific clinical development and regulatory performance events, as approved by the compensation committee. The Company recognizes stock-based compensation expense for the fair value of these PSUs for the awards that are probable of vesting over the service period. The Company granted 45,322 PSU’s during the year ended December 31, 2025. During each financial period, management estimates the probable number of PSU’s that would vest until the ultimate achievement of the performance goal is known. As of December 31, 2025 none of these PSU’s have vested.

A summary of the Company's performance stock unit activity for the year ended December 31, 2025 is as follows:


	Number of PSUs	Weighted- Average Grant Date Fair Value
Unvested as of December 31, 2024	249,322	$	13.24
Granted	45,322	56.60
Vested	—	—
Cancelled	—	—
Unvested as of December 31, 2025	294,644	$	41.11

The following table summarizes the classification of the Company's stock-based compensation expenses related to stock options, restricted stock units, performance stock units and the employee stock purchase plan recognized in the Company's consolidated statements of operations and comprehensive loss (in thousands).


	Year Ended
	December 31,
	2025		2024		2023
Research and development	$	19,802	$	10,514	$	8,449
Selling, general, and administrative	47,016		29,168		24,104
Total	$	66,818	$	39,682	$	32,553

Stock-based compensation expense by award type recognized during the years ended December 31, 2025, 2024 and 2023 was as follows (in thousands):


	Year Ended
	December 31,
	2025		2024		2023
Stock options	$	22,225	$	21,788	$	17,671
Employees stock purchase plan	685		538		581
Restricted stock units	39,256		16,886		5,998
Performance stock units	4,652		470		8,303
Total	$	66,818	$	39,682	$	32,553

As of December 31, 2025, the Company has unrecognized compensation cost of $42.6 million related to non-vested employee, non-employee and director stock option awards under all equity plans that are expected to be recognized over a weighted-average period of 2.09 years. The Company has unrecognized compensation cost of $126.1 million related to non-vested employee restricted stock unit and performance stock unit awards under all equity plans that are expected to be recognized over a weighted-average period of 2.45 years.

Summary of Significant Accounting Policies - Property and Equipment (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Property and equipment
Property and equipment, gross	$ 6,353	$ 5,399
Less accumulated depreciation and amortization	(5,249)	(4,767)
Property and equipment, net	1,104	632
Depreciation and amortization expense	500	700	$ 900
Leasehold improvements
Property and equipment
Property and equipment, gross	$ 2,733	2,705
Office equipment
Property and equipment
Useful Life	5 years
Property and equipment, gross	$ 154	154
Computers and software
Property and equipment
Useful Life	3 years
Property and equipment, gross	$ 1,291	1,291
Furniture, fixtures and equipment
Property and equipment
Useful Life	5 years
Property and equipment, gross	$ 1,249	1,249
Construction in progress
Property and equipment
Property and equipment, gross	$ 926	$ 0

Income Taxes - Components of provision for income taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Current:
U.S. Federal	$ 0	$ 0	$ 0
U.S. State and Local	0	0	1
Foreign	497	346	563
Total Current Expense	497	346	564
Deferred:
U.S. Federal	0	0	0
U.S. State and Local	0	0	0
Foreign	0	0	0
Total Deferred Expense	$ 0	$ 0	$ 0

Long-Term Obligations	12 Months Ended
Long-Term Obligations	Dec. 31, 2025
Debt Disclosure [Abstract]
Long-Term Obligations	Long-Term Obligations On June 16, 2022, we entered into a RIFA with entities managed by HealthCare Royalty Management, LLC, collectively referred to as the Investors. Pursuant to the RIFA and subject to customary closing conditions, the Investors have agreed to pay the Company an aggregate investment amount of up to $100.0 million, or the Investment Amount. Under the terms of the RIFA, we received $37.5 million on June 29, 2022 upon FDA approval of IMCIVREE in BBS, referred to as the Initial Investment Amount, and we received an additional $37.5 million on September 29, 2022 of the Investment Amount upon EMA approval for BBS. On September 12, 2023, we received the remaining $24.4 million of the Investment Amount, net of debt issuance costs, following the achievement of a specified amount of cumulative net sales of IMCIVREE between July 1, 2022 and September 30, 2023. As consideration for the Investment Amount and pursuant to the RIFA, we agreed to pay the Investors a tiered royalty on our annual net revenues, or Revenue Interest, including worldwide net product sales and upfront payments and milestones. The applicable tiered percentage will initially be 11.5% on annual net revenues up to $125.0 million, 7.5% on annual net revenues of between $125.0 million and $300.0 million and 2.5% on annual net revenues exceeding $300.0 million. If the Investors have not received cumulative minimum payments equal to 60% of the amount funded by the Investors to date by March 31, 2027, or 120% of the amount funded by the Investors to date by March 31, 2029, we must make a cash payment immediately following each applicable date to the Investors sufficient to gross the Investors up to such minimum amounts after giving full consideration of the cumulative amounts paid by us to the Investors through each date, referred to as the Under Performance Payment. As the repayment of the funded amount is contingent upon worldwide net product sales and upfront payments, milestones, and royalties, the repayment term may be shortened or extended depending on actual worldwide net product sales and upfront payments, milestones, and royalties. As of December 31, 2025 we have made $40.3 million of payments. The Investors’ rights to receive the Revenue Interests will terminate on the date on which the Investors have received payments equal to a certain percentage of the funded portion of the Investment Amount including the aggregate of all payments made to the Investors as of such date, each percentage tier referred to as the Hard Cap, unless the RIFA is earlier terminated. The total Revenue Interests payable by us to the Investors is capped between 185% and 250% of the Investment Amount paid, dependent on the aggregate royalty paid between 2028 and 2032. If a change of control of occurs, the Investors may accelerate payments due under the RIFA up to the Hard Cap plus any other obligations payable under the RIFA. The repayment period commenced on July 8, 2022 for the Initial Investment Amount, and expires on the earlier of (i) the date at which the Investors received cash payments totaling an aggregate of a Hard Cap ranging from 185% to 250% of the Initial Investment Amount or (ii) the legal maturity date of July 8, 2034. If the Investors have not received payments equal to 250% of the Investment Amount by the twelve-year anniversary of the initial closing date, we will be required to pay an amount equal to the Investment Amount plus a specific annual rate of return less payments previously received by Investors. In the event of a change of control, we are obligated to pay Investors an amount equal to the Hard Cap in effect at the time, ranging from 185% to 250% plus any Under Performance Payment of the Investment Amount less payments previously received by Investors. In addition, upon the occurrence of an event of default, including, among others, our failure to pay any amounts due to Investors under the deferred royalty obligation, insolvency, our failure to pay indebtedness when due, the revocation of regulatory approval of IMCIVREE in the U.S. or our breach of any covenant contained in the RIFA and our failure to cure the breach within the prescribed time frame, we are obligated to pay Investors an amount equal to the Hard Cap in effect at the time of default ranging from 185% to 250% plus any Under Performance Payment of the Investment Amount less payments previously received by Investors. In addition, upon an event of default, Investors may exercise all other rights and remedies available under the RIFA, including foreclosing on the collateral that was pledged to Investors, which consists of all of our present and future assets relating to IMCIVREE. We have evaluated the terms of the RIFA and concluded that the features are similar to those of a debt instrument. Accordingly, we have accounted for the transaction as long-term debt and presented it as a deferred royalty obligation on our consolidated balance sheets. We have further evaluated the terms of the RIFA and determined that the repayment of the Hard Cap in effect at the time which ranges from 185% to 250% of the Investment Amount, less any payments made to date, upon a change of control is an embedded derivative that requires bifurcation from the debt instrument and fair value recognition. We determined the fair value of the derivative using an option pricing Monte Carlo simulation model taking into account the probability of change of control occurring and potential repayment amounts and timing of such payments that would result under various scenarios, as further described in Note 5, Fair Value of Financial Instruments, to our consolidated financial statements. During the second quarter of 2022, the Company recorded $1.6 million for the initial fair value of the embedded derivative liability (asset). The fair value of the embedded derivative asset was $(1.2) million and $(0.3) million as of December 31, 2025 and December 31, 2024, respectively. We will remeasure the embedded derivative to fair value each reporting period until the time the features lapse and/or termination of the deferred royalty obligation. For the years ended December 31, 2025 and December 31, 2024, we recognized other income of $0.9 million and $1.4 million, due to the remeasurement of the embedded derivative liability (asset). The carrying value of the deferred royalty obligation at December 31, 2025 was $108.2 million based on $100.0 million of proceeds, net of the initial fair value of the bifurcated embedded derivative liability upon execution of the RIFA, and debt issuance costs incurred. The carrying value is classified as $7.3 million within current liabilities and $100.9 million within long-term liabilities on the consolidated balance sheet as of December 31, 2025. The carrying value of the deferred royalty obligation approximated fair value at December 31, 2025. The effective interest rate as of December 31, 2025 was 17.05%. In connection with the deferred royalty obligation, we incurred debt issuance costs totaling $3.3 million. Debt issuance costs have been netted against the debt and are being amortized over the estimated term of the debt using the effective interest method, adjusted on a prospective basis for changes in the underlying assumptions and inputs. The assumptions used in determining the expected repayment term of the debt and amortization period of the issuance costs requires that we make estimates that could impact the classification of these costs, as well as the period over which these costs will be amortized.

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 8A
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480434/815-10-50-8A

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480738/235-10-S50-1

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(c))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480678/235-10-S99-1

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481544/470-10-50-6

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481544/470-10-50-6

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 405
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477092/405-40-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 405
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)(3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477092/405-40-50-1

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 405
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)(2)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477092/405-40-50-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 405
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)(1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477092/405-40-50-1

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 405
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477092/405-40-50-1

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 12: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 470
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org/470/tableOfContent

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 835
-SubTopic 30
-Name Accounting Standards Codification
-Section 45
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482925/835-30-45-2

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (i)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1B

Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1C

Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1C

Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1C

Reference 20: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1E
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1E

Reference 21: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1I
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1I

Reference 22: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1I
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1I

Reference 23: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Paragraph 1I
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481139/470-20-50-1I

+ Details

Subsequent Events	12 Months Ended
Subsequent Events	Dec. 31, 2025
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. The Company has evaluated all subsequent events and determined that there are no material recognized or unrecognized subsequent events requiring disclosure, other than as disclosed within the above notes to these consolidated financial statements, and except as described below. On January 26, 2026, and in accordance with the terms described below, the same holder referred to in Note 9, Series A Convertible Preferred Stock, exercised its right to convert another 17,500 shares of Series A Convertible Preferred Stock to common shares. Each of the 17,500 preferred shares converted to 20.8333 shares of common stock, resulting in a total of 364,582 shares of common stock. The Company reclassified the carrying value of the converted shares of $17.4 million from Series A convertible Preferred Stock to common stock and additional paid in capital.

Insider Trading Policies and Procedures	12 Months Ended
Insider Trading Policies and Procedures	Dec. 31, 2025
Insider Trading Policies and Procedures [Line Items]
Insider Trading Policies and Procedures Adopted	true

Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Payables and Accruals [Abstract]
Research and development costs	$ 17,084	$ 17,871
Professional fees	5,827	4,280
Payroll related	22,433	18,216
Royalties	2,870	2,091
Sales allowances	31,564	15,850
Other	4,078	4,350
Accrued expenses and other current liabilities	$ 83,855	$ 62,658

Stock-based Compensation - RSU and PSU Narrative (Details) - USD ($) $ in Thousands	1 Months Ended		12 Months Ended
	Apr. 30, 2024	Nov. 30, 2021	Dec. 31, 2025	Dec. 31, 2024
2017 Equity Incentive Plan \| Employees, directors and nonemployees
Stock-based Compensation
Unrecognized compensation expense related to non-vested portion of awards			$ 42,600
Weighted average period for recognition of unrecognized compensation expense			2 years 1 month 2 days
2017 Equity Incentive Plan \| Employee
Stock-based Compensation
Unrecognized compensation expense related to non-vested portion of awards			$ 126,100
Weighted average period for recognition of unrecognized compensation expense			2 years 5 months 12 days
Restricted stock units
Stock-based Compensation
Aggregate intrinsic value			$ 308,800
Number of shares released upon vesting			1
Vested (in shares)			635,186
Granted (in shares)			1,771,564
Performance stock units
Stock-based Compensation
Number of shares released upon vesting		1
Number of shares of common stock shares issued upon vesting (in shares)		956,145
Vested (in shares)			0
Granted (in shares)			45,322
Performance stock units \| 2017 Equity Incentive Plan
Stock-based Compensation
Number of shares released upon vesting	1
Number of shares of common stock shares issued upon vesting (in shares)	340,000
Performance stock units \| Granted in 2021
Stock-based Compensation
Vested (in shares)				581,346
Performance stock units \| Granted in 2024
Stock-based Compensation
Vested (in shares)			0

Stock-based Compensation - Common stock option activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Number of Options
Outstanding at beginning of the period (in shares)	6,611,391
Granted (in shares)	740,919
Exercised (in shares)	(843,525)
Cancelled (in shares)	(42,607)
Outstanding at end of the period (in shares)	6,466,178	6,611,391
Options exercisable at end of the period (in shares)	4,940,330
Weighted-Average Exercise Price
Outstanding at beginning of the period (in USD per share)	$ 23.29
Granted (in USD per share)	57.70
Exercised (in USD per share)	16.13
Cancelled (in USD per share)	26.03
Outstanding at end of the period (in USD per share)	28.15	$ 23.29
Options exercisable at end of the period (in USD per share)	$ 23.09
Weighted-Average Remaining Contractual Term
Outstanding at end of the period	6 years 1 month 13 days	6 years 7 months 28 days
Options exercisable at end of the period	5 years 5 months 23 days
Aggregate Intrinsic Value
Exercised	$ 52,980
Options vested and expected to vest	510,138	$ 216,413
Options exercisable	$ 414,757
2017 Equity Incentive Plan
Number of Options
Granted (in shares)	740,919	926,543	842,528

Significant Agreements - Ipsen Pharma S.A.S. (Details) - Ipsen Pharma S.A.S. - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023	Mar. 31, 2022	Mar. 31, 2021	Feb. 26, 2010
License agreement
Significant Agreements
Amount of commercial milestone capitalized				$ 4,000	$ 5,000
Milestone expenses	$ 0	$ 0	$ 0
Maximum \| License agreement
Significant Agreements
Aggregate payment upon achievement of development and commercial milestones						$ 40,000
Maximum \| Sublicense agreement
Significant Agreements
Payment based on revenue received, as percentage						20.00%
Minimum \| Sublicense agreement
Significant Agreements
Payment based on revenue received, as percentage						10.00%

Stock-based Compensation - 2017 Equity Incentive Plan (Details) - 2017 Equity Incentive Plan - shares					12 Months Ended
	Jan. 01, 2026	Jan. 01, 2025	Jan. 01, 2024	Jan. 01, 2023	Dec. 31, 2025
Stock-based Compensation
Percentage increase in authorized shares under the plan					4.00%
Number of additional shares added (in shares)		2,495,626	2,377,062	2,264,497
Authorized shares (in shares)					13,717,040
Shares available for future grant (in shares)					4,636,850
Common stock reserved for issuance (in shares)					9,080,190
Subsequent events
Stock-based Compensation
Number of additional shares added (in shares)	2,688,212
Exercise Price Not Less Than 100% Of Fair Market Value
Stock-based Compensation
Exercise price of incentive stock options over fair market value of the common stock					100.00%
Voting power of stock					10.00%
Exercise Price Not Less Than 110% Of Fair Market Value
Stock-based Compensation
Exercise price of incentive stock options over fair market value of the common stock					110.00%
Voting power of stock					10.00%

Summary of Significant Accounting Policies - Product Revenue Allowance and Reserve Categories (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
Product revenue allowance and reserve categories
Beginning Balance	$ 16,103	$ 9,674
Provision related to sales in the current year	49,481	29,632
Credit and payments made	(33,717)	(23,203)
Ending balance	31,867	16,103
Provision for Cash Discounts
Product revenue allowance and reserve categories
Beginning Balance	253	199
Provision related to sales in the current year	3,670	2,317
Credit and payments made	(3,620)	(2,263)
Ending balance	303	253
Fees, Rebates and Other Incentives
Product revenue allowance and reserve categories
Beginning Balance	15,850	9,475
Provision related to sales in the current year	45,811	27,315
Credit and payments made	(30,097)	(20,940)
Ending balance	$ 31,564	$ 15,850

Stock-based Compensation - RSU and PSU activity (Details)	12 Months Ended
	Dec. 31, 2025 $ / shares shares
Restricted stock units
Number of Units
Outstanding unvested at beginning of the period (in shares) \| shares	1,826,709
Granted (in shares) \| shares	1,771,564
Vested (in shares) \| shares	(635,186)
Cancelled (in shares) \| shares	(78,152)
Outstanding unvested at end of the period (in shares) \| shares	2,884,935
Weighted Average Grant Date Fair Value
Outstanding unvested at beginning of the period (in USD per share) \| $ / shares	$ 36.40
Granted (in USD per share) \| $ / shares	65.24
Vested (in USD per share) \| $ / shares	33.36
Cancelled (in USD per share) \| $ / shares	47.84
Outstanding unvested at end of the period (in USD per share) \| $ / shares	$ 54.42
Performance stock units
Number of Units
Outstanding unvested at beginning of the period (in shares) \| shares	249,322
Granted (in shares) \| shares	45,322
Vested (in shares) \| shares	0
Cancelled (in shares) \| shares	0
Outstanding unvested at end of the period (in shares) \| shares	294,644
Weighted Average Grant Date Fair Value
Outstanding unvested at beginning of the period (in USD per share) \| $ / shares	$ 13.24
Granted (in USD per share) \| $ / shares	56.60
Vested (in USD per share) \| $ / shares	0
Cancelled (in USD per share) \| $ / shares	0
Outstanding unvested at end of the period (in USD per share) \| $ / shares	$ 41.11

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2025
Audit Information [Abstract]
Auditor Firm ID	42
Auditor Name	Ernst & Young LLP
Auditor Location	Boston, Massachusetts

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Schedule of product revenue allowance and reserve categories

The table below summarizes balances and activity in each of the product revenue allowance and reserve categories as follows (in thousands):


	Provision for Cash Discounts		Fees, Rebates and Other Incentives		Total



Beginning Balance at December 31, 2023	$	199	$	9,475	$	9,674
Provision related to sales in the current year	2,317		27,315		29,632
Credit and payments made	(2,263)		(20,940)		(23,203)
Ending balance December 31, 2024	$	253	$	15,850	$	16,103
Provision related to sales in the current year	3,670		45,811		49,481
Credit and payments made	(3,620)		(30,097)		(33,717)
Ending balance December 31, 2025	$	303	$	31,564	$	31,867

Schedule of inventory

Inventory consists of the following (in thousands):


	December 31, 2025		December 31, 2024
Raw Materials	$	6,745	$	6,776
WIP	1,631		1,250
Finished Goods	17,377		10,715
Total Inventory	$	25,753	$	18,741

Schedule of prepaid expenses and other current assets

Prepaid expenses and other current assets consists of the following (in thousands):


	December 31,
	2025		2024
Prepaid research and development costs	$	16,166	$	7,580
Other current assets	9,967		8,802
Prepaid expenses and other current assets	$	26,133	$	16,382

Schedule of other long-term assets

Other long-term assets consists of the following (in thousands):


	December 31,
	2025		2024
Long-term research and development costs	$	1,660	$	6,209
Other long-term assets	1,626		1,117
Other long-term assets	$	3,286	$	7,326

Schedule of property, plant and equipment

Property and equipment consists of the following (in thousands):


	Useful Life	December 31,
		2025		2024
Leasehold improvements	*	$	2,733	$	2,705
Office equipment	5 years	154		154
Computers and software	3 years	1,291		1,291
Furniture, fixtures and equipment	5 years	1,249		1,249
Construction in progress		926		—
Property and equipment, gross		6,353		5,399
Less accumulated depreciation and amortization		(5,249)		(4,767)
Property and equipment, net		$	1,104	$	632

*Shorter of asset life or lease term.

Schedule of potential common shares, presented based on amounts outstanding at each period end, excluded from computation of diluted net loss per share attributable to common stockholders

The following table includes the potential common shares, presented based on amounts outstanding at each period end, that were excluded from the computation of diluted net loss per share due to their anti-dilutive effect, under either the treasury stock or if-converted method for the periods indicated (in thousands):


	Year Ended December 31,
	2025	2024	2023
Stock options	6,466,178	6,611,391	6,551,025
Restricted stock units	2,884,935	1,805,412	1,079,382
Performance stock units	294,644	249,322	581,246
Common stock reserved for the conversion of Series A convertible preferred stock	2,760,413	3,124,995	—
Potential common shares	12,406,170	11,791,120	8,211,653

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Basis of Presentation

The Company's consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”).

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even if such assumptions are reasonable when made. Significant estimates relied upon in preparing these financial statements include but are not limited to, estimates related to determining our net product revenue, and accruals related to research and development expenses. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ materially from those estimates.

Principles of Consolidation

The consolidated financial statements include the accounts of Rhythm Pharmaceuticals, Inc. and its wholly-owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation.

Segment Information

Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company currently operates in two business segments, which are U.S. and international segments for the development and commercialization of therapies for patients with rare diseases. A single management team that reports to the Chief Executive Officer comprehensively manages the entire business. The Company meets the aggregation criteria of ASC 280 and therefore has one reportable segment for the year ended December 31, 2025.

In November 2023, the FASB issued ASU 2023-07 – Segment Reporting (Topic 280) – Improvements to Reportable Segment Disclosures, which improves segment disclosure requirements, primarily through enhanced disclosure requirements for significant segment expenses. The improved disclosure requirements apply to all public entities that are required to report segment information, including those with only one reportable segment. The Company adopted the guidance in the fiscal year beginning January 1, 2024. There was no impact on the Company’s reportable segments identified and additional required disclosures have been included in Note 15.

Off-Balance Sheet Risk and Concentrations of Credit Risk

Financial instruments, which potentially subject the Company to significant concentration of credit risk, consist primarily of cash and cash equivalents and short-term investments, which are maintained at two federally insured financial institutions. The deposits held at these two institutions are in excess of federally insured limits. The Company has not experienced any losses in such accounts and management believes that the Company is not exposed to significant credit risk due to the financial position of the depository institutions in which those deposits are held. The Company has no off-balance sheet risk, such as foreign exchange contracts, option contracts, or other foreign hedging arrangements.

The Company is exposed to risks associated with extending credit to customers related to the sale of products. The Company does not require collateral to secure amounts due from its customers. For the years ended December 31, 2025 and 2024, approximately 69% and 74%, respectively, of all of the Company’s revenue was generated from a single customer in the United States. Additionally, a single international customer also generated approximately 10% of the Company's revenue during the year ended December 31, 2025. As of December 31, 2025 and 2024, approximately 56% and 67%, respectively, of the Company’s accounts receivable was outstanding from a single customer in the United States.

The Company relies on third-party manufacturers and suppliers for manufacturing and supply of its product. The inability of the suppliers or manufacturers to fulfill supply requirements of the Company could materially impact future operating results. A change in the relationship with the suppliers or manufacturer, or an adverse change in their business, could materially impact future operating results.

The Company relies on separate third parties to perform genetic testing in the United States and Europe, respectively. The inability of the vendor to fulfill testing services for the Company could materially impact future operating results and adversely impact our ability to further develop setmelanotide. A change in the relationship with the genetic testing service providers, or an adverse change in their business, could materially impact future operating results.

Cash and Cash Equivalents

The Company considers all highly liquid investments with remaining maturity from the date of purchase of three months or less to be cash equivalents. Cash and cash equivalents includes bank demand deposits, U.S. treasury bills and money market funds that invest primarily in U.S. government treasuries.

Short-Term Investments

Short-term investments consist of investments with maturities greater than 90 days, as of the date of purchase. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such marketable securities represent the investment of cash that is available for current operations. The Company considers its investment portfolio available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices. Unrealized gains and losses are reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity. To the extent the amortized cost basis of the available-for-sale debt securities exceeds the fair value, management assesses the debt securities for credit loss; however, management considers the risk of credit loss to be minimized by the Company's policy of investing in financial instruments issued by highly-rated financial institutions. When assessing the risk of credit loss, management considers factors such as the severity and the reason of the decline in value (i.e., any changes to the rating of the security by a rating agency or other adverse conditions specifically related to the security) and management's intended holding period and time horizon for selling. During the years ended December 31, 2025, 2024, and 2023, the Company did not recognize any credit losses related to its available-for-sale debt securities. Further, as of December 31, 2025 and 2024, the Company did not record an allowance for credit losses related to its available-for-sale debt securities.

Restricted Cash

Restricted cash consists of security deposits in the form of letters of credit placed in separate restricted bank accounts as required under the terms of the Company’s lease arrangement for its corporate office in Boston, Massachusetts and the Company’s corporate travel credit card.

Accounts Receivable, net

Accounts receivable consists of amounts due from customers, net of customer allowances for cash discounts and any estimated expected credit losses. The Company's measurement of expected credit losses is based on relevant information about past events, including historical experience, current conditions, and reasonable and supportable forecasts that affect the collectability of the reported amount. To date, the Company has not experienced any material credit losses. The Company's contracts with its customers have customary payment terms that generally require payment within 90 days. The Company analyzes amounts that are past due for collectability, and periodically evaluates the creditworthiness of its customer. At December 31, 2025 and 2024, the Company determined an allowance for doubtful account was not required based upon its review of contractual payments and its customer payment histories.

Revenue Recognition

The Company recognizes revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers, or ASC 606. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services.

Product Revenue, Net

In the United States, which accounts for the largest portion of our total revenues, the Company sells its product through a specialty pharmacy. The product is distributed through third-party logistics, or 3PL, distribution agent that does not take title to the product. Once the product is delivered to the Company’s specialty pharmacy provider, our customer in the United States, the customer (or “wholesaler”) takes title to the product. The wholesaler then distributes the product to patients. In our distribution agreement with the 3PL company, the Company acts as principal because we retain control of the product. Internationally, we make sales primarily to specialty distributors and retail pharmacy chains, as well as hospitals, many of which are government-owned or supported. The Company offers returns of product sold to the customer on a limited basis, however, no material returns have been recognized to date.

Revenue from product sales is recognized when the customer obtains control of our product, which occurs at a point in time, upon transfer of title to the customer because at that point in time we have no ongoing obligations to the customer. There are no other performance obligations besides the sale of product. We classify payments to our customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses in our consolidated statements of operations and comprehensive loss. Otherwise, payments to a customer or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. Taxes collected from the customer relating to product sales and remitted to governmental authorities are excluded from revenue. Because our payment terms are generally ninety days or less, the Company concluded there is not a significant financing component because the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. The Company expenses incremental costs of obtaining a contract as and when incurred since the expected amortization period of the asset that we would have recognized is one year or less.

Reserves for Variable Consideration

Revenues from product sales are recorded at the net sales price, or the transaction price, which includes estimates of variable consideration for which reserves are established and which result from discounts, rebates, and co-pay assistance that are offered within contracts between us and our customers, health care providers and other indirect customers relating to the sale of IMCIVREE. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known.

The following are the components of variable consideration related to product revenue:

Government rebates: The Company is subject to discount obligations under government programs, including U.S. Medicaid programs, Medicare and Tricare in the United States as well as certain government rebates and pricing adjustments in certain international markets that we operate. We estimate these rebates based upon a range of possible outcomes that take into consideration among other things, historical experience of claims submitted by the various states and jurisdictions, contractual rebate rates, estimated lag time of the rebate invoices and the estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses and other current liabilities on our consolidated balance sheets. On a quarterly basis, we update our estimates and record any necessary adjustments in the period that we identify the adjustments.

Trade discounts and allowances: The Company provides customary invoice discounts on IMCIVREE sales to certain of our customers for items such as prompt payment. These are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive and pay for various distribution services from our customers in the distribution channel. For services that are not distinct from the sale of our product, such fees are classified as a reduction of product revenue.

Product returns: Our customers have limited return rights related to the product’s damage or defect. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue

and a refund liability in the period the related product revenue is recognized. Based on the distribution model for IMCIVREE, the Company believes there will be minimal returns and these reserves have not been material to date.

Other incentives: Other incentives include co-payment assistance the Company provides to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. The estimate is recorded as a reduction of revenue in the same period the related revenue is recognized.

Provisions for trade discounts, chargebacks and allowances are recorded as reductions to accounts receivable, and returns, government rebates, and other incentives are recorded as a component of accrued expenses.

The table below summarizes balances and activity in each of the product revenue allowance and reserve categories as follows (in thousands):


	Provision for Cash Discounts		Fees, Rebates and Other Incentives		Total



Beginning Balance at December 31, 2023	$	199	$	9,475	$	9,674
Provision related to sales in the current year	2,317		27,315		29,632
Credit and payments made	(2,263)		(20,940)		(23,203)
Ending balance December 31, 2024	$	253	$	15,850	$	16,103
Provision related to sales in the current year	3,670		45,811		49,481
Credit and payments made	(3,620)		(30,097)		(33,717)
Ending balance December 31, 2025	$	303	$	31,564	$	31,867

Provision for cash discounts are recorded as reductions of accounts receivable, and fees, rebates, and other incentives are recorded as components of accrued expenses.

License Agreements

We generate revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of our products and product candidates. Such agreements may include the transfer of intellectual property rights in the form of licenses, transfer of technological know-how, delivery of drug substances, research and development services, and participation on certain committees with the counterparty. Payments made by the customers may include non-refundable upfront fees, payments upon the exercise of customer options, payments based upon the achievement of defined milestones, and royalties on sales of products and product candidates if they are approved and commercialized.

If a license to our intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, we recognize the transaction price allocated to the license as revenue upon transfer of control of the license. We evaluate all other promised goods or services in the agreement to determine if they are distinct. If they are not distinct, they are combined with other promised goods or services to create a bundle of promised goods or services that is distinct. Optional future services where any additional consideration paid to us reflects their standalone selling prices do not provide the customer with a material right and, therefore, are not considered performance obligations. If optional future services are priced in a manner which provides the customer with a significant or incremental discount, they are material rights, and are accounted for as separate performance obligations.

We utilize judgment to determine the transaction price. In connection therewith, we evaluate contingent milestones at contract inception to estimate the amount which is not probable of a material reversal to include in the transaction price using the most likely amount method. Milestone payments that are not within our control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore the variable consideration is constrained. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which we recognize revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, we re-evaluate the probability of achieving development milestone payments that may not be subject to a material reversal and, if necessary, adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect license and other revenue, as well as earnings, in the period of adjustment.

We then determine whether the performance obligations or combined performance obligations are satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. We evaluate the measure of progress, as applicable, for each reporting period and, if necessary, adjust the measure of performance and related revenue recognition.

When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded within deferred revenue. Contract liabilities within deferred revenue are recognized as revenue after control of the goods or services is transferred to the customer and all revenue recognition criteria have been met.

For arrangements that include sales-based royalties, including sales-based milestone payments, and a license of intellectual property that is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of when the related sales occur or when the performance obligation to which some or all of the royalties have been allocated has been satisfied (or partially satisfied). Refer to Note 10, Significant Agreements, for discussion related to the Company’s accounting for the RareStone Group, Ltd. agreement.

Deferred Royalty Obligation

We treat the debt obligation to HealthCare Royalty Management, LLC as discussed further in Note 11, “Long-Term Obligations”, as a deferred royalty obligation, amortized using the effective interest rate method over the estimated life of the revenue streams. We recognize interest expense thereon using the effective rate, which is based on our current estimates of future revenues over the life of the arrangement. In connection therewith, we periodically assess our expected revenues using internal projections, impute interest on the carrying value of the deferred royalty obligation, and record interest expense using the imputed effective interest rate. To the extent our estimates of future revenues are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will account for any such changes by adjusting the effective interest rate on a prospective basis, with a corresponding impact to the reclassification of our deferred royalty obligation. The assumptions used in determining the expected repayment term of the deferred royalty obligation and amortization period of the issuance costs requires that we make estimates that could impact the classification of such costs, as well as the period over which such costs will be amortized.

Inventory

Prior to receiving approval from the FDA in November 2020 to sell IMCIVREE in the United States, the Company expensed all costs incurred related to the manufacture of IMCIVREE as research and development expense because of the inherent risks associated with the development of a drug candidate, the uncertainty about the regulatory approval process and the lack of history for the Company of regulatory approval of drug candidates. The Company values inventories at the lower of cost or estimated net realizable value. The Company determines the cost of inventories, which includes amounts related to materials and manufacturing overhead, on a first-in, first-out basis. Raw materials and work in process includes all inventory costs prior to packaging and labelling, including raw materials, active pharmaceutical ingredient, and drug product. Finished goods include packaged and labelled products. Raw materials and work in process that may be used for either research and development or commercial sale are classified as inventory until the material is consumed or otherwise allocated for research and development. If the material is intended to be used for research and development, it is expensed as research and development once that determination is made.

Inventory consists of the following (in thousands):


	December 31, 2025		December 31, 2024
Raw Materials	$	6,745	$	6,776
WIP	1,631		1,250
Finished Goods	17,377		10,715
Total Inventory	$	25,753	$	18,741

Cost of Product Sales

Cost of product sales consists of manufacturing costs, transportation and freight, amortization of capitalized intangibles, royalty payments and indirect overhead costs associated with the manufacturing and distribution of IMCIVREE. Cost of product sales may also include periodic costs related to certain manufacturing services and inventory

adjustment charges. Finally, cost of sales may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances.

Intangible Assets, net

Definite-lived intangible assets related to capitalized milestones under license agreements are amortized on a straight-line basis over their remaining useful lives, which are estimated to be the remaining patent life. If our estimate of the product’s useful life is shorter than the remaining patent life, then a shorter period is used. Amortization expense is recorded as a component in cost of sales in the consolidated statements of operations and comprehensive loss.

Impairment of Long-Lived Assets

The Company evaluates its long-lived assets, which consist primarily of property and equipment and finite lived intangible assets, for impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Factors that the Company considers in deciding when to perform an impairment review include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. The Company measures recoverability of assets to be held and used by comparing the carrying amount of an asset to the future undiscounted net cash flows expected to be generated by the asset. If such assets are considered to be impaired, the Company measures the impairment to be recognized by the amount by which the carrying amount of the asset exceeds the fair value of the asset, less the cost to sell. No events or changes in circumstances existed to require an impairment assessment during the years ended December 31, 2025, 2024 and 2023.

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consist primarily of costs incurred in advance of services being received, including services related to clinical trial programs. Prepaid expenses and other current assets consists of the following (in thousands):


	December 31,
	2025		2024
Prepaid research and development costs	$	16,166	$	7,580
Other current assets	9,967		8,802
Prepaid expenses and other current assets	$	26,133	$	16,382

Other Long-Term Assets

Other long-term assets consist primarily of costs incurred in advance of services being received, including services related to clinical trial programs. Since the Company will not receive services within one year of the balance sheet date, these assets are considered long-term. Other long-term assets consists of the following (in thousands):


	December 31,
	2025		2024
Long-term research and development costs	$	1,660	$	6,209
Other long-term assets	1,626		1,117
Other long-term assets	$	3,286	$	7,326

Property and Equipment

Property and equipment consists of the following (in thousands):


	Useful Life	December 31,
		2025		2024
Leasehold improvements	*	$	2,733	$	2,705
Office equipment	5 years	154		154
Computers and software	3 years	1,291		1,291
Furniture, fixtures and equipment	5 years	1,249		1,249
Construction in progress		926		—
Property and equipment, gross		6,353		5,399
Less accumulated depreciation and amortization		(5,249)		(4,767)
Property and equipment, net		$	1,104	$	632

*Shorter of asset life or lease term.

Depreciation and amortization expense related to property and equipment for the years ended December 31, 2025, 2024 and 2023 was $0.5 million, $0.7 million, and $0.9 million respectively.

Property and equipment are recorded at cost. Depreciation and amortization is calculated using the straight-line method over the estimated useful lives of the assets. Upon disposal, retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in the results of operations. Expenditures for repairs and maintenance that do not improve or extend the lives of the respective assets are charged to expense as incurred.

Acquired IPR&D and Milestone Expense

In an asset acquisition, payments incurred prior to regulatory approval to acquire rights to in-process research and development projects are expensed as acquired IPR&D and recorded as a component of research and development expense in the consolidated statements of operations and comprehensive net loss unless the project has an alternative future use. These costs include upfront and development milestone payments related to licensing arrangements, or other asset acquisitions that provide rights to develop, manufacture and/or sell pharmaceutical products. Where contingent development milestone payments are due to third parties, prior to regulatory approval, the payment obligations are expensed when the milestone results are achieved. Regulatory and commercial milestone payments made to third parties subsequent to regulatory approval are capitalized as intangible assets and amortized to cost of products sold over the remaining useful life of the related product.

Foreign Currency Translation

The assets and liabilities of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars at exchange rates in effect at the balance sheet date. Revenue and expense amounts for these subsidiaries are translated using the average exchange rates for the period. Changes resulting from foreign currency translation are included in accumulated other comprehensive income (loss) on the Company’s consolidated statement of stockholders’ equity. Net foreign currency exchange transaction gains (losses), which are included in other (expense) income, net on our consolidated statements of operations, were immaterial for the years ended December 31, 2025, 2024 and 2023.

Fair Value Measurements

Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. Financial assets and liabilities carried at fair value are classified and disclosed in one of the following three categories:

Level 1 — Quoted market prices in active markets for identical assets or liabilities.

Level 2 — Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities.

Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.

The Company’s cash equivalents and marketable securities and derivative liability at December 31, 2025 and 2024 were carried at fair value, determined according to the fair value hierarchy. See Note 5 for further discussion.

The carrying amounts reflected in the consolidated balance sheets for accounts payable and accrued expenses and other current liabilities approximate their fair values due to their short-term maturities at December 31, 2025 and 2024, respectively.

Research and Development Expenses

Costs incurred in the research and development of the Company’s products are expensed to operations as incurred. Research and development expenses consist of costs incurred in performing research and development activities, including salaries and benefits, facilities costs, overhead costs, contract services and other outside costs. The value of goods and services received from contract research organizations, or CROs, or contract manufacturing organizations, or CMOs, in the reporting period are estimated based on the level of services performed and progress in the period for which the Company has not yet received an invoice from the supplier. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies or clinical trials, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates are made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs.

Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses, and expensed as the related goods are delivered or the services are performed.

Income Taxes

The Company is taxed as a C corporation for federal income tax purposes. Income taxes for the Company are recorded in accordance with FASB ASC Topic 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. Income taxes have been calculated on a separate tax return basis.

The Company accounts for income taxes under the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements. Under this method, the Company determines deferred tax assets and liabilities on the basis of the differences between the financial statement and tax bases of assets and liabilities by using enacted tax rates in effect for the year in which the differences are expected to reverse. The effect of a change in tax rates on deferred tax assets and liabilities is recognized in income in the period that includes the enactment date. The Company recognizes deferred tax assets to the extent that it believes that these assets are more likely than not to be realized. In making such a determination, the Company considers all available positive and negative evidence, including future reversals of existing taxable temporary differences, projected future taxable income, tax-planning strategies, and results of recent operations. If the Company determines that it would be able to realize its deferred tax assets in the future in excess of their net recorded amount, it would make an adjustment to the deferred tax asset valuation allowance, which would reduce the provision for income taxes.

The Company records uncertain tax positions in accordance with ASC 740 on the basis of a two-step process in which (1) it determines whether it is more likely than not that the tax positions will be sustained on the basis of the technical merits of the position and (2) for those tax positions that meet the more-likely-than-not recognition threshold, the Company recognizes the largest amount of tax benefit that is more than 50% likely to be realized upon ultimate settlement with the related tax authority.

The Company recognizes interest and penalties related to unrecognized tax benefits as provision for income taxes in the accompanying consolidated statements of operations. As of December 31, 2025 and 2024, no accrued interest or penalties are included as a component of accrued expenses in the consolidated balance sheets.

Net Loss Per Share

Basic net loss per share is computed by dividing net loss available to common shareholders by the weighted-average number of common shares outstanding during the period, without consideration of potential dilutive securities. Diluted net loss per share is computed by adjusting the weighted-average shares outstanding for the potential dilutive effects of common stock equivalents outstanding during the period calculated in accordance with the more dilutive of the if-converted or treasury stock method. For purposes of the diluted net loss per share calculation, stock options, restricted stock units and performance stock units are considered to be common stock equivalents but have been excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive for all periods presented. Therefore, basic and diluted net loss per share were the same for all periods presented.


	Year Ended December 31,
	2025	2024	2023
Stock options	6,466,178	6,611,391	6,551,025
Restricted stock units	2,884,935	1,805,412	1,079,382
Performance stock units	294,644	249,322	581,246
Common stock reserved for the conversion of Series A convertible preferred stock	2,760,413	3,124,995	—
Potential common shares	12,406,170	11,791,120	8,211,653

Comprehensive Loss

Comprehensive loss represents the net change in stockholders’ equity during a period from sources other than transactions with shareholders. As reflected in the accompanying consolidated statements of operations and comprehensive loss, our comprehensive loss is comprised of net losses, foreign currency translation adjustments and unrealized gains and losses on marketable debt securities. These changes in equity are reflected net of tax.

Patent Costs

Costs to secure and defend patents are expensed as incurred and are classified as general and administrative expenses. Patent costs were $2.0 million, $1.3 million and $0.6 million for the years ended December 31, 2025, 2024 and 2023, respectively.

Subsequent Events

The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. Subsequent events have been evaluated as required. See Note 16.

Application of New or Revised Accounting Standards

From time to time, new accounting pronouncements are issued by the FASB and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its financial position or results of operations upon adoption.

Recently adopted accounting pronouncements

In December 2024, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures, or ASU 2023-09. The new guidance requires that an entity, on an annual basis, disclose additional income tax information, primarily related to the rate reconciliation and income taxes paid. The amendments in the ASU are intended to enhance the transparency and decision usefulness of income tax disclosures. The amendments in this update are effective for us beginning in fiscal year ending December 31, 2025. The Company has adopted this update on a prospective basis. The adoption of this guidance resulted in expanded disclosures in its consolidated financial statements.

Recently issued accounting pronouncements, not yet adopted

In December 2025, the FASB issued ASU 2025-12 “Codification Improvements” to address suggestions received from stakeholders on the Accounting Standards Codification and to make other incremental improvements to U.S. GAAP. The update represents changes to the Codification that clarify, correct errors, or make minor improvements. The amendments make the Codification easier to understand and apply. The guidance is effective for fiscal years beginning after December 15, 2026, including interim periods within those fiscal years. The Company is in the process of evaluating this guidance to determine the impact it may have on its consolidated financial statements.

In December 2025, the FASB issued ASU 2025-11, “Interim Reporting (Topic 270) Narrow-Scope Improvements.” The amendments in this update provide clarity on interim disclosure requirements and the applicability of Topic 270. The amendments in this update also include a disclosure principle that requires entities to disclose events since the end of the last annual reporting period that have a material impact on the entity. The amendments in this ASU are required to be adopted for interim periods within annual reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is in the process of evaluating this guidance to determine the impact it may have on its consolidated financial statements.

In November 2024, the FASB issued ASU 2024-03 “Income Statement: Reporting Comprehensive Income—Expense Disaggregation Disclosures,” which requires more detailed information about specified categories of expenses (purchases of inventory, employee compensation, depreciation, amortization, and depletion) included in certain expense captions presented on the face of the income statement, as well as disclosures about selling expenses. ASU 2024-03 is effective for fiscal years beginning after December 15, 2026 and for interim periods within fiscal years beginning after December 15, 2027. Early adoption is permitted. The amendments may be applied either prospectively to financial statements issued for reporting periods after the effective date of this ASU or retrospectively to all prior periods presented in the financial statements. The Company is in the process of evaluating this guidance to determine the impact it may have on its consolidated financial statements disclosures.

Income Taxes - Deferred tax assets and liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Deferred tax assets:
Net operating loss carryforwards	$ 236,991	$ 167,375
Research and development credits	19,853	17,604
Orphan drug credit	46,211	34,603
Capitalized license fee	23,418	21,661
Stock-based compensation	8,573	5,113
Section 174 Costs	33,718	67,624
Deferred revenue	0	336
Section 163(j) Interest Limitation	5,589	4,417
Accrued Expenses & Other	7,811	7,971
Total deferred tax assets	382,164	326,704
Valuation allowance	(381,166)	(325,503)
Net deferred tax assets	998	1,201
Deferred tax liabilities:
Operating lease right-of-use asset and other	(998)	(1,201)
Net deferred tax liabilities	(998)	(1,201)
Total deferred tax assets / liabilities	$ 0	$ 0

Commitments and Contingencies	12 Months Ended
Commitments and Contingencies	Dec. 31, 2025
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	Commitments and Contingencies Legal Proceedings The Company, from time to time, may be party to various litigation arising in the ordinary course of business. The Company is not presently subject to any pending or threatened litigation that it believes, if determined adversely to the Company, individually, or taken together, would reasonably be expected to have a material adverse effect on its business or financial results. Other The Company is party to various agreements, principally relating to licensed technology, that require future payments relating to milestones that may be met in subsequent periods, or royalties on future sales of specified products. See Note 10 for discussion of these arrangements. Additionally, the Company is party to various contracts with CROs and CMOs that generally provide for termination on notice, with the exact amounts in the event of termination to be based on the timing of the termination and the terms of the agreement. Based on the Company’s current development plans as of December 31, 2025, the Company does not expect to make material milestone payments due to third parties during the next 12 months from the filing of this Annual Report on Form 10-K, in connection with our license agreements. These milestones generally become due and payable upon achievement of such milestones or sales and achievement of development milestones. When the achievement of these milestones or sales have not occurred, such contingencies are not recorded in the Company’s consolidated financial statements.

Summary of Significant Accounting Policies - Off-Balance Sheet Risk and Concentrations of Credit Risk (Details) - Institution	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
Product Information [Line Items]
Number of federally insured financial institutions	2
Revenue And Geographical \| Geographic Concentration Risk \| International
Product Information [Line Items]
Concentration risk, percentage	10.00%
Customer one \| Revenue benchmark \| Customer concentration risk
Product Information [Line Items]
Concentration risk, percentage	69.00%	74.00%
Customer one \| Accounts receivable \| Customer concentration risk
Product Information [Line Items]
Concentration risk, percentage	56.00%	67.00%

Fair Value of Financial Assets and Liabilities - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands		12 Months Ended
	Apr. 15, 2024	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023	Apr. 01, 2024
Series A Convertible Preferred Stock
Carrying amount of cash and cash equivalents and short term investments		$ 388,900	$ 320,600
Derivative Asset, Statement of Financial Position [Extensible Enumeration]		Deferred royalty obligation, current
Derivative liability		$ 0	0
Gain on settlement of forward contract		$ 0	8,900	$ 0
Minimum
Series A Convertible Preferred Stock
Fair value risk free rates (as a percent)	4.60%
Volatility (as a percent)	68.00%
Maximum
Series A Convertible Preferred Stock
Fair value risk free rates (as a percent)	5.00%
Volatility (as a percent)	67.00%
Convertible preferred stock \| Forward Contracts
Series A Convertible Preferred Stock
Consideration payable	$ 150,000
Derivative liability	$ 8,900				$ 0
Gain on settlement of forward contract			$ 8,900
Temporary equity, contract terms	20 years
Per share or per unit amount of temporary equity issued (in USD per share)	$ 48.00

Stock-based Compensation (Tables)

12 Months Ended

Dec. 31, 2025

Share-Based Payment Arrangement [Abstract]

Schedule of significant assumptions used to compute the fair values of employee and director stock options awarded

The fair value of stock options granted to employees and directors was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions:


	Year ended December 31,
	2025		2024		2023
Risk‑free interest rate	4.32	%	4.29	%	2.35	%
Expected term (in years)	6.25		6.13		6.11
Expected volatility	71.67	%	74.59	%	76.11	%
Expected dividend yield	—		—		—

Summary of common stock option activity

A summary of the Company's stock option activity for the year ended December 31, 2025 is as follows:


	Number of Options	Weighted- Average Exercise Price		Weighted‑ Average Remaining Contractual Term	Aggregate Intrinsic Value
Outstanding as of December 31, 2024	6,611,391	$	23.29	6.66	$	216,413
Granted	740,919	$	57.70	—	$	—
Exercised	(843,525)	$	16.13	—	$	52,980
Cancelled	(42,607)	$	26.03	—	$	—
Outstanding as of December 31, 2025	6,466,178	$	28.15	6.12	$	510,138
Options exercisable at December 31, 2025	4,940,330	$	23.09	5.48	$	414,757

Summary of restricted stock unit activity

A summary of the Company's restricted stock unit activity for the year ended December 31, 2025 is as follows:


	Number of RSUs	Weighted- Average Grant Date Fair Value
Unvested as of December 31, 2024	1,826,709	$	36.40
Granted	1,771,564	65.24
Vested	(635,186)	33.36
Cancelled	(78,152)	47.84
Unvested as of December 31, 2025	2,884,935	$	54.42

Summary of performance stock unit activity

A summary of the Company's performance stock unit activity for the year ended December 31, 2025 is as follows:


	Number of PSUs	Weighted- Average Grant Date Fair Value
Unvested as of December 31, 2024	249,322	$	13.24
Granted	45,322	56.60
Vested	—	—
Cancelled	—	—
Unvested as of December 31, 2025	294,644	$	41.11

Summary of stock-based compensation expense


	Year Ended
	December 31,
	2025		2024		2023
Research and development	$	19,802	$	10,514	$	8,449
Selling, general, and administrative	47,016		29,168		24,104
Total	$	66,818	$	39,682	$	32,553

Summary of stock-based compensation expense by award type

Stock-based compensation expense by award type recognized during the years ended December 31, 2025, 2024 and 2023 was as follows (in thousands):


	Year Ended
	December 31,
	2025		2024		2023
Stock options	$	22,225	$	21,788	$	17,671
Employees stock purchase plan	685		538		581
Restricted stock units	39,256		16,886		5,998
Performance stock units	4,652		470		8,303
Total	$	66,818	$	39,682	$	32,553

Intangible Assets, net

12 Months Ended

Dec. 31, 2025

Goodwill and Intangible Assets Disclosure [Abstract]

Intangible Assets, net


		As of December 31, 2025						As of December 31, 2024
	Estimated life (years)	Cost		Accumulated Amortization		Net		Cost		Accumulated Amortization		Net
		(In thousands)
Capitalized Milestones	11	$	9,000	$	(3,681)	$	5,319	$	9,000	$	(2,826)	$	6,174

As of December 31, 2025, the Company’s finite-lived intangible assets, which totaled $5.3 million, resulted from the capitalization of certain milestone payments made to Ipsen Pharma, S.A.S., or Ipsen, in accordance with the terms of the Company’s license agreement with Ipsen, in connection with the Company’s first commercial sale of IMCIVREE in the U.S. in March 2021 and in France in March 2022.

As of December 31, 2025, amortization expense for the next five years and beyond is summarized as follows (in thousands):


2026	$	855
2027	855
2028	855
2029	855
2030	855
Thereafter	1,044
Total	$	5,319

The Company began amortizing its finite-lived intangible assets in April 2021 over an 11 year period based on IMCIVREE’s expected patent exclusivity period. Amortization expense totaled $0.9 million, $0.9 million and $0.9 million for the years ended December 31, 2025, 2024 and 2023, respectively. Amortization expense is recorded as a component of cost of sales on the consolidated statements of operations and comprehensive loss.

Stock-based Compensation - 2022 Inducement Plan (Details) - shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Feb. 09, 2022
Stock-based Compensation
Stock options outstanding (in shares)	6,466,178	6,611,391
2022 Employment Inducement Plan
Stock-based Compensation
Authorized shares (in shares)			1,000,000
Vesting period	4 years
Stock options outstanding (in shares)	371,148
Restricted stock unit awards outstanding (in shares)	194,419
Shares available for future grant (in shares)	59,067
2022 Employment Inducement Plan \| Maximum
Stock-based Compensation
Expiration period	10 years

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

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+ Details

Summary of Significant Accounting Policies - Net loss per share (Details) - shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Shares excluded from the computation of diluted net loss per share
Potential common shares (in shares)	12,406,170	11,791,120	8,211,653
Stock options
Shares excluded from the computation of diluted net loss per share
Potential common shares (in shares)	6,466,178	6,611,391	6,551,025
Restricted stock units
Shares excluded from the computation of diluted net loss per share
Potential common shares (in shares)	2,884,935	1,805,412	1,079,382
Performance stock units
Shares excluded from the computation of diluted net loss per share
Potential common shares (in shares)	294,644	249,322	581,246
Common stock reserved for the conversion of Series A convertible preferred stock
Shares excluded from the computation of diluted net loss per share
Potential common shares (in shares)	2,760,413	3,124,995	0

Long-Term Obligations (Details) - USD ($) $ in Thousands					12 Months Ended
Long-Term Obligations (Details) - USD ($) $ in Thousands	Sep. 12, 2023	Sep. 29, 2022	Jun. 29, 2022	Jun. 16, 2022	Dec. 31, 2025	Dec. 31, 2024	Jun. 30, 2022
Long-Term Obligations
Derivative asset					$ 1,180	$ 270
Deferred royalty obligation, current					7,296	1,541
Deferred royalty obligation					100,886	108,269
Revenue Interest Financing Agreement
Long-Term Obligations
Proceeds from royalty financing agreement	$ 24,400	$ 37,500	$ 37,500		100,000
Repayment of debt					40,300
Revenue interest payment period				12 years
Fair value of embedded derivative liability							$ 1,600
Remeasurement of the embedded derivative liability					900	$ 1,400
Deferred royalty obligation					$ 108,200
Effective interest rate					17.05%
Royalty Interest Financing Agreement
Long-Term Obligations
Debt issuance costs					$ 3,300
Royalty Interest Financing Agreement \| Annual net revenues up to 125 million
Long-Term Obligations
Royalty interest (as a percent)				11.50%
Threshold annual net revenues				$ 125,000
Royalty Interest Financing Agreement \| Annual net revenues between 125 million and 300 million
Long-Term Obligations
Royalty interest (as a percent)				7.50%
Royalty Interest Financing Agreement \| Annual net revenues exceeding 300 million
Long-Term Obligations
Royalty interest (as a percent)				2.50%
Threshold annual net revenues				$ 300,000
Royalty Interest Financing Agreement \| March 31, 2027
Long-Term Obligations
Threshold cumulative minimum payments (as a percent)				60.00%
Royalty Interest Financing Agreement \| March 31, 2029
Long-Term Obligations
Threshold cumulative minimum payments (as a percent)				120.00%
Maximum \| Revenue Interest Financing Agreement
Long-Term Obligations
Aggregate investment amount				$ 100,000
Maximum \| Royalty Interest Financing Agreement
Long-Term Obligations
Revenue interest cap (as a percent)				250.00%
Maximum \| Royalty Interest Financing Agreement \| Annual net revenues between 125 million and 300 million
Long-Term Obligations
Threshold annual net revenues				$ 300,000
Minimum \| Royalty Interest Financing Agreement
Long-Term Obligations
Revenue interest cap (as a percent)				185.00%
Minimum \| Royalty Interest Financing Agreement \| Annual net revenues between 125 million and 300 million
Long-Term Obligations
Threshold annual net revenues				$ 125,000

CONSOLIDATED BALANCE SHEETS (Parenthetical) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Common stock, par value per share (in USD per share)	$ 0.001	$ 0.001
Common stock, authorized (in shares)	120,000,000	120,000,000
Common stock, issued (in shares)	67,205,321	62,390,654
Common stock, outstanding (in shares)	67,205,321	62,390,654
Convertible preferred stock
Convertible preferred stock, par value (in USD per share)	$ 0.001	$ 0.001
Convertible preferred stock, authorized (in shares)	150,000	150,000
Convertible preferred stock, issued (in shares)	132,500	150,000
Convertible preferred stock, outstanding (in shares)	132,500	150,000
Liquidation preference	$ 132,500	$ 150,000

Subsequent Events (Details) - USD ($) $ in Thousands		12 Months Ended
Subsequent Events (Details) - USD ($) $ in Thousands	Jan. 26, 2026	Dec. 31, 2025	Apr. 15, 2024
Subsequent events
Conversion of Series A preferred stock redeemable to common stock		$ 17,242
Common stock reserved for the conversion of Series A convertible preferred stock
Subsequent events
Shares issued upon conversion (in shares)			20.8333
Subsequent events
Subsequent events
Shares issued upon conversion (in shares)	20.8333
Debt conversion, converted instrument, shares issued (in shares)	364,582
Subsequent events \| Common stock reserved for the conversion of Series A convertible preferred stock
Subsequent events
Conversion of stock, converted (in shares)	17,500
Conversion of Series A preferred stock redeemable to common stock	$ 17,400

Stock-based Compensation - 2017 Employee Stock Purchase Plan (Details) - 2017 Employee Stock Purchase Plan - shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Stock-based Compensation
Common stock reserved for issuance (in shares)	1,240,958
Percentage increase in authorized shares under the plan	1.00%
Number of shares issued (in shares)	37,514	44,554	49,819
Purchase price of ESPP meeting certain criteria as of the offering period (as a percent)	85.00%
Discount recognized as compensation expense	15.00%
Purchase period	6 months

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Segment and Geographic Information - Narrative (Details)	12 Months Ended
	Dec. 31, 2025 segment
Segment Reporting [Abstract]
Number of operating segments	2
Number of reporting segments	1

Summary of Significant Accounting Policies - Income Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024
Accounting Policies [Abstract]
Accrued interest or penalties	$ 0	$ 0

Summary of Significant Accounting Policies - Prepaid expenses and other current assets (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Accounting Policies [Abstract]
Prepaid research and development costs	$ 16,166	$ 7,580
Other current assets	9,967	8,802
Prepaid expenses and other current assets	$ 26,133	$ 16,382

Fair Value of Financial Assets and Liabilities - Financial Assets and Liabilities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Fair value of financial assets and liabilities
Marketable securities:	$ 334,648	$ 231,428
Derivative asset	1,180	270
Total	369,777	306,016
US treasury securities
Fair value of financial assets and liabilities
Marketable securities:	71,877	65,118
Corporate debt securities and commercial paper
Fair value of financial assets and liabilities
Marketable securities:	262,771	166,310
Commercial Paper
Fair value of financial assets and liabilities
Cash equivalents:		2,984
Money market funds
Fair value of financial assets and liabilities
Cash equivalents:	33,949	71,334
Level 1
Fair value of financial assets and liabilities
Derivative asset	0	0
Total	105,826	136,452
Level 1 \| US treasury securities
Fair value of financial assets and liabilities
Marketable securities:	71,877	65,118
Level 1 \| Corporate debt securities and commercial paper
Fair value of financial assets and liabilities
Marketable securities:	0	0
Level 1 \| Commercial Paper
Fair value of financial assets and liabilities
Cash equivalents:		0
Level 1 \| Money market funds
Fair value of financial assets and liabilities
Cash equivalents:	33,949	71,334
Level 2
Fair value of financial assets and liabilities
Derivative asset	0	0
Total	262,771	169,294
Level 2 \| US treasury securities
Fair value of financial assets and liabilities
Marketable securities:	0	0
Level 2 \| Corporate debt securities and commercial paper
Fair value of financial assets and liabilities
Marketable securities:	262,771	166,310
Level 2 \| Commercial Paper
Fair value of financial assets and liabilities
Cash equivalents:		2,984
Level 2 \| Money market funds
Fair value of financial assets and liabilities
Cash equivalents:	0	0
Level 3
Fair value of financial assets and liabilities
Derivative asset	1,180	270
Total	1,180	270
Level 3 \| US treasury securities
Fair value of financial assets and liabilities
Marketable securities:	0	0
Level 3 \| Corporate debt securities and commercial paper
Fair value of financial assets and liabilities
Marketable securities:	0	0
Level 3 \| Commercial Paper
Fair value of financial assets and liabilities
Cash equivalents:		0
Level 3 \| Money market funds
Fair value of financial assets and liabilities
Cash equivalents:	$ 0	$ 0

Summary of Significant Accounting Policies - Segment Information (Details)	12 Months Ended
	Dec. 31, 2025 segment
Accounting Policies [Abstract]
Number of operating segments	2
Number of reporting segments	1

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2025

Accounting Policies [Abstract]

Basis of Presentation

Use of Estimates

Principles of Consolidation

Segment Information

Off-Balance Sheet Risk and Concentrations of Credit Risk

Cash and Cash Equivalents

Short-Term Investments

Restricted Cash

Accounts Receivable, net

Revenue Recognition

Product Revenue, Net

Reserves for Variable Consideration

The following are the components of variable consideration related to product revenue:

The table below summarizes balances and activity in each of the product revenue allowance and reserve categories as follows (in thousands):


	Provision for Cash Discounts		Fees, Rebates and Other Incentives		Total



Beginning Balance at December 31, 2023	$	199	$	9,475	$	9,674
Provision related to sales in the current year	2,317		27,315		29,632
Credit and payments made	(2,263)		(20,940)		(23,203)
Ending balance December 31, 2024	$	253	$	15,850	$	16,103
Provision related to sales in the current year	3,670		45,811		49,481
Credit and payments made	(3,620)		(30,097)		(33,717)
Ending balance December 31, 2025	$	303	$	31,564	$	31,867

Provision for cash discounts are recorded as reductions of accounts receivable, and fees, rebates, and other incentives are recorded as components of accrued expenses.

License Agreements

Deferred Royalty Obligation

Inventory

Inventory consists of the following (in thousands):


	December 31, 2025		December 31, 2024
Raw Materials	$	6,745	$	6,776
WIP	1,631		1,250
Finished Goods	17,377		10,715
Total Inventory	$	25,753	$	18,741

Cost of Product Sales

Intangible Assets, Net

Intangible Assets, net

Impairment of Long-Lived Assets

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consist primarily of costs incurred in advance of services being received, including services related to clinical trial programs.

Other Long-Term Assets

Property and Equipment

Property and equipment consists of the following (in thousands):


	Useful Life	December 31,
		2025		2024
Leasehold improvements	*	$	2,733	$	2,705
Office equipment	5 years	154		154
Computers and software	3 years	1,291		1,291
Furniture, fixtures and equipment	5 years	1,249		1,249
Construction in progress		926		—
Property and equipment, gross		6,353		5,399
Less accumulated depreciation and amortization		(5,249)		(4,767)
Property and equipment, net		$	1,104	$	632

*Shorter of asset life or lease term.

Depreciation and amortization expense related to property and equipment for the years ended December 31, 2025, 2024 and 2023 was $0.5 million, $0.7 million, and $0.9 million respectively.

Acquired IPR&D and Milestone Expense

Foreign Currency Translation

Fair Value Measurements

Level 1 — Quoted market prices in active markets for identical assets or liabilities.

Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.

Research and Development Expenses

Income Taxes

The Company recognizes interest and penalties related to unrecognized tax benefits as provision for income taxes in the accompanying consolidated statements of operations.

Net Loss Per Share

Comprehensive Loss

Patent Costs

Costs to secure and defend patents are expensed as incurred and are classified as general and administrative expenses.

Subsequent Events

Application of New or Revised Accounting Standards, Recently adopted accounting pronouncements, and Recently issued accounting pronouncements, not yet adopted

Application of New or Revised Accounting Standards

Recently adopted accounting pronouncements

Recently issued accounting pronouncements, not yet adopted

Income Taxes - Reconciliation of income tax rate (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Amount
U.S. Federal Statutory Tax Rate	$ (41,169)
State and Local Income Tax, Net of Federal (National) Income Tax Effect	0
Foreign Tax Effects:
Effective Income Tax Rate Reconciliation, Foreign Income Tax Rate Differential, Amount	529
Tax Credits:
Research & Development Credits	(13,073)
Changes in Valuation Allowances	51,008
Nontaxable or Nondeductible Items:
Executive Compensation Limit	8,661
Stock-Based Compensation	(2,283)
LG Chem	(3,930)
Deferred royalty financing	913
Other	207
Other Adjustments	(366)
Income Tax Expense (Benefit)	$ 497	$ 346	$ 564
Percentage
U.S. Federal Statutory Tax Rate	21.00%	21.00%	21.00%
State and Local Income Tax, Net of Federal (National) Income Tax Effect	0.00%	5.61%	10.24%
Foreign Tax Effects:
Other Foreign Jurisdictions	(0.27%)
Tax Credits:
Research and development credits	6.67%	3.75%	4.02%
Changes in Valuation Allowances	(26.02%)
Nontaxable or Nondeductible Items:
Executive Compensation	4.42%	(2.26%)	(0.32%)
Stock-Based Compensation	1.16%	(0.57%)	(1.37%)
LG Chem	2.00%
Deferred royalty financing	(0.46%)
Other	(0.10%)
Other	0.19%	(1.61%)	(0.16%)
Rate changes		(2.04%)	(6.52%)
License Agreement		(1.51%)	0.00%
Change in valuation allowance		(22.50%)	(27.10%)
Effective tax rate	(0.25%)	(0.13%)	(0.21%)

Segment and Geographic Information - Profit (loss) (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Segment and Geographic Information
Total revenues	$ 189,757	$ 130,126	$ 77,428
Cost of sales	19,492	13,368	9,302
Net loss	(196,539)	(260,602)	(184,678)
Product revenue, net
Segment and Geographic Information
Total revenues	194,771	130,126	77,428
License revenue
Segment and Geographic Information
Total revenues	(5,014)	0	0
US \| Product revenue, net
Segment and Geographic Information
Total revenues	133,551	96,108	62,425
International \| Product revenue, net
Segment and Geographic Information
Total revenues	61,220	34,018	15,003
Single reportable segment
Segment and Geographic Information
Total revenues	189,757	130,126	77,428
Cost of sales	19,492	13,368	9,302
Global headcount expense	172,648	119,084	96,413
Preclinical, clinical and development expense	87,322	82,180	74,465
Commercial & medical affairs	66,963	55,767	47,905
Corporate, general & administrative	35,348	32,135	27,154
Other segment expenses	0	93,094	6,571
Other (income) expense, net	(1,264)	(11,139)	317
Interest (income) expense, net	5,290	5,893	(585)
Income taxes	497	346	564
Single reportable segment \| Product revenue, net
Segment and Geographic Information
Total revenues	194,771	130,126	77,428
Single reportable segment \| License revenue
Segment and Geographic Information
Total revenues	(5,014)	0	0
Single reportable segment \| US \| Product revenue, net
Segment and Geographic Information
Total revenues	133,551	96,108	62,425
Single reportable segment \| International \| Product revenue, net
Segment and Geographic Information
Total revenues	$ 61,220	$ 34,018	$ 15,002

Stock-based Compensation - Stock Options (Details) - $ / shares	12 Months Ended
	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023
Stock-based Compensation
Number of common stock options granted (in shares)	740,919
Employees stock purchase plan
Stock-based Compensation
Grant cliff vesting period	1 year
Employees stock purchase plan \| Minimum
Stock-based Compensation
Vesting period of stock option awards	1 year
Employees stock purchase plan \| Maximum
Stock-based Compensation
Vesting period of stock option awards	4 years
2017 Equity Incentive Plan
Stock-based Compensation
Number of common stock options granted (in shares)	740,919	926,543	842,528
Weighted average grant date fair value relating to outstanding stock options (in USD per share)	$ 57.70	$ 32.69	$ 17.72
2022 Employment Inducement Plan
Stock-based Compensation
Vesting period of stock option awards	4 years
Number of common stock options granted (in shares)	0	57,100	229,360
Weighted average grant date fair value relating to outstanding stock options (in USD per share)		$ 29.16	$ 15.30

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Dec. 31, 2025 shares
Trading Arrangements, by Individual
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Pamela Cramer [Member]
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	On December 15, 2025, Pamela Cramer, the Company's Chief Human Resources Officer, adopted a trading plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act for the sale of up to 52,617 shares of the Company's common stock until March 15, 2027.
Name	Pamela Cramer
Title	Company's Chief Human Resources Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	December 15, 2025
Expiration Date	March 15, 2027
Arrangement Duration	455 days
Aggregate Available	52,617
Jennifer Lee [Member]
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	On December 15, 2025, Jennifer Lee, the Company's EVP, Head of North America, adopted a trading plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act for the sale of up to 62,459 shares of the Company's common stock until October 1, 2026.
Name	Jennifer Lee
Title	Company's EVP, Head of North America
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	December 15, 2025
Expiration Date	October 1, 2026
Arrangement Duration	290 days
Aggregate Available	62,459
Joseph Shulman [Member]
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	On December 15, 2025, Joseph Shulman, the Company's Chief Technical Officer, adopted a trading plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act for the sale of up to 66,910 shares of the Company's common stock until March 12, 2027.
Name	Joseph Shulman
Title	Company's Chief Technical Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	December 15, 2025
Expiration Date	March 12, 2027
Arrangement Duration	452 days
Aggregate Available	66,910
Lynn Tetrault [Member]
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	On December 15, 2025, Lynn Tetrault, a member of the Company's Board of Directors, adopted a trading plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act for the sale of up to 55,000 shares of the Company's common stock until September 16, 2026.
Name	Lynn Tetrault
Title	member of the Company's Board of Directors
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	December 15, 2025
Expiration Date	September 16, 2026
Arrangement Duration	275 days
Aggregate Available	55,000

CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK & STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Total	At-the-market offering	IPO	Common stock reserved for the conversion of Series A convertible preferred stock	Common Stock	Common Stock At-the-market offering	Common Stock IPO	Additional Paid-In Capital	Additional Paid-In Capital At-the-market offering	Additional Paid-In Capital IPO	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit
Temporary Equity
Accretion of preferred stock dividends	$ 0
Ending balance (in shares) at Dec. 31, 2023				0
Ending balance at Dec. 31, 2023				$ 0
Beginning balance (in shares) at Dec. 31, 2022					56,612,429
Beginning balance at Dec. 31, 2022	264,262				$ 56			$ 974,356			$ (92)	$ (710,058)
Increase (Decrease) in Stockholders' Equity
Stock compensation expense	32,553							32,553
Issuance of common stock in connection with ESPP (in shares)					49,819
Issuance of common stock in connection with ESPP	1,053							1,053
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units (in shares)					745,066
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units	7,468				$ 1			7,467
Issuance of common stock, net of offering costs (in shares)					2,019,245
Issuance of common stock, net of offering costs	48,875				$ 2			48,873
Foreign currency translation adjustment	(140)										(140)
Net unrealized gain on marketable securities	366										366
Net loss	(184,678)											(184,678)
Ending balance at Dec. 31, 2023	169,759				$ 59			1,064,302			134	(894,736)
Ending balance (in shares) at Dec. 31, 2023					59,426,559
Temporary Equity
Issuance of Series A Preferred Stock, net of issuance costs (in shares)				150,000
Issuance of Series A Preferred Stock, net of $2,250 of issuance costs				$ 138,850
Accretion of preferred stock dividends	3,970			$ 3,970
Ending balance (in shares) at Dec. 31, 2024				150,000
Ending balance at Dec. 31, 2024	142,820			$ 142,820
Increase (Decrease) in Stockholders' Equity
Stock compensation expense	39,682							39,682
Issuance of common stock in connection with ESPP (in shares)					44,554
Issuance of common stock in connection with ESPP	1,270							1,270
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units (in shares)					1,742,803
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units	15,974				$ 2			15,972
Issuance of common stock as consideration for LGC license (in shares)					432,143
Issuance of common stock as consideration for LGC license	18,716							18,716
Issuance of common stock, net of offering costs (in shares)						744,595
Issuance of common stock, net of offering costs		$ 41,073							$ 41,073
Accretion of preferred stock dividends	(3,970)							(3,970)
Foreign currency translation adjustment	2										2
Net unrealized loss on marketable securities	(175)										(175)
Net loss	(260,602)											(260,602)
Ending balance at Dec. 31, 2024	21,729				$ 61			1,177,045			(39)	(1,155,338)
Ending balance (in shares) at Dec. 31, 2024					62,390,654
Temporary Equity
Accretion of preferred stock dividends	5,378			$ 5,378
Conversion of Series A preferred stock redeemable to common stock (in shares)				(17,500)
Conversion of Series A preferred stock redeemable to common stock				$ (17,241)
Ending balance (in shares) at Dec. 31, 2025				132,500
Ending balance at Dec. 31, 2025	130,957			$ 130,957
Increase (Decrease) in Stockholders' Equity
Stock compensation expense	66,818							66,818
Issuance of common stock in connection with ESPP (in shares)					37,514
Issuance of common stock in connection with ESPP	1,584				$ 1			1,583
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units (in shares)					1,457,414
Issuance of common stock in connection with exercise of stock options and vesting of restricted stock units	13,604				$ 1			13,603
Issuance of common stock, net of offering costs (in shares)						587,510	2,367,647
Issuance of common stock, net of offering costs		$ 32,109	$ 188,657			$ 1	$ 2		$ 32,108	$ 188,655
Accretion of preferred stock dividends	(5,378)							(5,378)
Conversion of Series A preferred stock redeemable to common stock (in shares)					364,582
Conversion of Series A preferred stock redeemable to common stock	17,242				$ 1			17,241
Foreign currency translation adjustment	(1,261)										(1,261)
Net unrealized loss on marketable securities	504										504
Net loss	(196,539)											(196,539)
Ending balance at Dec. 31, 2025	$ 139,069				$ 67			$ 1,491,675			$ (796)	$ (1,351,877)
Ending balance (in shares) at Dec. 31, 2025					67,205,321

Intangible Assets, net - Schedule of finite-lived intangible assets (Details) - USD ($) $ in Thousands	Dec. 31, 2025	Dec. 31, 2024
Finite-Lived Intangible Assets [Line Items]
Total	$ 5,319	$ 6,174
Capitalized Milestones
Finite-Lived Intangible Assets [Line Items]
Estimated life (years)	11 years
Cost	$ 9,000	9,000
Accumulated Amortization	(3,681)	(2,826)
Total	$ 5,319	$ 6,174

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Intangible Assets, net (Tables)

12 Months Ended

Dec. 31, 2025

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of finite-lived intangible assets


		As of December 31, 2025						As of December 31, 2024
	Estimated life (years)	Cost		Accumulated Amortization		Net		Cost		Accumulated Amortization		Net
		(In thousands)
Capitalized Milestones	11	$	9,000	$	(3,681)	$	5,319	$	9,000	$	(2,826)	$	6,174

Finite-Lived Intangible Assets Amortization Expense

As of December 31, 2025, amortization expense for the next five years and beyond is summarized as follows (in thousands):


2026	$	855
2027	855
2028	855
2029	855
2030	855
Thereafter	1,044
Total	$	5,319

Common Stock (Details) - USD ($)									1 Months Ended	3 Months Ended	12 Months Ended
Common Stock (Details) - USD ($)	Dec. 04, 2025	Jul. 11, 2025	Jul. 09, 2025	Feb. 29, 2024	Aug. 21, 2023	Oct. 18, 2022	Sep. 19, 2022	Nov. 02, 2021	Jan. 21, 2025	Dec. 31, 2024	Dec. 31, 2025	Dec. 31, 2024	Dec. 31, 2023	Apr. 01, 2024
Stock
Share price (in USD per share)						$ 26.00	$ 26.00
Net proceeds from issuance of common stock						$ 14,200,000	$ 116,887				$ 34,035,000	$ 39,146,000	$ 48,875,000
Issuance of common stock, net of offering costs (in shares)						580,000	4,800,000
Issuance of common stock, net of offering costs													$ 48,875,000
Period for option to purchase additional shares by underwriter							30 days
Follow On Offering
Stock
Number of shares to be issued (in shares)		2,367,647	2,058,824
Share price (in USD per share)			$ 85.00
Number of days to exercise underwriting option			30 days
Net proceeds from issuance of common stock		$ 188,700,000
Payments for underwriting expense		$ 12,600,000
Underwriter option to purchase
Stock
Number of shares to be issued (in shares)			308,823
Issuance of common stock, net of offering costs (in shares)							720,000
At-the-market offering
Stock
Issuance of common stock, net of offering costs											$ 32,109,000	$ 41,073,000
At-the-market offering \| Maximum
Stock
Consideration received on transaction								$ 100,000,000.0
Convertible preferred stock
Stock
Conversion of Series A preferred stock redeemable to common stock (in shares)	17,500										17,500
Conversion of Series A preferred stock redeemable to common stock (in shares)	364,582
Share price (in USD per share)														$ 1,000
Common stock \| At-the-market offering
Stock
Net proceeds from issuance of common stock					$ 48,900,000
Consideration received on transaction				$ 200,000,000.0
Issuance of common stock, net of offering costs (in shares)					2,000,000				587,510	744,595
Issuance of common stock, net of offering costs									$ 32,100,000	$ 41,200,000

Cover - USD ($) $ in Billions	12 Months Ended
Cover - USD ($) $ in Billions	Dec. 31, 2025	Feb. 24, 2026	Jun. 30, 2025
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2025
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-38223
Entity Registrant Name	RHYTHM PHARMACEUTICALS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	46-2159271
Entity Address, Address Line One	222 Berkeley Street
Entity Address, Address Line Two	12th Floor
Entity Address, City or Town	Boston
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02116
City Area Code	857
Local Phone Number	264-4280
Title of 12(b) Security	Common Stock, $0.001 par value per share
Trading Symbol	RYTM
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Public Float			$ 3.8
Entity Common Stock, Shares Outstanding		68,285,039
Documents Incorporated by Reference	The registrant intends to file a definitive proxy statement for the registrant's 2026 Annual Meeting of Stockholders within 120 days of the end of the fiscal year ended December 31, 2025. Portions of such definitive proxy statement are incorporated by reference into Part III of this Annual Report on Form 10-K.
Entity Central Index Key	0001649904
Document Fiscal Period Focus	FY
Amendment Flag	false
Document Fiscal Year Focus	2025

Nature of Business	12 Months Ended
Nature of Business	Dec. 31, 2025
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of Business	Nature of Business Rhythm Pharmaceuticals, Inc. (the “Company” or “we”) is a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients and their families living with rare neuroendocrine diseases. We are focused on advancing our melanocortin-4 receptor agonists, including our lead asset, IMCIVREE (setmelanotide), as a precision medicine designed to treat hyperphagia and severe obesity caused by MC4R pathway diseases. While obesity affects hundreds of millions of people worldwide, we are developing therapies for a subset of individuals who have hyperphagia, a pathological hunger, and severe obesity due to an impaired MC4R pathway, which may be caused by traumatic injury or genetic variants. The MC4R pathway is an endocrine pathway in the brain that is responsible for regulating hunger, caloric intake and energy expenditure, which consequently affect body weight. IMCIVREE, an MC4R agonist for which we hold worldwide rights, is the first-ever therapy developed for patients with certain rare diseases that is approved or authorized in the United States, European Union (EU), Great Britain, Canada and other countries and regions. The Company is a Delaware corporation organized in February 2013 under the name Rhythm Metabolic, Inc., and as of October 2015, under the name Rhythm Pharmaceuticals, Inc. The Company has wholly owned subsidiaries in the US, the United Kingdom, the Netherlands, France, Germany, Italy, Spain, Switzerland, Japan and Canada. The Company is subject to risks and uncertainties common to commercial-stage companies in the biotechnology industry, including but not limited to, risks associated with the commercialization of approved products, completing preclinical studies and clinical trials, receiving regulatory approvals for product candidates, development by competitors of new biopharmaceutical products, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Commercialization of approved products will require significant resources and in order to market IMCIVREE, the Company must continue to build its sales, marketing, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even though the Company has an approved product, and even if the Company’s further product development efforts are successful, it is uncertain when, if ever, the Company will realize sufficient revenue from product sales to fund operations. Liquidity The Company has incurred operating losses and negative cash flows from operations since inception, and expects operating losses to continue for the foreseeable future. As of December 31, 2025, the Company had an accumulated deficit of $1.4 billion. The Company has primarily funded these losses through the proceeds from the sales of our common stock and preferred stock, asset sales, royalty financing, out-license arrangements, as well as capital contributions received from the former parent company, Rhythm Holdings LLC. The Company has devoted substantially all of its resources to its drug development efforts, comprising of research and development, the acquisition of in process research and development assets, manufacturing, conducting clinical trials for its product candidates, protecting its intellectual property, commercialization activities and general and administrative functions relating to these operations. The future success of the Company is dependent on its ability to continue to develop its product candidates and ultimately upon its ability to attain profitable operations. At December 31, 2025, the Company had $388.9 million of cash and cash equivalents and short-term investments on hand. In the future, the Company will be dependent on obtaining funding from third parties, such as proceeds from the issuance of debt, sale of equity, proceeds from out-license arrangements, revenue and revenue growth, and funded research and development programs to maintain the Company's operations and meet the Company's obligations. There is no guarantee that additional equity or other financings will be available to the Company on acceptable terms, or at all. If the Company fails to obtain additional funding when needed, the Company would be forced to scale back, terminate its operations or seek to merge with or be acquired by another company. Management believes that the Company's existing cash resources will be sufficient to fund the Company's operations through at least the next twelve months from the filing of this Annual Report on Form 10-K with the SEC.

Segment and Geographic Information

12 Months Ended

Dec. 31, 2025

Segment Reporting [Abstract]

Segment and Geographic Information

Operating segments are defined as components of an entity about which separate discrete information is available for evaluation by the chief operating decision maker, ("CODM"), or decision making group, in deciding how to allocate resources and in assessing performance. The company has identified that there is a single management team that reports to the Chief Executive Officer, who is the CODM and comprehensively manages the entire business. The CODM reviews financial information on a consolidated basis, specifically but not limited to, revenue and operating expenses by key functional area to evaluate performance and allocate resources. Based on the manner in which the CODM reviews operating results, the Company has identified two operating segments: United States ("U.S.") and international. These two operating segments have been aggregated into a single reportable segment in accordance with the aggregation criteria in ASC 280, Segment Reporting, as they have similar economic characteristics and are similar in several qualitative areas including: the nature of products and services provided, the type of customers served, the methods of distribution, and the regulatory environment in which they operate.

The table below is a summary of the segment profit or loss, including significant segment expenses (in thousands):

Segment Analysis


	2025		2024		2023

Net product revenue - U.S.	$	133,551	$	96,108	$	62,425
Net product revenue - International	61,220		34,018		15,002
Total net product revenue	194,771		130,126		77,428
License revenue	(5,014)		-		—
Total net revenue	189,757		130,126		77,428

Cost of sales	19,492		13,368		9,302
Global headcount expense	172,648		119,084		96,413
Preclinical, clinical and development expense	87,322		82,180		74,465
Commercial & medical affairs	66,963		55,767		47,905
Corporate, general & administrative	35,348		32,135		27,154
Other segment expenses	—		93,094		6,571
Other (income) expense, net	(1,264)		(11,139)		317
Interest (income) expense, net	5,290		5,893		(585)
Income taxes	497		346		564
Net loss	$	(196,539)	$	(260,602)	$	(184,678)

Geographic Data

The Company allocates, for the purpose of geographic data reporting, its revenue based upon the location of its customers. Total revenue by geographic area was as follows (in thousands):


	Year ended December 31,
	2025		2024		2023
US	$	133,551	$	96,108	$	62,425
International	61,220		34,018		15,003
Total net product revenue	$	194,771	$	130,126	$	77,428

As of December 31, 2025 and 2024, long-lived assets at locations outside the United States were not material.

- Definition

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

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+ Details