Rhythm Pharmaceuticals Reports Second Quarter 2026 Financial Results and Business Update
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-- Second quarter 2026 net product revenue from global sales of IMCIVREE of
-- Weekly injectable MC4R agonist
-- Phase 3 TRANSCEND Trial Results in Acquired HO published in the
-- Management to host conference call today at 8:00 a.m. ET --
“Rhythm continues to demonstrate strong commercial and clinical development progress, highlighted by a strong start in the
Recent Business and Development Highlights
- Today, the Company announced that more than 400 patient start forms had been received for IMCIVREE for acquired HO from approximately 300 prescribers as of
June 30, 2026 , since the approval by theU.S. Food and Drug Administration (FDA ) onMarch 19, 2026 ; - Revenue from global sales of IMCIVREE was
$71.3 million for the second quarter of 2026, an increase of 19% on a sequential basis from the first quarter of 2026. The number of patients on reimbursed therapy globally increased by greater than 20% as compared to the first quarter of 2026.U.S .: Revenue of$51.0 million , or 72% of product revenue, was generated inthe United States in the second quarter of 2026, an increase of$14.1 million or 38% compared to the first quarter of 2026. The sequential quarter over quarter growth inU.S . revenue was driven primarily by increased patient demand for IMCIVREE in acquired HO, as well as continued growth in demand in Bardet-Biedl syndrome (BBS).- Ex-
U.S .: Revenue of$20.3 million , or 28% of product revenue, was generated outsidethe United States , a sequential decrease of$(2.9) million or (13)% compared to the first quarter of 2026. The Company incurred a$3.8 million retrospective charge associated with the Contribution M mechanism inFrance , of which$3.1 million was related to revenue booked in periods prior to the second quarter of 2026. The number of reimbursed patients on IMCIVREE continued to increase in the second quarter of 2026.
- Today, the Company announced preliminary data from Part C of the Phase 1/2 study of
RM-718 in acquired HO patients. - Today, the Company announced that six abstracts, including three oral presentations and three poster presentations have been accepted for presentation at the European Society for Pediatric Endocrinology’s Annual Meeting (ESPE 2026)
September 8-10, 2026 . The presentations include:- Efficacy and safety of setmelanotide in pediatric patients with acquired hypothalamic obesity: final phase 3 trial results;
- Impact of setmelanotide on metabolic index scores in pediatric participants with acquired hypothalamic obesity - A Phase 3 trial post-hoc analysis;
- Pediatric patients with acquired hypothalamic obesity treated with setmelanotide: real-world BMI and hunger data for up to 12 months in
France ; - Long-term weight outcomes of setmelanotide in pediatric participants with Bardet-Biedl syndrome or with POMC or LEPR deficiency and obesity;
- The association of clinical features of Bardet-Biedl syndrome in a large population of patients with obesity and a positive genetic test for biallelic BBS variants;
- Clinical features associated with pathogenic or likely pathogenic biallelic Bardet-Biedl syndrome variants in a large population of patients with obesity;
- On
July 8, 2026 , results from Rhythm’s pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in patients with acquired hypothalamic obesity were published inThe New England Journal of Medicine by Dr.Jennifer Miller , et al.; The publication was accompanied by a 'Science Behind the Study' editorial authored by ProfessorSadaf Farooqi , M.B., Ch.B., Ph.D., titled Treating Acquired Hypothalamic Obesity; - On
June 13, 2026 , atEndocrine Society's Annual Meeting (ENDO 2026), the Company presented positive six-month data from its Phase 2 trial of setmelanotide that demonstrated patients with Prader-Willi Syndrome (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures; - Also during ENDO 2026, held June 13–16, 2026, the Company presented new data in multiple presentations, highlights included durable BMI reduction for patients with acquired HO after 2.5 years of setmelanotide; durable weight reduction after 1 year of bivamelagon treatment in patients in acquired HO; post-hoc analysis from the TRANSCEND trial in patients with acquired HO with a history of bariatric surgery demonstrated BMI reductions and weight category improvement after 1 year of setmelanotide treatment; and positive real-world efficacy analyses of
U.S . patients with BBS; - On
May 28, 2026 , the Company announced the publication of a new evidence-based, consensus-driven diagnostic algorithm for (BBS) in the peer-reviewedAmerican Journal of Medical Genetics ; - On
May 12-15, 2026 , at theEuropean Congress on Obesity (ECO 2026), the Company presented data on weight outcomes at five years of setmelanotide treatment in patients with BBS and obesity, weight outcomes at six years in patients with POMC or LEPR deficiency obesity, and a 52-week indirect comparison of patients aged 2-5 years with BBS treated with setmelanotide versus patients with BBS from a natural history cohort in the International Clinical Registry Investigating Bardet-Biedl Syndrome. - On
May 12, 2026 , the Company announced six data presentations atThe European Congress of Endocrinology (ECE); highlights included real-world efficacy results in patients with acquired HO enrolled in France’s early-access program; study results on hyperphagia severity in patients with BBS; post-hoc TRANSCEND trial results of patients with acquired HO treated with setmelanotide that demonstrated significant improvements in multiple metabolic index scores; a study that highlighted the persistent and under-recognized health burden of acquired HO across life stages in patients with childhood-onset craniopharyngioma fromthe Netherlands ,Germany , and theUK .
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
- Complete enrollment in the setmelanotide substudy in congenital HO in the second half of 2026;
- Complete enrollment in the Phase 1/2, Part D trial evaluating
RM-718 in PWS in the second half of 2026; - Anticipate launch of IMCIVREE for acquired HO in
Japan pending a decision byJapan's Ministry of Health, Labour and Welfare (MHLW) by year-end 2026; - Initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired HO by year-end 2026;
- Anticipate country-level launches of IMCIVREE for acquired HO in
Europe beginning in 2027.
Second Quarter 2026 Financial Results
Cash Position: As of June 30, 2026, cash, cash equivalents and short-term investments were approximately
Revenue: Net product revenues from global sales of IMCIVREE were $71.3 million for the second quarter of 2026, as compared to $48.5 million for the second quarter of 2025.
R&D Expenses: R&D expenses were
SG&A Expenses: SG&A expenses were
Other income (expense), net: Other expense, net was $(0.3) million for the second quarter of 2026, as compared to other expense, net of
Net Loss attributable to common stockholders: Net loss attributable to common stockholders was $(50.4) million for the second quarter of 2026, or a net loss per basic and diluted share of $(0.73), as compared to a net loss attributable to common stockholders of $(48.0) million for the second quarter of 2025, or a net loss per basic and diluted share of $(0.75).
Year to Date 2026 Financial Results
Revenue: Net product revenues relating to sales of IMCIVREE were
R&D Expenses: R&D expenses were
SG&A Expenses: SG&A expenses were
Other income (expense), net: Other income (expense), net was
Net Loss attributable to common stockholders: Net loss attributable to common stockholders was
Financial Guidance: For the year ending
- GAAP total operating expenses, inclusive of:
- R&D expenses of approximately $175 million to $195 million;
- SG&A expenses of approximately $188 million to $202 million; and
- Excluding stock-based compensation.
Non-GAAP Operating Expenses is defined as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing (see below under "Non-GAAP Financial Measures" for more details).
Based on its current operating plans, Rhythm expects that its cash, cash equivalents and short-term investments as of June 30, 2026 will be sufficient to fund the Company’s planned operations for at least 24 months.
Conference Call Information
A webcast of the call will also be available under "Events and Presentations" in the Investor Relations section of the
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. The European Commission (EC) has authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.
Setmelanotide Indication
In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired HO, and in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).
In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Limitations of Use
Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:
- Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign
- Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity
Contraindication
Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported.
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.
Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur.
Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE.
Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs.
ADVERSE REACTIONS
Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.
USE IN SPECIFIC POPULATIONS
Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe.
Please see the full Prescribing Information for additional Important Safety Information.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, including, setmelanotide, bivamelagon, and RM-718; the use of setmelanotide in patients with acquired HO and the success of our commercial launch; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates, including potential marketing approval and launch in Japan and launches in the European Union and the timing thereof; the commercial growth of IMCIVREE; the estimated market size and addressable population for our drug products, including setmelanotide for the treatment of acquired HO; the future announcement of data from our ongoing clinical trials, including the substudy evaluating setmelanotide for patients with congenital hypothalamic obesity, Part C and Part D of the Phase 1 trial evaluating RM-718, and the open-label Phase 2 trial evaluating setmelanotide in patients with PWS, and ongoing enrollment in our clinical trials; existing or future collaboration agreements; the Company’s business strategy and plans; our anticipated financial performance and financial position for any period of time, including our estimated Non-GAAP Operating Expenses for the year ending December 31, 2026; and the sufficiency of our cash, cash equivalents and short-term investments to fund our operations for at least 24 months; and the timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise.
Non-GAAP Financial Measures
This press release includes Non-GAAP Operating Expenses, a supplemental measure of our performance that is not required by, or presented in accordance with, U.S. GAAP and should not be considered as an alternative to operating expenses or any other performance measure derived in accordance with GAAP.
We define Non-GAAP Operating Expenses as GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing.
We caution investors that amounts presented in accordance with our definition of Non-GAAP Operating Expenses may not be comparable to similar measures disclosed by our competitors because not all companies and analysts calculate this non-GAAP financial measure in the same manner. We present this non-GAAP financial measure because we consider it to be an important supplemental measure of our performance and believe it is frequently used by securities analysts, investors, and other interested parties in the evaluation of companies in our industry. Management believes that investors’ understanding of our performance is enhanced by including this non-GAAP financial measure as a reasonable basis for comparing our ongoing results of operations.
Management uses this non-GAAP financial measure for planning purposes, including the preparation of our internal annual operating budget and financial projections; to evaluate the performance and effectiveness of our operational strategies; and to evaluate our capacity to expand our business. This non-GAAP financial measure has limitations as an analytical tool, and should not be considered in isolation, or as an alternative to, or a substitute for operating expenses or other financial statement data presented in accordance with GAAP in our consolidated financial statements.
Rhythm has not provided a quantitative reconciliation of forecasted Non-GAAP Operating Expenses to forecasted GAAP operating expenses because the Company is unable, without making unreasonable efforts, to calculate the reconciling item, stock-based compensation expenses, with confidence. This item, which could materially affect the computation of forward-looking GAAP operating expenses, is inherently uncertain and depends on various factors, some of which are outside of Rhythm's control.
Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Media Contact:
Layne Cosgrove
Real Chemistry
410-916-1035
llitsinger@realchemistry.com
Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) |
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| Three months ended |
Six months ended |
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| 2026 | 2025 | 2026 | 2025 | |||||||||||
| Revenues: | ||||||||||||||
| Product revenue, net | $ | 71,255 | $ | 48,502 | $ | 131,367 | $ | 86,220 | ||||||
| License revenue | — | — | — | (5,014 | ) | |||||||||
| Total revenues | 71,255 | 48,502 | 131,367 | 81,206 | ||||||||||
| Costs and expenses: | ||||||||||||||
| Cost of sales | 8,931 | 5,543 | 16,088 | 9,191 | ||||||||||
| Research and development | 43,428 | 42,308 | 85,153 | 79,281 | ||||||||||
| Selling, general, and administrative | 67,448 | 45,947 | 131,039 | 85,034 | ||||||||||
| Total costs and expenses | 119,807 | 93,798 | 232,280 | 173,506 | ||||||||||
| Loss from operations | (48,552 | ) | (45,296 | ) | (100,913 | ) | (92,300 | ) | ||||||
| Other income (expense): | ||||||||||||||
| Other income (expense), net | 1,018 | 1,576 | (686 | ) | 932 | |||||||||
| Interest expense | (4,540 | ) | (5,817 | ) | (9,123 | ) | (11,226 | ) | ||||||
| Interest income | 3,220 | 3,242 | 6,774 | 6,881 | ||||||||||
| Total other income (expense), net | (302 | ) | (999 | ) | (3,035 | ) | (3,413 | ) | ||||||
| Loss before income taxes | (48,854 | ) | (46,295 | ) | (103,948 | ) | (95,713 | ) | ||||||
| Provision (benefit) for income taxes | 444 | 337 | 989 | 417 | ||||||||||
| Net loss | $ | (49,298 | ) | $ | (46,632 | ) | $ | (104,937 | ) | $ | (96,130 | ) | ||
| Accrued dividends on convertible preferred stock | (1,065 | ) | (1,349 | ) | (2,169 | ) | (2,671 | ) | ||||||
| Net loss attributable to common stockholders | $ | (50,363 | ) | $ | (47,981 | ) | $ | (107,106 | ) | $ | (98,801 | ) | ||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.73 | ) | $ | (0.75 | ) | $ | (1.57 | ) | $ | (1.56 | ) | ||
| Weighted-average common shares outstanding, basic and diluted | 68,592,661 | 63,684,359 | 68,285,135 | 63,373,489 | ||||||||||
| Net loss | $ | (49,298 | ) | $ | (47,981 | ) | $ | (104,937 | ) | $ | (98,801 | ) | ||
| Other comprehensive income (loss): | ||||||||||||||
| Foreign currency translation adjustment | (991 | ) | (2,104 | ) | 793 | (2106 | ) | |||||||
| Unrealized gain (loss), net on marketable securities | $ | (365 | ) | $ | (93 | ) | $ | (909 | ) | (103 | ) | |||
| Comprehensive loss | $ | (50,654 | ) | $ | (50,178 | ) | $ | (105,053 | ) | $ | (101,010 | ) | ||
Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) |
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2026 |
2025 |
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| Assets | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 65,614 | $ | 54,301 | |||
| Short-term investments | 265,283 | 334,648 | |||||
| Accounts receivable, net | 40,408 | 26,081 | |||||
| Inventory | 30,517 | 25,753 | |||||
| Prepaid expenses and other current assets | 24,741 | 26,133 | |||||
| Total current assets | 426,563 | 466,916 | |||||
| Property and equipment, net | 103 | 1,104 | |||||
| Right-of-use asset | 2,817 | 3,049 | |||||
| Intangible assets, net | 4,892 | 5,319 | |||||
| Restricted cash | 603 | 522 | |||||
| Other long-term assets | 2,288 | 3,286 | |||||
| Total assets | $ | 437,266 | $ | 480,196 | |||
| Liabilities, Convertible Preferred Stock and Stockholders’ equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 16,165 | $ | 13,947 | |||
| Accrued expenses and other current liabilities | 94,016 | 83,855 | |||||
| Lease liability | 703 | 650 | |||||
| Deferred revenue | — | 194 | |||||
| Deferred royalty obligation, current | 12,703 | 7,296 | |||||
| Total current liabilities | 123,587 | 105,942 | |||||
| Long-term liabilities: | |||||||
| Deferred royalty obligation | 93,399 | 100,886 | |||||
| Lease liability, non-current | 2,979 | 3,342 | |||||
| Total liabilities | 219,965 | 210,170 | |||||
| Commitments and contingencies (Note 14) | |||||||
| Series A convertible preferred stock, |
114,318 | 130,957 | |||||
| Stockholders’ equity: | |||||||
| Preferred stock, |
— | — | |||||
| Common stock, |
69 | 67 | |||||
| Additional paid-in capital | 1,560,640 | 1,491,675 | |||||
| Accumulated other comprehensive loss | (912 | ) | (796 | ) | |||
| Accumulated deficit | (1,456,814 | ) | (1,351,877 | ) | |||
| Total stockholders’ equity | 102,983 | 139,069 | |||||
| Total liabilities, convertible preferred stock and stockholders’ equity | $ | 437,266 | $ | 480,196 | |||
Source: Rhythm Pharmaceuticals, Inc.