Rhythm Pharmaceuticals Reports Third Quarter 2025 Financial Results and Business Update
-- Third quarter 2025 net product revenue from global sales of IMCIVREE® (setmelanotide) of
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-- Company is on track to report preliminary results from setmelanotide Phase 2 trial in Prader-Willi syndrome in fourth quarter of 2025 --
-- German observational study showed setmelanotide therapy was associated with improvement in measures of MASLD and kidney function in BBS patients --
-- Management to host conference call today at 8:00 a.m. ET --
“Rhythm delivered continued momentum during the third quarter, highlighted by the FDA acceptance of our supplemental new drug application (sNDA) for acquired hypothalamic obesity (HO) and steady growth from our Bardet-Biedl syndrome (BBS) commercial efforts.” said
Recent Business Highlights
Revenue from global sales of IMCIVREE® (setmelanotide) was
Recent Clinical Development Highlights
- Today, Rhythm announced that four abstracts have been accepted for presentation – one oral presentation and three posters – at the Obesity Society’s Annual Meeting at ObesityWeek 2025 this week in
Atlanta , GA:- “Efficacy of Setmelanotide in Patients with Acquired HO Previously or Concurrently on GLP-1 Therapy," an oral presentation by
Christian Roth , M.D., Seattle Children’sResearch Institute ,Thursday, Nov. 6 , 9:00–9:15 a.m.; - “Cardiometabolic Results from a Phase 3 Trial of Setmelanotide in Acquired Hypothalamic Obesity,” a poster presentation by
Jill Garrison , Director, Medical Affairs,Rhythm Pharmaceuticals ,Nov. 4 , 7:30–8:30 p.m.; - “Patient- and Caregiver-Reported Experience with Acquired Hypothalamic Obesity in the TRANSCEND Trial,” a poster presentation by Dr. Roth
Nov. 4 , 7:30–8:30 p.m.; and - “Hyperphagia and the Identification of Genetic Variants in Patients with Early-Onset Obesity,” a poster presentation by
Roohi Kharofa , M.D., Cincinnati Children’sHospital Medical Center ,Nov. 5 , 2:30–3:30 p.m.
- “Efficacy of Setmelanotide in Patients with Acquired HO Previously or Concurrently on GLP-1 Therapy," an oral presentation by
- In September, researchers at the University Hospital Essen published results from a prospective, observational study that showed setmelanotide treatment was associated with improvement in measures of metabolic dysfunction-associated steatotic liver disease (MASLD) and kidney function in patients with BBS in the
Journal of Clinical Endocrinology & Metabolism 1. All patients (N=26) in this study had MASLD at baseline and, after six months of setmelanotide treatment, >80% of them achieved either resolution of MASLD or stabilization at grade S1; and - On
August 20, 2025 , Rhythm announced that theU.S. Food and Drug Administration (FDA) accepted for filing the Company’s sNDA for setmelanotide seeking approval for the treatment of conditions associated with acquired hypothalamic obesity. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date ofDecember 20, 2025 . Additionally, theEuropean Medicines Agency (EMA) confirmed validation of the Type II variation submission to the Marketing Authorization Application (MAA) for setmelanotide for the same indication.
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
- Disclose preliminary results from the Company’s Phase 2 trial evaluating setmelanotide in Prader-Willi syndrome in the fourth quarter of 2025;
- Receive a decision from the FDA on our sNDA (anticipated to be received by the PDUFA goal date of
December 20, 2025 ) for setmelanotide in conditions associated with acquired hypothalamic obesity; - Complete enrollment in the Phase 1, Part C trial evaluating the weekly, MC4R agonist
RM-718 in patients with acquired hypothalamic obesity in the first quarter of 2026; - Announce topline data in the 12-patient Japanese cohort of the setmelanotide Phase 3 trial in acquired hypothalamic obesity in the first quarter of 2026;
- Announce topline data in the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases in the first quarter of 2026;
- Complete enrollment in the setmelanotide Phase 3 trial substudy in congenital hypothalamic obesity in the first half of 2026; and
- Pending further feedback from
U.S. and European regulatory agencies, initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired hypothalamic obesity in 2026.
Third Quarter 2025 Financial Results:
Cash Position: As of
Revenue: Net product revenues relating to global 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: Net loss attributable to common stockholders was
Year to Date 2025 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: Net loss attributable to common stockholders was
Financial Guidance: Today, Rhythm updated its financial guidance for the year ending
- GAAP total operating expenses, inclusive of:
- SG&A expenses of approximately
$145 million to$150 million ; - R&D expenses of approximately
$150 million to$165 million ; and - Excluding stock-based compensation.
- SG&A expenses of approximately
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
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 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 is approved by the FDA to reduce excess body weight and maintain weight reduction long term in 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
Setmelanotide Indication
In
In the
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
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
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
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,
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:
Head of Investor Relations and Corporate Communications
857-264-4280
dconnolly@rhythmtx.com
Media Contact:
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 |
Nine months ended |
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| 2025 |
2024 |
2025 |
2024 |
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| Revenues: | |||||||||||||||
| Product revenue, net | $ | 51,298 | $ | 33,251 | $ | 137,518 | $ | 88,296 | |||||||
| License revenue | — | — | (5,014 | ) | — | ||||||||||
| Total revenues | 51,298 | 33,251 | 132,504 | 88,296 | |||||||||||
| Costs and expenses: | |||||||||||||||
| Cost of sales | 5,499 | 3,828 | 14,690 | 9,581 | |||||||||||
| Research and development | 46,027 | 37,931 | 125,307 | 196,789 | |||||||||||
| Selling, general, and administrative | 52,425 | 35,377 | 137,460 | 106,174 | |||||||||||
| Total costs and expenses | 103,951 | 77,136 | 277,457 | 312,544 | |||||||||||
| Loss from operations | (52,653 | ) | (43,885 | ) | (144,953 | ) | (224,248 | ) | |||||||
| Other income (expense): | |||||||||||||||
| Other income (expense), net | 274 | 1,088 | 1,207 | 2,434 | |||||||||||
| Gain on settlement of forward contract | — | — | — | 8,900 | |||||||||||
| Interest expense | (4,742 | ) | (5,242 | ) | (15,968 | ) | (15,156 | ) | |||||||
| Interest income | 4,328 | 4,054 | 11,208 | 11,196 | |||||||||||
| Total other income (expense), net | (140 | ) | (100 | ) | (3,553 | ) | 7,374 | ||||||||
| Loss before income taxes | (52,793 | ) | (43,985 | ) | (148,506 | ) | (216,874 | ) | |||||||
| Provision (benefit) for income taxes | 111 | (344 | ) | 528 | 436 | ||||||||||
| Net loss | $ | (52,904 | ) | $ | (43,641 | ) | $ | (149,034 | ) | $ | (217,310 | ) | |||
| Accrued dividends on convertible preferred stock | (1,375 | ) | (1,329 | ) | (4,046 | ) | (2,631 | ) | |||||||
| Net loss attributable to common stockholders | $ | (54,279 | ) | $ | (44,970 | ) | $ | (153,080 | ) | $ | (219,941 | ) | |||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.82 | ) | $ | (0.73 | ) | $ | (2.38 | ) | $ | (3.62 | ) | |||
| Weighted-average common shares outstanding, basic and diluted | 66,261,055 | 61,219,918 | 64,346,588 | 60,793,329 | |||||||||||
| Other comprehensive loss: | |||||||||||||||
| Net loss attributable to common stockholders | $ | (54,279 | ) | $ | (44,970 | ) | $ | (153,080 | ) | $ | (219,941 | ) | |||
| Foreign currency translation adjustment | 834 | (602 | ) | (1,272 | ) | (975 | ) | ||||||||
| Unrealized gain (loss), net on marketable securities | 488 | 615 | 385 | 237 | |||||||||||
| Comprehensive loss | $ | (52,957 | ) | $ | (44,957 | ) | $ | (153,967 | ) | $ | (220,679 | ) | |||
Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) |
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| 2025 |
2024 |
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| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 52,078 | $ | 89,137 | ||||
| Short-term investments | 363,974 | 231,428 | ||||||
| Accounts receivable, net | 33,034 | 18,512 | ||||||
| Inventory | 22,107 | 18,741 | ||||||
| Prepaid expenses and other current assets | 23,609 | 16,382 | ||||||
| Total current assets | 494,802 | 374,200 | ||||||
| Property and equipment, net | 237 | 632 | ||||||
| Right-of-use asset | 3,158 | 3,477 | ||||||
| Intangible assets, net | 5,533 | 6,174 | ||||||
| Restricted cash | 671 | 464 | ||||||
| Other long-term assets | 2,473 | 7,326 | ||||||
| Total assets | $ | 506,874 | $ | 392,273 | ||||
| Liabilities, Convertible Preferred Stock and Stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 13,631 | $ | 12,328 | ||||
| Accrued expenses and other current liabilities | 84,456 | 62,658 | ||||||
| Other current liability - LG Chem | — | 37,704 | ||||||
| Lease liability | 624 | — | ||||||
| Deferred revenue | — | 1,286 | ||||||
| Deferred royalty obligation, current | 5,556 | 1,541 | ||||||
| Total current liabilities | 104,267 | 115,517 | ||||||
| Long-term liabilities: | ||||||||
| Deferred royalty obligation | 103,409 | 108,269 | ||||||
| Lease liability, non-current | 3,515 | 3,938 | ||||||
| Total liabilities | 211,191 | 227,724 | ||||||
| Commitments and contingencies (Note 14) | ||||||||
| Series A convertible preferred stock, |
146,866 | 142,820 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
67 | 61 | ||||||
| Additional paid-in capital | 1,454,048 | 1,177,045 | ||||||
| Accumulated other comprehensive (loss) | (926 | ) | (39 | ) | ||||
| Accumulated deficit | (1,304,372 | ) | (1,155,338 | ) | ||||
| Total stockholders’ equity | 148,817 | 21,729 | ||||||
| Total liabilities, convertible preferred stock and stockholders’ equity | $ | 506,874 | $ | 392,273 | ||||
1 Dr.
Source: Rhythm Pharmaceuticals, Inc.