Rhythm Pharmaceuticals Reports Second Quarter 2025 Financial Results and Business Update
-- Second quarter 2025 net product revenue from global sales of IMCIVREE® (setmelanotide) of
--
-- Bivamelagon Phase 2 trial met primary endpoint with statistically significant, clinically meaningful BMI reductions in patients with acquired hypothalamic obesity --
-- Raised approximately
-- Management to host conference call today at 8:00 a.m. ET --
“Rhythm has made significant progress in advancing our melanocortin-4 receptor agonism platform and executing on our global mission to transform the lives of patients with rare neuroendocrine diseases,” said
Second Quarter and Recent Business Highlights
- Revenue from global sales of IMCIVREE was
$48.5 million for the second quarter of 2025, an increase of 29% percent on a sequential basis from the first quarter of 2025, primarily driven by sales of IMCIVREE for the treatment of patients with Bardet-Biedl syndrome (BBS). In the second quarter of 2025, revenue of$32.0 million , or 66% of product revenue, was generated inthe United States , an increase of 31% on a sequential basis. Revenue of$16.5 million , or 34% of product revenue, was generated outsidethe United States , a sequential increase of 24%; and - On
July 11, 2025 , Rhythm closed an upsized public offering of 2,367,647 shares of its common stock at a price of$85 per share, resulting in net proceeds of approximately$189.2 million , net of underwriting discounts and commissions, but excluding certain other offering expenses payable by the Company.
Second Quarter and Recent Clinical Development Highlights
- Today, Rhythm announced that it has enrolled the first patient with hypothalamic obesity in Part C of its Phase 1 trial evaluating
RM-718 , a weekly-administered melanocortin-4 receptor (MC4R) agonist; - On
July 12, 2025 , at the Endocrine Society’s Annual Meeting, data from the Company’s pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in acquired hypothalamic obesity, the largest randomized, placebo-controlled trial in acquired hypothalamic obesity to date, were delivered in an oral presentation. Highlights of the presentation included:- -19.8% placebo-adjusted difference in BMI reduction (N=120); and
- Statistically significant BMI reductions following setmelanotide treatment were consistently observed across subgroups stratified by age (<12, 12 to 17, <18, and 18 years and older; ranging from -15.6% to -17.2%) and by sex (-16.3% female; -16.8% male);
- On
July 9, 2025 , Rhythm announced bivamelagon achieved statistically significant and clinically meaningful reductions in body mass index (BMI) at 14 weeks of treatment in its Phase 2 trial in patients with acquired hypothalamic obesity, including;- -9.3% BMI reduction from baseline in the 600mg cohort (n=8) (p-value=0.0004);
- -7.7% BMI reduction from baseline in the 400mg cohort (n=7) (p-value=0.0002);
- Post-hoc analyses showing bivamelagon demonstrated BMI reductions consistent with BMI reductions achieved with setmelanotide therapy as observed in similar patient populations at comparable dosing durations; and
- Safety and tolerability results were consistent with MC4R agonism and mechanism of action during the placebo-controlled portion of the trial; and
- During the
Joint Congress between theEuropean Society for Paediatric Endocrinology and theEuropean Society of Endocrinology (ESPE-ESE) and theEuropean Congress on Obesity (ECO) inMay 2025 , Rhythm presented new, real-world data that showed consistent improvements in body mass index, BMI-Z, and hunger scores in 30 patients with acquired hypothalamic obesity and five (5) patients with congenital hypothalamic obesity who were treated with setmelanotide for up to nine months.
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
- Complete submissions of a supplemental New Drug Application to the
U.S. Food and Drug Administration (FDA) and a Type II variation request to theEuropean Medicines Agency seeking approval for setmelanotide for the treatment of acquired hypothalamic obesity in the third quarter of 2025; - Disclose preliminary results from the Company’s setmelanotide Phase 2 trial in Prader-Willi syndrome in the second half of 2025;
- 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 alignment with
U.S and European regulatory agencies, initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired hypothalamic obesity in 2026.
In addition, Rhythm announced today that it plans to host “Commercial Readiness for Acquired Hypothalamic Obesity”, an in-person and webcasted event for investors and analysts, on
Second 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: For the year ending
- GAAP total operating expenses, inclusive of:
- SG&A expenses of approximately
$135 million to$145 million ; - R&D expenses of approximately
$150 million to$170 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 designed to treat hyperphagia and severe obesity, 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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| 2025 | 2024 | 2025 | 2024 | ||||||||||||
| Revenues: | |||||||||||||||
| Product revenue, net | $ | 48,502 | $ | 29,078 | $ | 86,220 | $ | 55,045 | |||||||
| License revenue | — | — | (5,014 | ) | — | ||||||||||
| Total revenues | 48,502 | 29,078 | 81,206 | 55,045 | |||||||||||
| Costs and expenses: | |||||||||||||||
| Cost of sales | 5,543 | 2,947 | 9,191 | 5,753 | |||||||||||
| Research and development | 42,308 | 30,194 | 79,281 | 158,858 | |||||||||||
| Selling, general, and administrative | 45,947 | 36,415 | 85,034 | 70,797 | |||||||||||
| Total costs and expenses | 93,798 | 69,556 | 173,506 | 235,408 | |||||||||||
| Loss from operations | (45,296 | ) | (40,478 | ) | (92,300 | ) | (180,363 | ) | |||||||
| Other income (expense): | |||||||||||||||
| Other income (expense), net | 1,576 | 302 | 932 | 824 | |||||||||||
| Gain on settlement of forward contract | — | 8,900 | — | 8,900 | |||||||||||
| Interest expense | (5,817 | ) | (4,603 | ) | (11,226 | ) | (9,358 | ) | |||||||
| Interest income | 3,242 | 4,097 | 6,881 | 7,143 | |||||||||||
| Total other (expense), net | (999 | ) | 8,696 | (3,413 | ) | 7,509 | |||||||||
| Loss before income taxes | (46,295 | ) | (31,782 | ) | (95,713 | ) | (172,854 | ) | |||||||
| Provision for income taxes | 337 | 479 | 417 | 779 | |||||||||||
| Net loss | $ | (46,632 | ) | $ | (32,261 | ) | $ | (96,130 | ) | $ | (173,633 | ) | |||
| Accrued dividends on convertible preferred stock | (1,349 | ) | (1,302 | ) | (2,671 | ) | (1,302 | ) | |||||||
| Net loss attributable to common stockholders | $ | (47,981 | ) | $ | (33,563 | ) | $ | (98,801 | ) | $ | (174,935 | ) | |||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.75 | ) | $ | (0.55 | ) | $ | (1.56 | ) | $ | (2.89 | ) | |||
| Weighted-average common shares outstanding, basic and diluted | 63,684,359 | 61,011,824 | 63,373,489 | 60,577,691 | |||||||||||
| Other comprehensive loss: | |||||||||||||||
| Net loss attributable to common stockholders | $ | (47,981 | ) | $ | (33,563 | ) | $ | (98,801 | ) | $ | (174,935 | ) | |||
| Foreign currency translation adjustment | (2,104 | ) | (302 | ) | (2,106 | ) | (373 | ) | |||||||
| Unrealized (loss), net on marketable securities | (93 | ) | (134 | ) | (103 | ) | (378 | ) | |||||||
| Comprehensive loss | $ | (50,178 | ) | $ | (33,999 | ) | $ | (101,010 | ) | $ | (175,686 | ) | |||
Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) |
||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 135,586 | $ | 89,137 | ||||
| Short-term investments | 155,444 | 231,428 | ||||||
| Accounts receivable, net | 26,122 | 18,512 | ||||||
| Inventory | 18,872 | 18,741 | ||||||
| Prepaid expenses and other current assets | 24,656 | 16,382 | ||||||
| Total current assets | 360,680 | 374,200 | ||||||
| Property and equipment, net | 297 | 632 | ||||||
| Right-of-use asset | 3,262 | 3,477 | ||||||
| Intangible assets, net | 5,747 | 6,174 | ||||||
| Restricted cash | 527 | 464 | ||||||
| Other long-term assets | 2,220 | 7,326 | ||||||
| Total assets | $ | 372,733 | $ | 392,273 | ||||
| Liabilities, Convertible Preferred Stock and Stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 15,982 | $ | 12,328 | ||||
| Accrued expenses and other current liabilities | 69,185 | 62,658 | ||||||
| Other current liability - LG Chem | 40,000 | 37,704 | ||||||
| Lease liability | 510 | — | ||||||
| Deferred revenue | — | 1,286 | ||||||
| Deferred royalty obligation, current | 3,778 | 1,541 | ||||||
| Total current liabilities | 129,455 | 115,517 | ||||||
| Long-term liabilities: | ||||||||
| Deferred royalty obligation | 106,014 | 108,269 | ||||||
| Lease liability, non-current | 3,681 | 3,938 | ||||||
| Total liabilities | 239,150 | 227,724 | ||||||
| Commitments and contingencies (Note 13) | ||||||||
| Series A convertible preferred stock, |
145,491 | 142,820 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
64 | 61 | ||||||
| Additional paid-in capital | 1,241,744 | 1,177,045 | ||||||
| Accumulated other comprehensive (loss) | (2,248 | ) | (39 | ) | ||||
| Accumulated deficit | (1,251,468 | ) | (1,155,338 | ) | ||||
| Total stockholders’ equity | (11,908 | ) | 21,729 | |||||
| Total liabilities, convertible preferred stock and stockholders’ equity | $ | 372,733 | $ | 392,273 | ||||
Source: Rhythm Pharmaceuticals, Inc.