Rhythm Pharmaceuticals Reports First Quarter 2025 Financial Results and Business Update
-- First quarter 2025 net product revenue from global sales of IMCIVREE® (setmelanotide) of $37.7 million --
-- Setmelanotide pivotal Phase 3 TRANSCEND trial met primary endpoint with -19.8% placebo-adjusted BMI reduction in patients (N=120) with acquired hypothalamic obesity --
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-- Topline data from Phase 2 trial of oral MC4R agonist bivamelagon on track to be announced in third quarter of 2025 --
-- Cash on-hand expected to support planned operations into 2027 --
-- Management to host conference call today at 8:00 a.m. ET --
“Following compelling topline results from our pivotal Phase 3 trial of setmelanotide in acquired hypothalamic obesity (HO), we look forward to completing
First Quarter and Recent Business Highlights
- Revenue from global sales of IMCIVREE (setmelanotide) was
$37.7 million for the first quarter of 2025. The number of patients on reimbursed therapy increased 14% in the first quarter of 2025 compared to the fourth quarter of 2024, as patient demand for IMCIVREE remained strong. Revenue of$24.5 million , or 65% of product revenue, was generated inthe United States . The number of patients on reimbursed therapy inthe United States continued to increase during the quarter.U.S. revenue was affected by an$8.3 million decrease in inventory at the specialty pharmacy that dispenses IMCIVREE to patients and a$1.1 million increase in product dispensed to patients, resulting in a net decrease inU.S. product revenue of$7.2 million in the first quarter of 2025 compared to the fourth quarter of 2024. Revenue of$13.2 million , or 35% of product revenue, was generated outsidethe United States , an increase of$3.2 million quarter over quarter. - On
April 7, 2025 , Rhythm announced its pivotal Phase 3 TRANSCEND trial met its primary endpoint with a -19.8% placebo-adjusted body mass index (BMI) reduction with setmelanotide in patients (N=120) with acquired HO;- Patients with acquired HO on setmelanotide therapy (n=81) achieved mean BMI change of -16.5% compared with +3.3% for placebo (n=39) at 52 weeks (p<0.0001);
- -19.2% placebo-adjusted BMI reduction achieved in adult patients 18 years old and older (n=49) at 52 weeks;
- -20.2% placebo-adjusted BMI reduction achieved in patients younger than 18 years old (n=71) at 52 weeks;
- 80% of patients on setmelanotide achieved BMI reduction of 5% or greater at 52 weeks; and
- No new safety signals with setmelanotide were observed, in line with setmelanotide’s well-established and well-understood safety profile.
- Today, Rhythm announced new data from the Phase 3 TRANSCEND trial that demonstrated a consistent and statistically significant mean BMI reduction across three stratified age groups:
- -19.5% placebo-adjusted BMI reduction achieved in pediatric patients ages 4 to younger than 12 years old (n=31: 20 setmelanotide, 11 placebo) at 52 weeks (p<0.0001);
- -21.0% placebo-adjusted BMI reduction achieved in adolescent patients ages 12 to younger than 18 years old (n=40: 28 setmelanotide, 12 placebo) at 52 weeks (p<0.0001); and
- -19.2% placebo-adjusted BMI reduction achieved in adult patients ages 18 and older (n=49: 33 setmelanotide, 16 placebo) at 52 weeks (p<0.0001).
- On
April 7, 2025 , Rhythm announced it dosed the first patients with Prader-Willi syndrome (PWS) in a 26-week, open-label Phase 2 trial of setmelanotide. The trial will assess the safety and efficacy of a daily dose of subcutaneous setmelanotide in approximately 20 patients for up to 26 weeks; - On
March 20, 2025 , Rhythm announced it reacquired the rights to IMCIVREE inChina , including mainlandChina ,Hong Kong andMacau , as the Company agreed to terminate its 2021 licensing agreement withRareStone Group Ltd. - On
March 19, 2025 , Rhythm announced it received orphan drug designation from Japan’sMinistry of Health, Labour and Welfare (MHLW) for setmelanotide as a treatment for acquired hypothalamic obesity; and - On
March 18, 2025 , Rhythm announced a new research collaboration with theRaymond A. Wood Foundation , a patient advocacy organization for survivors of craniopharyngioma and hypothalamic-pituitary brain tumors, to study the impact of fatigue on persons with craniopharyngioma.
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
- Submit a supplemental New Drug Application to the FDA and a Type II variation request to the
European Medicines Agency for setmelanotide for the treatment of patients with acquired HO in the third quarter of 2025; - Announce topline data from the bivamelagon Phase 2 trial in acquired HO in the third quarter of 2025;
- Complete enrollment in the setmelanotide Phase 2 trial in PWS 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 HO in the second half of 2025; - Complete enrollment in the setmelanotide Phase 3 trial substudy in congenital HO in the second half of 2025;
- Announce topline data in the 12-patient Japanese cohort of the setmelanotide Phase 3 trial in acquired HO in the first quarter of 2026; and
- Announce topline data in the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases in the first quarter of 2026.
First 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 expense, net: Other 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 existing 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
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
(949) 903-4750
sseapy@realchemistry.com
| Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) |
||||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 106,095 | $ | 89,137 | ||||
| Short-term investments | 208,393 | 231,428 | ||||||
| Accounts receivable, net | 17,825 | 18,512 | ||||||
| Inventory | 19,316 | 18,741 | ||||||
| Prepaid expenses and other current assets | 17,659 | 16,382 | ||||||
| Total current assets | 369,288 | 374,200 | ||||||
| Property and equipment, net | 462 | 632 | ||||||
| Right-of-use asset | 3,367 | 3,477 | ||||||
| Intangible assets, net | 5,960 | 6,174 | ||||||
| Restricted cash | 464 | 464 | ||||||
| Other long-term assets | 7,144 | 7,326 | ||||||
| Total assets | $ | 386,685 | $ | 392,273 | ||||
| Liabilities, Convertible Preferred Stock and Stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 11,912 | $ | 12,328 | ||||
| Accrued expenses and other current liabilities | 59,314 | 62,658 | ||||||
| Other current liability - LG Chem | 38,783 | 37,704 | ||||||
| Lease liability | 224 | — | ||||||
| Deferred revenue | — | 1,286 | ||||||
| Deferred royalty obligation, current | 1,590 | 1,541 | ||||||
| Total current liabilities | 111,823 | 115,517 | ||||||
| Long-term liabilities: | ||||||||
| Deferred royalty obligation | 107,934 | 108,269 | ||||||
| Lease liability, non-current | 3,839 | 3,938 | ||||||
| Total liabilities | 223,596 | 227,724 | ||||||
| Commitments and contingencies (Note 13) | ||||||||
| Series A convertible preferred stock, |
144,142 | 142,820 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
62 | 61 | ||||||
| Additional paid-in capital | 1,223,772 | 1,177,045 | ||||||
| Accumulated other comprehensive (loss) | (51 | ) | (39 | ) | ||||
| Accumulated deficit | (1,204,836 | ) | (1,155,338 | ) | ||||
| Total stockholders’ equity | 18,947 | 21,729 | ||||||
| Total liabilities, convertible preferred stock and stockholders’ equity | $ | 386,685 | $ | 392,273 | ||||
Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) |
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| Three months ended |
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| 2025 | 2024 | |||||||
| Revenues: | ||||||||
| Product revenue, net | $ | 37,718 | $ | 25,967 | ||||
| License revenue | (5,014 | ) | — | |||||
| Total revenues | 32,704 | 25,967 | ||||||
| Costs and expenses: | ||||||||
| Cost of sales | 3,648 | 2,807 | ||||||
| Research and development | 36,973 | 128,665 | ||||||
| Selling, general, and administrative | 39,087 | 34,382 | ||||||
| Total costs and expenses | 79,708 | 165,854 | ||||||
| Loss from operations | (47,004 | ) | (139,887 | ) | ||||
| Other income (expense): | ||||||||
| Other income (expense), net | (644 | ) | 524 | |||||
| Interest expense | (5,409 | ) | (4,755 | ) | ||||
| Interest income | 3,639 | 3,046 | ||||||
| Total other (expense), net | (2,414 | ) | (1,185 | ) | ||||
| Loss before income taxes | (49,418 | ) | (141,072 | ) | ||||
| Provision for income taxes | 80 | 300 | ||||||
| Net loss | $ | (49,498 | ) | $ | (141,372 | ) | ||
| Accrued dividends on convertible preferred stock | (1,322 | ) | — | |||||
| Net loss attributable to common stockholders | $ | (50,820 | ) | $ | (141,372 | ) | ||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.81 | ) | $ | (2.35 | ) | ||
| Weighted-average common shares outstanding, basic and diluted | 63,059,165 | 60,143,558 | ||||||
| Other comprehensive loss: | ||||||||
| Net loss attributable to common stockholders | $ | (50,820 | ) | $ | (141,372 | ) | ||
| Foreign currency translation adjustment | (2 | ) | (71 | ) | ||||
| Unrealized (loss), net on marketable securities | (10 | ) | (244 | ) | ||||
| Comprehensive loss | $ | (50,832 | ) | $ | (141,687 | ) | ||
Source: Rhythm Pharmaceuticals, Inc.