Rhythm Pharmaceuticals Reports First Quarter 2026 Financial Results and Business Update
-- IMCIVREE® (setmelanotide) launched in the
-- First quarter 2026 net product revenue from global sales of IMCIVREE of
-- IMCIVREE granted Marketing Authorization by
-- Japanese regulatory review of New Drug Application for setmelanotide to treat acquired hypothalamic obesity underway --
-- Management to host conference call today at 8:00 a.m. ET --
“The
Recent Business and Development Highlights
- Today, the Company announced that more than 150 patient start forms had been received for IMCIVREE in acquired HO, within the first six weeks following approval by the
U.S. Food and Drug Administration (FDA) onMarch 19, 2026 ; - Revenue from global sales of IMCIVREE was
$60.1 million for the first quarter of 2026, an increase of 5% on a sequential basis from the fourth quarter of 2025, primarily driven by sales of IMCIVREE for the treatment of patients with Bardet-Biedl syndrome (BBS) and an increase in the number of patients on reimbursed therapy globally. In the first quarter of 2026, revenue of$36.9 million , or 61% of product revenue, was generated inthe United States , a decrease of 5% on a sequential basis primarily driven by specialty pharmacy inventory and some patients receiving drug under the Company's bridging program as they transitioned insurance plans during the quarter. Revenue of$23.2 million , or 39% of product revenue, was generated outsidethe United States , a sequential increase of$4.9 million or 27%; - Today, the Company announced positive data from the Japanese cohort of its Phase 3 TRANSCEND study and that its Japanese New Drug Application (JNDA) for setmelanotide to treat acquired HO has been accepted, validated and is now under review by Japan’s
Pharmaceuticals and Medical Devices Agency (PMDA). The Company anticipates PMDA's decision on the application in the second half of 2026 and, if positive, expects commercial launch by the end of 2026; - On
May 4, 2026 , the Company announced new data presented at thePediatric Endocrine Society Annual Meeting demonstrating sustained reductions in BMI and BMI Z‑score through 2.5 years of setmelanotide treatment and observed weight category improvements in pediatric patients (n=10) with acquired HO and a second presentation demonstrating weight category improvement in the pediatric subpopulation of participants after 1 year of setmelanotide treatment; - On
May 1, 2026 , the Company announced theEuropean Commission granted Marketing Authorization to IMCIVREE for the treatment of obesity and control of hunger in patients with acquired HO, following the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adoption of a positive opinion recommending expansion of the current marketing authorization to include the treatment of acquired HO onMarch 26, 2026 ; - On
April 3, 2026 , the Company announced the appointment ofKim Popovits , the former Chairman of the Board, Chief Executive Officer and President of Genomic Health, to Rhythm’s Board of Directors and the resignation ofEd Mathers ; - On
March 19, 2026 , the Company announced IMCIVREE was approved by the FDA for the treatment of acquired HO; - On
March 16, 2026 , Rhythm announced topline results from the Phase 3 EMANATE trial. The trial did not achieve the primary endpoint in each of its four independent substudies evaluating setmelanotide in genetically caused MC4R pathway diseases. The Company reported positive signals from post-hoc analyses in two genetic indications, SRC1 (NCOA1) deficiency and POMC insufficiency, and continues to explore a path forward with next-generation MC4R agonists bivamelagon andRM-718 ; - On
March 1, 2026 , the Company announced additional positive data from its global Phase 3 TRANSCEND trial of setmelanotide in patients with acquired HO. This new data set included 12 patients from a Japanese cohort and 10 supplemental patients who were enrolled in addition to the primary 120-patient pivotal cohort; and - On
February 26, 2026 , the Company announced it completed an End-of-Phase-2 meeting with FDA regarding bivamelagon in acquired HO and disclosed encouraging open-label extension data from its Phase 2 trial that showed bivamelagon achieved persistent BMI reductions at six and nine months of therapy.
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
- Announce six-month results from the ongoing exploratory Phase 2 trial of setmelanotide in Prader-Willi Syndrome (PWS) in the second quarter of 2026;
- Announce results from the Phase 1/2, Part C trial evaluating the weekly, MC4R agonist
RM-718 in patients with acquired HO mid-year 2026; - Decision by Japan’s PMDA for IMCIVREE in acquired HO anticipated in the second half of 2026;
- 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; - Initiate a pivotal Phase 3 trial evaluating bivamelagon in acquired HO by year-end 2026.
First Quarter 2026 Financial Results
Cash Position: As of March 31, 2026, cash, cash equivalents and short-term investments were approximately
Revenue: Net product revenues from global sales of IMCIVREE were $60.1 million for the first quarter of 2026, as compared to $37.7 million for the first quarter of 2025.
R&D Expenses: R&D expenses were
SG&A Expenses: SG&A expenses were
Other expense, net: Other expense, net was $(2.7) million for the first quarter of 2026, as compared to other expense, net of
Net Loss: Net loss attributable to common stockholders was $(56.7) million for the first quarter of 2026, or a net loss per basic and diluted share of $(0.83), as compared to a net loss attributable to common stockholders of $(50.8) million for the first quarter of 2025, or a net loss per basic and diluted share of $(0.81).
Financial Guidance: For the year ending
- GAAP total operating expenses, inclusive of:
- R&D expenses of approximately $197 million to $213 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 March 31, 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 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
410-916-1035
llitsinger@realchemistry.com
Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) |
||||||||
| Three months ended |
||||||||
| 2026 | 2025 | |||||||
| Revenues: | ||||||||
| Product revenue, net | $ | 60,112 | $ | 37,718 | ||||
| License revenue | — | (5,014 | ) | |||||
| Total revenues | 60,112 | 32,704 | ||||||
| Costs and expenses: | ||||||||
| Cost of sales | 7,157 | 3,648 | ||||||
| Research and development | 41,725 | 36,973 | ||||||
| Selling, general, and administrative | 63,591 | 39,087 | ||||||
| Total costs and expenses | 112,473 | 79,708 | ||||||
| Loss from operations | (52,361 | ) | (47,004 | ) | ||||
| Other income (expense): | ||||||||
| Other income (expense), net | (1,704 | ) | (644 | ) | ||||
| Interest expense | (4,583 | ) | (5,409 | ) | ||||
| Interest income | 3,554 | 3,639 | ||||||
| Total other income (expense), net | (2,733 | ) | (2,414 | ) | ||||
| Loss before income taxes | (55,094 | ) | (49,418 | ) | ||||
| Provision (benefit) for income taxes | 545 | 80 | ||||||
| Net loss | $ | (55,639 | ) | $ | (49,498 | ) | ||
| Accrued dividends on convertible preferred stock | (1,104 | ) | (1,322 | ) | ||||
| Net loss attributable to common stockholders | $ | (56,743 | ) | $ | (50,820 | ) | ||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.83 | ) | $ | (0.81 | ) | ||
| Weighted-average common shares outstanding, basic and diluted | 67,974,193 | 63,059,165 | ||||||
| Net loss | $ | (55,639 | ) | $ | (50,820 | ) | ||
| Other comprehensive income (loss): | ||||||||
| Foreign currency translation adjustment | 1,784 | (2 | ) | |||||
| Unrealized gain (loss), net on marketable securities | $ | (544 | ) | (10 | ) | |||
| Comprehensive loss | $ | (54,399 | ) | $ | (50,832 | ) | ||
Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) |
||||||||
2026 |
2025 |
|||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 62,137 | $ | 54,301 | ||||
| Short-term investments | 278,488 | 334,648 | ||||||
| Accounts receivable, net | 34,192 | 26,081 | ||||||
| Inventory | 28,651 | 25,753 | ||||||
| Prepaid expenses and other current assets | 26,622 | 26,133 | ||||||
| Total current assets | 430,090 | 466,916 | ||||||
| Property and equipment, net | 1,065 | 1,104 | ||||||
| Right-of-use asset | 2,936 | 3,049 | ||||||
| Intangible assets, net | 5,106 | 5,319 | ||||||
| Restricted cash | 601 | 522 | ||||||
| Other long-term assets | 2,518 | 3,286 | ||||||
| Total assets | $ | 442,316 | $ | 480,196 | ||||
| Liabilities, Convertible Preferred Stock and Stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 12,299 | $ | 13,947 | ||||
| Accrued expenses and other current liabilities | 80,045 | 83,855 | ||||||
| Lease liability | 676 | 650 | ||||||
| Deferred revenue | 48 | 194 | ||||||
| Deferred royalty obligation, current | 10,130 | 7,296 | ||||||
| Total current liabilities | 103,198 | 105,942 | ||||||
| Long-term liabilities: | ||||||||
| Deferred royalty obligation | 98,339 | 100,886 | ||||||
| Lease liability, non-current | 3,163 | 3,342 | ||||||
| Total liabilities | 204,700 | 210,170 | ||||||
| Commitments and contingencies (Note 14) | ||||||||
| Series A convertible preferred stock, |
114,710 | 130,957 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
69 | 67 | ||||||
| Additional paid-in capital | 1,529,909 | 1,491,675 | ||||||
| Accumulated other comprehensive income (loss) | 444 | (796 | ) | |||||
| Accumulated deficit | (1,407,516 | ) | (1,351,877 | ) | ||||
| Total stockholders’ equity | 122,906 | 139,069 | ||||||
| Total liabilities, convertible preferred stock and stockholders’ equity | $ | 442,316 | $ | 480,196 | ||||
Source: Rhythm Pharmaceuticals, Inc.