Rhythm Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company, which engages in the development and commercialization of therapeutics for patients with rare diseases. The firm offers IMCIVREE (setmelanotide), a precision medicine designed to treat hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) diseases. It is also involved in the clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of investigational candidates for the treatment of congenital hyperinsulinism. The company was founded by Bart Henderson in November 2008 and is headquartered in Boston, MA.
Clinical Trials
74
43 active
Approvals
2
Total approvals
Agencies
2
Regulatory bodies
Founded
2010
Active, not recruiting
42
56.8%
Approved For Marketing
1
1.4%
Completed
24
32.4%
Enrolling By Invitation
1
1.4%
Recruiting
6
8.1%
- Rhythm Pharmaceuticals announced the appointment of Kim Popovits as a new Board Director, bringing over 40 years of life sciences leadership experience including her role as former CEO of Genomic Health. - Ed Mathers will depart the Board after serving since 2013, with CEO David Meeker thanking him for his dedicated service and strategic guidance during Rhythm's evolution. - Popovits' expertise in commercialization, reimbursement, and governance is expected to support Rhythm's mission and long-term growth as the company continues developing treatments for rare neuroendocrine diseases. - The leadership change comes as Rhythm advances its commercial-stage MC4R agonist setmelanotide (IMCIVREE) and pipeline candidates for rare obesity disorders and congenital hyperinsulinism.
- Rhythm Pharmaceuticals experienced a late-2025 stock decline after the FDA extended its review of setmelanotide for acquired hypothalamic obesity by three months to March 20, 2026. - The company's Phase 3 TRANSCEND trial demonstrated impressive results with setmelanotide achieving a -19.8% placebo-adjusted BMI reduction, and combination therapy with GLP-1 drugs showed BMI reductions up to -27.1%. - Despite regulatory delays, Rhythm maintains leadership in rare genetic obesity with 54% revenue growth and strong analyst support, while advancing oral candidate Bivamelagon through Phase 2 trials. - The FDA delay has intensified short interest to 30% of float and created opportunities for competitors like Soleno Therapeutics, which received approval for Vykat XR in Prader-Willi Syndrome.
- Rhythm Pharmaceuticals announced positive preliminary results from its exploratory Phase 2 trial of setmelanotide in Prader-Willi syndrome, showing BMI and hyperphagia reductions at Month 3 and Month 6. - Six of eight patients achieved BMI reductions at Month 3, while six of seven evaluable patients showed meaningful reduction in hyperphagia scores, with 17 of 18 enrolled patients remaining on therapy. - The company plans to advance setmelanotide into a Phase 3 registrational trial for PWS and has initiated a Phase 1 study of weekly MC4R agonist RM-718 in PWS patients. - PWS affects approximately 400,000 people worldwide with limited therapeutic options currently available to address the extreme hyperphagia and low resting energy expenditure associated with the condition.
- Rhythm Pharmaceuticals has secured Product Listing Agreements for IMCIVREE (setmelanotide) in five Canadian provinces and under the Federal Non-Insured Health Benefits Program for patients with Bardet-Biedl syndrome and obesity. - The reimbursement covers eligible adult and pediatric patients aged 6 years and older with clinically or genetically confirmed Bardet-Biedl syndrome and obesity in Ontario, Alberta, British Columbia, Saskatchewan, and Nova Scotia. - IMCIVREE was approved by Health Canada in May 2023 following priority review and was recently added to the common list of new drugs for rare diseases under Canada's National Strategy for Drugs for Rare Diseases. - The MC4R agonist setmelanotide addresses the root cause of obesity in Bardet-Biedl syndrome, offering hope for improved quality of life for patients and families affected by this rare genetic condition.
- Rhythm Pharmaceuticals achieved $51.3 million in global IMCIVREE sales for Q3 2025, representing a 6% sequential increase driven primarily by growth in Bardet-Biedl syndrome patients. - The FDA has set a December 20, 2025 PDUFA goal date for the company's supplemental new drug application seeking approval of setmelanotide for acquired hypothalamic obesity treatment. - A German observational study demonstrated that setmelanotide therapy improved measures of metabolic dysfunction-associated steatotic liver disease and kidney function in Bardet-Biedl syndrome patients. - The company expects to report preliminary results from its Phase 2 Prader-Willi syndrome trial in Q4 2025 and maintains a strong cash position of $416.1 million.
- Septerna has appointed Keith Gottesdiener, M.D., a veteran biotechnology executive with over 30 years of experience, to its board of directors and as chairperson of a newly constituted R&D Committee. - Dr. Gottesdiener brings extensive leadership experience from Prime Medicine, Rhythm Pharmaceuticals, and Merck Research Laboratories, where he oversaw the approval of more than 20 novel therapeutics. - The appointment comes at a pivotal time as Septerna advances its GPCR-targeted drug pipeline, including the promising PTH1R program for hypoparathyroidism. - Septerna's proprietary Native Complex Platform™ enables novel approaches to GPCR drug discovery and has led to a diverse pipeline of oral small molecule drug candidates.
- Rhythm Pharmaceuticals' setmelanotide achieved a 19.8% BMI reduction at 52 weeks in the Phase 3 TRANSCEND trial for acquired hypothalamic obesity, with 80% of patients achieving clinically meaningful weight loss. - The company plans to submit regulatory applications to FDA and EMA by Q3 2025 for the first-ever approved therapy for acquired hypothalamic obesity, affecting approximately 50,000 patients globally. - With no current approved treatments for this rare condition caused by hypothalamic damage, setmelanotide could capture a market worth $1.5-2.5 billion in peak sales at rare disease pricing levels.
- Obesity medication prescriptions among US adolescents aged 12-17 years increased by 301.7% between 2020 and 2023, driven primarily by FDA approvals of semaglutide and phentermine-topiramate for pediatric use. - Despite the dramatic increase, only 0.5% of adolescents with obesity received medication prescriptions in 2023, highlighting significant treatment gaps in this population. - Substantial disparities emerged in prescribing patterns, with Black adolescents receiving medications at lower rates than White adolescents despite having higher rates of severe obesity. - Access barriers including high out-of-pocket costs, insurance coverage limitations, and provider knowledge gaps continue to limit widespread adoption of evidence-based obesity treatments.
- DelveInsight's 2025 pipeline report reveals a robust obesity therapeutics landscape with over 80 companies developing more than 100 pipeline drugs across various clinical stages. - Major pharmaceutical companies including Eli Lilly, Novo Nordisk, Boehringer Ingelheim, and Carmot Therapeutics are advancing multiple Phase II and III trials scheduled for February 2025. - Leading drug candidates include survodutide (Zealand Pharma/Boehringer Ingelheim), a dual GLP-1/glucagon receptor agonist in Phase III trials, and CT-868 (Carmot Therapeutics), a dual GLP-1/GIP receptor modulator in Phase II development. - The pipeline encompasses diverse therapeutic approaches including dual hormone receptor agonists, combination therapies, and novel mechanisms targeting different molecular pathways for weight management.
- Rhythm Pharmaceuticals' Imcivree (setmelanotide) demonstrated a 19.8% placebo-adjusted reduction in BMI over 52 weeks in patients with acquired hypothalamic obesity, a rare condition caused by hypothalamic damage. - The TRANSCEND Phase III trial met its primary endpoint with 83% of treated patients achieving at least 5% BMI reduction, while placebo patients experienced a 3.3% BMI increase, supporting potential regulatory filings in Q3 2025. - If approved for hypothalamic obesity, analysts project Imcivree could exceed $1 billion in annual sales by 2028, significantly expanding its current indications for certain genetic obesity disorders.
