Rhythm Pharmaceuticals, Inc.

Rhythm Pharmaceuticals Announces Commercial Launch of IMCIVREE® (setmelanotide) in Japan for Patients with Acquired Hypothalamic Obesity

Rhythm Pharmaceuticals launched IMCIVREE (setmelanotide) commercially in Japan for acquired hypothalamic obesity after inclusion on Japan's National Health Insurance drug price list. The launch follows August MHLW approval, the first approved therapy in Japan for acquired HO, supported by the Phase 3 TRANSCEND trial showing a statistically significant 18.8% placebo-adjusted BMI reduction. Rhythm estimates 5,000-8,000 acquired HO patients in Japan, making this a new addressable market in its global rare-disease commercialization strategy.

•19d ago

Rhythm Pharmaceuticals Announces Health Canada Approval of IMCIVREE® (setmelanotide) for Weight Management in Adult and Pediatric Patients 4 years of Age and Older with Acquired Hypothalamic Obesity

Health Canada approved IMCIVREE (setmelanotide) for weight management in adult and pediatric patients 4 years of age and older with acquired hypothalamic obesity, making it the first targeted therapy approved in Canada for the disease. The approval is based on the pivotal Phase 3 TRANSCEND trial in 142 patients, which met its primary endpoint with a statistically significant 18.8% placebo-adjusted reduction in BMI; results were recently published in The New England Journal of Medicine. IMCIVREE was already approved in Canada in 2023 for obesity due to Bardet-Biedl syndrome or genetically confirmed POMC, PCSK1, or LEPR deficiency, so this decision broadens the Canadian label, and Rhythm will work to support patient access across Canada. The acquired-HO indication is already authorized in the US, EU, and UK, so Canada represents an incremental commercial addition rather than a new market for the lead asset.

•1mo ago

Rhythm Pharmaceuticals Announces New Data Presentations at the European Society for Paediatric Endocrinology Meeting

Rhythm Pharmaceuticals announced three posters and three oral presentations at the 64th Annual ESPE Meeting in Marseille covering its work in rare MC4R pathway diseases, centered on setmelanotide (IMCIVREE) in acquired hypothalamic obesity. Real-world data from 30 pediatric patients in France's early-access program showed mean BMI reductions of 11.5% at 6 months and 9.0% at 18 months, with decreases in patient-reported hunger and no new safety concerns. Phase 3 post-hoc analyses showed improvements across multiple cardiometabolic risk indices versus placebo, including a Fatty Liver Index change of -20.6 versus +6.1 and a LAP change of -25.6 versus +3.6. The data support the durability and breadth of the commercial franchise but are conference presentations of an already-approved therapy, making this incremental evidence rather than a new regulatory or trial catalyst.

•1mo ago

Rhythm Pharmaceuticals Announces MHLW Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity (HO) in Japan

Rhythm Pharmaceuticals received marketing authorization from Japan's Ministry of Health, Labour and Welfare (MHLW) for IMCIVREE (setmelanotide) to treat acquired hypothalamic obesity, making it the first therapy approved for this indication in Japan. The approval is supported by data from the Phase 3 TRANSCEND trial, which demonstrated a statistically significant 18.8% placebo-adjusted reduction in body mass index, with a commercial launch expected by the end of 2026 pending final pricing. The company estimates there are approximately 5,000 to 8,000 people living with acquired HO in Japan, representing a new patient population for the MC4R agonist.

•2mo ago

Rhythm Pharmaceuticals Announces Appointment of Manher (AJ) Joshi as Chief Medical Officer

Rhythm Pharmaceuticals appointed Manher “AJ” Joshi, M.D., as Chief Medical Officer effective August 17, bringing over two decades of experience in rare disease development. Dr. Joshi joins from Soleno Therapeutics, where he was Chief Development Officer, and previously held leadership roles at Neurona Therapeutics, Atara Biotherapeutics, and Genzyme. CEO David Meeker highlighted Dr. Joshi’s strategic vision as critical for advancing next-generation MC4R agonists through late-stage development.

•2mo ago

 Rhythm Pharmaceuticals Announces IMCIVREE® (setmelanotide) Receives Expanded Marketing Authorization in the United Kingdom for Treatment of Obesity and Control of Hunger in Patients with Acquired Hypothalamic Obesity due to Hypothalamic Injury or Impairment

Rhythm Pharmaceuticals received expanded marketing authorization in the UK for IMCIVREE to treat acquired hypothalamic obesity in adults and children aged 4 and older. The approval was supported by the Phase 3 TRANSCEND trial, which showed a statistically significant -19.8% placebo-adjusted reduction in BMI at 52 weeks. The MHRA also granted orphan drug designation for acquired HO, and Rhythm plans to work with the NHS to finalize coverage guidance.

•2mo ago

Rhythm Pharmaceuticals Reports Second Quarter 2026 Financial Results and Business Update

Rhythm Pharmaceuticals reported Q2 2026 net product revenue of $71.3 million, up 47% year-over-year and 19% sequentially, driven by a strong U.S. launch of IMCIVREE for acquired hypothalamic obesity with over 400 patient start forms to date. Operational expenses increased significantly year-over-year, leading to a net loss of $50.4 million for the quarter, though the company maintains a strong cash position of $330.9 million expected to fund operations for at least 24 months. Pipeline progress included positive preliminary data for RM-718, showing an 11.6% mean BMI reduction in acquired HO patients, and the publication of Phase 3 TRANSCEND trial results in the New England Journal of Medicine.

•2mo ago

Rhythm Pharmaceuticals Announces Preliminary Data from Phase 2 Trial that Showed RM-718 Demonstrated Positive Efficacy Signal in Acquired Hypothalamic Obesity

Rhythm Pharmaceuticals announced preliminary Phase 2 data for RM-718, showing an 11.6% mean reduction in BMI from baseline at Week 16 in patients with acquired hypothalamic obesity. The efficacy results were consistent with the approved drug setmelanotide and the investigational candidate bivamelagon, observed over similar treatment durations. Safety was generally well tolerated with only mild, localized hyperpigmentation reported, though two patients discontinued treatment due to adverse events.

•2mo ago

Rhythm Pharmaceuticals Announces The New England Journal of Medicine Publication of Phase 3 TRANSCEND Trial Results in Acquired Hypothalamic Obesity

Rhythm Pharmaceuticals announced the publication of Phase 3 TRANSCEND trial results for setmelanotide in The New England Journal of Medicine, demonstrating robust efficacy in patients with acquired hypothalamic obesity. Patients treated with setmelanotide achieved a 19.8% placebo-adjusted reduction in BMI at 52 weeks, with 80% of participants achieving at least 5% BMI reduction compared to placebo. These results underpin the FDA approval granted in March 2026 for IMCIVREE, and the company is currently seeking regulatory approval in Japan.

•3mo ago

Rhythm Pharmaceuticals Announces Multiple New Data Presentations from MC4R Agonists in Acquired Hypothalamic Obesity (HO), Bardet-Biedl Syndrome (BBS) and Prader-Willi Syndrome (PWS) at ENDO 2026

Rhythm Pharmaceuticals presented multiple new data sets at ENDO 2026 demonstrating robust efficacy of its MC4R agonists in rare obesity disorders. Setmelanotide achieved a mean BMI reduction of 18.9% in patients with acquired hypothalamic obesity over 2.5 years, while oral bivamelagon showed BMI reductions ranging from 6.7% to 16.6% at one year. Real-world data for Bardet-Biedl syndrome showed 62% of adults achieved ≥10% weight loss, alongside significant reductions in healthcare resource utilization and hyperphagia symptoms.

•4mo ago

 Rhythm Pharmaceuticals Presents Positive Interim Six-month Data from Phase 2 Trial of Setmelanotide in Patients with Prader-Willi Syndrome (PWS) at ENDO 2026

Rhythm Pharmaceuticals presented positive interim six-month data from its Phase 2 trial of setmelanotide in patients with Prader-Willi Syndrome (PWS) at ENDO 2026. The 17 patients on active therapy achieved a mean BMI reduction of 3.06%, with significant reductions in fat mass (-4.19%) and preservation of lean mass (+0.74%). Clinically meaningful improvements were also seen in hyperphagia scores and anxiety measures. These results reinforce the rationale for advancing setmelanotide into Phase 3 development for PWS, a rare genetic disorder affecting an estimated 12,500 to 16,000 patients in the US with limited effective treatment options.

•4mo ago

Rhythm Pharmaceuticals Announces Seven Abstracts Accepted for Presentation at ENDO 2026

Rhythm Pharmaceuticals announced that seven abstracts, including four late-breaking presentations, were accepted for ENDO 2026, the Endocrine Society’s annual meeting in Chicago. The presentations cover Phase 2 and Phase 3 data for setmelanotide in Prader-Willi syndrome and acquired hypothalamic obesity, as well as one-year weight reduction data for the oral pipeline asset bivamelagon. Additionally, Dr. Ashley Shoemaker from Vanderbilt Health will join Rhythm as a medical director effective June 1, 2026, following her presentation at the conference.

•5mo ago

Rhythm Pharmaceuticals Announces Publication of New Bardet-Biedl Syndrome Diagnostic Algorithm in the American Journal of Medical Genetics

Rhythm Pharmaceuticals announced the publication of a new, evidence-based diagnostic algorithm for Bardet-Biedl syndrome (BBS) in the American Journal of Medical Genetics. The algorithm, co-authored by Rhythm-funded experts including Professor Philip Beales, aims to streamline diagnosis and reduce the time to informed care for BBS patients. This supports the addressable market for Rhythm’s approved drug IMCIVREE (setmelanotide), which is indicated for obesity associated with BBS. The updated criteria will be presented at Rhythm’s inaugural MOMENTUM MC4R Pathway Summit in Chicago on June 12.

•5mo ago

Rhythm Pharmaceuticals Announces New Data Presentations in Acquired Hypothalamic Obesity at Pediatric Endocrine Society

Rhythm Pharmaceuticals presented positive long-term data at the Pediatric Endocrine Society meeting showing pediatric patients treated with setmelanotide sustained BMI reductions of 16.4% over 2.5 years. Separate Phase 3 TRANSCEND trial data showed 71.1% of pediatric patients improved by at least one weight category after one year of treatment, compared to 13.6% for placebo. The safety profile remained consistent with prior reports, supporting the long-term use of IMCIVREE in rare neuroendocrine diseases.

•5mo ago