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

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-- Setmelanotide therapy achieved robust, sustained and clinically significant weight loss in patients with acquired HO at 2.5 years -- - - Bivamelagon therapy achieved progressive reductions in BMI and hunger measures in patients (N=26) with acquired HO at one year -- -- Real-world data showed patients with BBS treated with setmelanotide (N=286) achieved improvements in weight-related outcomes, reduction in healthcare resource utilization -- -- Additional presentations highlight analyses of setmelanotide therapy in patients with acquired HO who achieved weight category improvements; BMI reductions after prior bariatric surgery in acquired HO patients treated with setmelanotide; and positive data following 6 months of treatment in patients with PWS -- BOSTON, June 15, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (NASDAQ: RYTM ), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced new data from multiple presentations during the Endocrine Society's Annual Meeting (ENDO 2026) in Chicago. "Rare MC4R pathway diseases such as acquired hypothalamic obesity (HO), Bardet-Biedl syndrome (BBS) and Prader-Willi syndrome (PWS) are severe, chronic diseases with limited or no effective treatment options," said Rhythm Chairman, CEO and President David Meeker, MD. "We are excited to share positive results across these diseases showing MC4R agonism continues to demonstrate its potential to meaningfully reduce hyperphagia and improve weight-related outcomes in these patients. ENDO provides an important platform to engage with the global endocrinology community, and we are focused on continuing to translate this science into treatments for patients with significant unmet need." Long-Term Efficacy with Setmelanotide in Patients with Acquired Hypothalamic Obesity Presented as an oral presentation by Christian Roth, M.D., Seattle Children's Research Institute, this analysis evaluated long-term efficacy and safety of setmelanotide therapy in patients with acquired HO for up to 2.5 years of treatment in a Phase 2 study and long-term extension. Key findings include: -18.9% mean BMI reduction across all participants (n=11); Mean change from baseline in BMI z-score was -1.60 The most common adverse events were nausea, skin hyperpigmentation, upper respiratory tract infection, and vomiting. Weight Reduction After 1 Year of Oral Bivamelagon in Acquired Hypothalamic Obesity Presented as a poster by Dr. Vidhu Thaker, M.D., Pediatric Endocrinology, Columbia University, this analysis evaluated 1-year efficacy and safety results from a Phase 2 study of oral bivamelagon in patients with acquired HO, including 14 weeks of double-blind treatment followed by a 38-week open-label extension. Twenty-six of 28 patients who originally enrolled in this Phase 2 trial remained on therapy in the open-label extension and reached at least 52 weeks on therapy. The mean change in BMI from baseline to Week 52 for patients (n=26) was: −8.7% for patients who transitioned from placebo to 600 mg (n=7); −6.7% for patients who received 200 mg then 600 mg (n=6); −10.8% for patients who received 400 mg then 600 mg (n=6); −16.6% for patients who received 600 mg throughout (n=7); The mean change in the weekly average of the maximal daily hunger score ranged from −1.9 to −4.8 across cohorts; In pediatric patients (n=13), mean change in BMI z-score from baseline to Week 52 ranged from −0.22 to −0.69 across treatment groups; and Common adverse events reported were vomiting, nausea, diarrhea, and headache. Setmelanotide Treatment in Patients with Acquired Hypothalamic Obesity and Previous Weight Loss Surgery Presented as a poster by Ashley Shoemaker, M.D., MSCI, Senior Medical Director at Rhythm Pharmaceuticals, this analysis evaluated outcomes in patients with acquired HO who had a pre-trial history of bariatric surgery and were treated with setmelanotide or placebo in a Phase 3 trial. Key findings in this post-hoc analysis of patients who tried bariatric surgery and subsequently enrolled in a trial of setmelanotide therapy and completed the trial include: Patients treated with setmelanotide (n=3) achieved reductions in BMI at 1 year, with changes ranging from −9.6% to −37.9%, compared with a 4.8% increase in the placebo-treated patient (n=1); Patients had a history of multiple bariatric procedures, including gastric sleeve and gastric bypass, with persistent obesity prior to study entry despite prior surgical intervention; and Treatment-related adverse events were reported in one participant (upper abdominal pain, constipation, nausea, and headache) who received setmelanotide. Weight Category Improvement Following Setmelanotide in Patients with Acquired Hypothalamic Obesity Presented as a poster by Ashley Shoemaker, M.D., MSCI, Senior Medical Director at Rhythm Pharmaceuticals, this analysis evaluated changes in weight category after 1 year of treatment with setmelanotide in patients with acquired hypothalamic obesity from a Phase 3 trial. Key findings include: Weight ... Full story available on Benzinga.com