Core Insights - Rhythm Pharmaceuticals is set to disclose topline results from a Phase 2 trial evaluating bivamelagon for acquired hypothalamic obesity on July 9, 2025 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [4] - The company's lead asset, IMCIVREE® (setmelanotide), is approved for treating obesity related to specific genetic conditions [4][5] - Rhythm is advancing a clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718 [4] Bivamelagon Phase 2 Trial Details - The Phase 2 trial is a randomized, placebo-controlled, double-blind study involving 28 patients aged 12 and older [3] - Patients received daily oral doses of bivamelagon (200 mg, 400 mg, or 600 mg) or placebo for 14 weeks [3] - The primary endpoint is the change in body mass index after 14 weeks of treatment, with an option for patients to continue therapy for up to 52 weeks [3]

Core Insights - Rhythm Pharmaceuticals, Inc. announced the acceptance of three late-breaking abstracts for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) scheduled for July 12-15 in San Francisco, CA [1] Group 1: Clinical Trials and Presentations - Dr. Susan Phillips will present data from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity, marking it as the largest randomized, placebo-controlled trial in this area to date [2] - A poster presentation will be given by Dr. Christian Roth detailing exit interviews from 30 patients or caregivers in the US who participated in the Phase 3 TRANSCEND trial [2] - Dr. Vidhu Thaker will present results from a 14-week Phase 2 trial of bivamelagon, an oral MC4R agonist, in participants with acquired hypothalamic obesity [2] Group 2: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, IMCIVREE® (setmelanotide), approved by the FDA for treating obesity related to specific genetic conditions [3][4] - Setmelanotide is indicated for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiencies [4][5][6] - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718, as well as a preclinical suite for congenital hyperinsulinism [3]

Core Insights - Rhythm Pharmaceuticals presented new real-world data demonstrating that setmelanotide leads to significant weight reduction in patients with acquired or congenital hypothalamic obesity [1][3][10] Group 1: Clinical Data Presentation - New data was presented at the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology, and the 32nd annual European Congress on Obesity, showcasing the effectiveness of setmelanotide [1][2] - The presentations included data from 35 patients treated with setmelanotide for up to nine months, showing consistent improvements in body mass index (BMI), BMI-z, and hunger scores [1][4] Group 2: Patient Outcomes - Among adult patients with acquired hypothalamic obesity, there was a mean BMI reduction of -11.9% at three months, -19.2% at six months, and -23.0% at nine months [5] - Pediatric patients also showed a decrease in BMI z-scores, with a mean reduction of 0.3 at three months and 0.4 at both six and nine months [5][11] Group 3: Safety Profile - Setmelanotide was generally well tolerated, with common adverse events including injection site reactions and skin hyperpigmentation, consistent with its established safety profile [6][10] Group 4: Future Developments - Rhythm Pharmaceuticals plans to submit a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency for setmelanotide in the third quarter of 2025 [10]