Rhythm Pharmaceuticals to Announce Topline Results from Pivotal Phase 3 TRANSCEND Trial Evaluating Setmelanotide in Patients with Acquired Hypothalamic Obesity

Core Viewpoint - Rhythm Pharmaceuticals is set to disclose topline results from the Pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity during a conference call on April 7, 2025 [1][2] Group 1: Conference Call Details - The conference call will take place at 8:00 a.m. ET on April 7, 2025, and will be accessible via registration and webcast [2] - An archived version of the webcast will be available on the company's website for at least 30 days following the call [2] Group 2: Phase 3 TRANSCEND Trial Overview - The TRANSCEND trial is a global, randomized, double-blind, placebo-controlled study involving 120 patients aged 4 years and older [3] - Participants were randomized in a 2:1 ratio to receive either setmelanotide or a placebo, with the primary endpoint being the mean percent change in body mass index (BMI) after 52 weeks [3] - Secondary endpoints include assessments of daily hunger, hyperphagia, weight, quality of life, safety, and tolerability [3] - A supplemental cohort of 12 Japanese patients is ongoing, with topline data expected in Q1 2026 [3] Group 3: Acquired Hypothalamic Obesity - Acquired hypothalamic obesity is a rare obesity form resulting from damage to the hypothalamus, affecting hunger and weight regulation [4] - Common causes include brain tumors, traumatic brain injury, and stroke, leading to rapid weight gain and hyperphagia [4] Group 4: Setmelanotide Indications - In the U.S., setmelanotide is approved for reducing excess body weight in patients aged 2 years and older with specific genetic obesity conditions [5] - In the EU and UK, it is indicated for treating obesity and controlling hunger in genetically confirmed cases of Bardet-Biedl syndrome or related deficiencies [6] Group 5: Limitations and Warnings - Setmelanotide is not indicated for patients with certain conditions, including benign variants of POMC, PCSK1, or LEPR deficiencies [7] - Warnings include potential serious adverse reactions such as hypersensitivity, sexual arousal disturbances, and depression [9][10][11]