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 announce topline results from the Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity on April 7, 2025 [1][2]. Group 1: Company Overview - Rhythm Pharmaceuticals is a global commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [1][15]. - The company's lead asset, setmelanotide, is an MC4R agonist approved for reducing excess body weight in patients with specific genetic obesity syndromes [15]. Group 2: Phase 3 TRANSCEND Trial - The TRANSCEND trial is a global, randomized, double-blind, placebo-controlled study involving 120 patients aged 4 years and older [3]. - The primary endpoint of the trial is the mean percent change in body mass index (BMI) after 52 weeks of treatment [3]. - Secondary endpoints include assessments of daily hunger, hyperphagia, weight, quality of life, safety, and tolerability [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]. - This condition often follows the treatment of specific brain tumors and can lead to severe obesity within 6 to 12 months post-injury [4]. Group 4: Setmelanotide Indications - In the U.S., setmelanotide is indicated for patients aged 2 years and older with syndromic or monogenic obesity due to specific genetic deficiencies [5]. - In the EU and UK, it is indicated for treating obesity and controlling hunger associated with genetically confirmed conditions in adults and children aged 2 years and above [6].