Rhythm(US:RYTM) Globenewswire·2025-12-11 12:30Core Insights - Rhythm Pharmaceuticals announced positive preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS), showing reductions in BMI and hyperphagia [1][4] - The company plans to advance setmelanotide into a Phase 3 registrational trial, pending the successful completion of the Phase 2 trial [2] - Rhythm has initiated a Part D arm in the Phase 1 trial of MC4R agonist RM-718, which will enroll up to 20 patients with PWS [2] Company Developments - Six out of eight patients who reached Month 3 of setmelanotide therapy achieved BMI reductions from baseline, and three out of five patients who reached Month 6 also saw reductions [6] - Six out of seven evaluable patients at Month 3 achieved meaningful reductions in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) scores [6] - Seventeen out of the 18 enrolled patients remain on active setmelanotide therapy, with safety and tolerability results consistent with the drug's established clinical profile [6] Clinical Trial Details - The Phase 2 trial is a single-site, open-label study evaluating setmelanotide for the treatment of PWS, originally designed for 26 weeks but extended to 52 weeks [8] - Eighteen patients aged 6 to 65 years with PWS and obesity were enrolled, with a primary focus on safety and tolerability, and key secondary endpoints assessing weight, hyperphagia, behavior, and pharmacokinetics [9] - The trial aims to address the profound unmet need in the PWS patient population, where hyperphagia and severe obesity present significant health challenges [3] Future Outlook - The company anticipates additional data in the first half of 2026 and remains committed to exploring the potential of MC4R agonism for patients with limited treatment options [4] - The first patient for the RM-718 study is expected to be screened in December 2025 [2][4]