Core Insights - Rhythm Pharmaceuticals Inc. has reported positive preliminary results from its exploratory Phase 2 trial of setmelanotide for patients with Prader-Willi syndrome (PWS) [1][3] - The company plans to advance setmelanotide into a Phase 3 registrational trial, contingent on the successful completion of the current Phase 2 trial [3] Trial Details - The Phase 2 trial enrolled 18 patients and is ongoing, with a focus on assessing the effects of setmelanotide on BMI and hyperphagia [2][6] - Preliminary results indicate that 6 of 8 patients achieved BMI reductions at Month 3, and 3 of 5 patients achieved reductions at Month 6, with some showing deeper reductions [6] Safety and Tolerability - Safety and tolerability results for setmelanotide have been consistent with its established clinical profile, with 17 of the 18 enrolled patients remaining on active therapy [6] Stock Performance - Following the announcement of the trial results, Rhythm Pharmaceuticals' stock increased by 14.24%, closing at $119.69 [5] Additional Developments - Rhythm has initiated a Part D arm in the Phase 1 trial of MC4R agonist RM-718, planning to enroll up to 20 patients, with the first patient expected to be screened in December 2025 [4]
Rhythm Pharma Strikes Chord As Early PWS Trial Results Show Promise