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Rhythm Pharmaceuticals Announces Preliminary Data from Exploratory Phase 2 Trial that showed Setmelanotide Demonstrated Positive Efficacy Signal in Prader-Willi Syndrome
-- BMI and hyperphagia reductions have been observed in patients with PWS treated with setmelanotide at Month 3 (n=8) and Month 6 (n=5); 17 of 18 enrolled
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- BMI and hyperphagia reductions have been observed in patients with PWS treated with setmelanotide at Month 3 (n=8) and Month 6 (n=5); 17 of 18 enrolled patients remain on therapy -- -- Promising results supportive of Phase 3, registrational trial of setmelanotide in PWS -- -- Company initiated Phase 1, Part D study to evaluate weekly MC4R agonist RM-718 in patients with PWS -- -- Company to hold conference call on Thursday, December 11 at 8:00 a.m. -- BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced positive preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS). The Company also announced plans to advance setmelanotide into a Phase 3 registrational trial in PWS, pending successful completion of this Phase 2 trial. Rhythm also announced that it has initiated a Part D arm in the Phase 1 trial of MC4R agonist RM-718 that will enroll up to 20 patients with PWS. Rhythm anticipates screening the first patient for this 26-week open-label trial of RM-718 in December 2025. “There remains a profound unmet need in the PWS patient population,” said Jennifer Miller, M.D., University of Florida Division of Endocrinology, Department of Pediatrics in the College of Medicine, the principal investigator for this Phase 2 trial. “Hyperphagia and severe obesity associated with PWS present serious challenges for patients and often lead to significant health complications over time. These interim data offer meaningful insight into the potential for a future treatment option that could help address the unique and critical needs of patients living with PWS.” Rhythm enrolled 18 patients with PWS aged 6-65 years old with a BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients younger than 18. The 52-week trial remains ongoing. Setmelanotide therapy demonstrated potential therapeutic benefit with BMI and hyperphagia reductions in patients with PWS at Month 3 (n=8) and Month 6 (n=5); Highlights from preliminary results, as of a cut-off date of Nov. 14, include: Six (6) of 8 patients who reached Month 3 of setmelanotide therapy achieved BMI reductions from base...