Clinical TrialJun 13, 2026, 08:02 AM
Rhythm Pharma Setmelanotide Phase 2 PWS Data: BMI, Hyperphagia Improved
AI Summary
Rhythm Pharmaceuticals announced positive preliminary Phase 2 trial data for setmelanotide in patients with Prader-Willi syndrome (PWS). The 52-week trial, with 17 patients remaining on active therapy, showed clinically meaningful reductions in BMI, fat mass, and improvements in hyperphagia and anxiety measures at the six-month analysis. These results reinforce the rationale for advancing setmelanotide into Phase 3 development for PWS.
Key Highlights
- Setmelanotide achieved a -3.06% mean reduction in BMI across 17 patients with PWS at Month 6.
- Patients showed a +0.74% mean gain in lean mass and -4.19% mean loss in fat mass across 16 patients.
- 8 of 10 patients with moderate to severe hyperphagia achieved a clinically meaningful ">=7-point" reduction in HQ-CT score.
- 10 of 15 patients with baseline PADQ score >11 achieved a clinically meaningful ">=11-point" improvement.
- 5 of 7 pediatric patients achieved a clinically meaningful BMI z-score reduction >0.2.
- Safety and tolerability results were consistent with setmelanotide's established profile.
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