Rhythm Pharmaceuticals Presents Positive Interim Six-month Data from Phase 2 Trial of Setmelanotide in Patients with Prader-Willi Syndrome (PWS) at ENDO 2026
Rhythm Pharmaceuticals presented positive interim six-month data from its Phase 2 trial of setmelanotide in patients with Prader-Willi Syndrome (PWS) at ENDO 2026. The 17 patients on active therapy achieved a mean BMI reduction of 3.06%, with significant reductions in fat mass (-4.19%) and preservation of lean mass (+0.74%). Clinically meaningful improvements were also seen in hyperphagia scores and anxiety measures.
Key figures
- Drug Name
- setmelanotide
- Phase Of Trial
- Phase 2
- Patient count
- 17
- Mean bmi reduction pct
- -3.06
- Mean fat mass loss pct
- -4.19
- Adult bmi reduction pct
- -3.11
- Mean lean mass gain pct
- 0.74
- Pws us patients estimated
- 12,500-16,000
- Pediatric bmi reduction pct
- -3
- Pws global patients estimated
- 400000
AI analysis
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