Rhythm Pharmaceuticals reported positive preliminary results from the exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).
The trial showed reductions in BMI and hyperphagia in patients with PWS after 3 and 6 months of treatment with setmelanotide.
Of the 18 enrolled patients, 17 remain on therapy, supporting the advancement of setmelanotide into a Phase 3 trial.
Therapeutic Benefit Demonstrated
Setmelanotide therapy showed potential therapeutic benefits with reductions in BMI and hyperphagia in patients with PWS at Month 3 and Month 6.
Promising Results
Preliminary results indicated that the majority of patients achieved reductions in BMI and improvements in HQ-CT scores with consistent safety and tolerability.
Future Trials Planned
Rhythm Pharmaceuticals plans to initiate a Phase 3 registrational trial for setmelanotide in PWS and has started a Phase 1 trial for MC4R agonist RM-718 in PWS patients.
- The positive results from the Phase 2 trial suggest a potential treatment option for addressing the challenges faced by PWS patients, including hyperphagia and severe obesity.
- Setmelanotide's efficacy in reducing BMI and hyperphagia, along with the high retention rate of patients on therapy, provide optimism for the upcoming Phase 3 trial and future treatment options.
Rhythm Pharmaceuticals' successful Phase 2 trial results pave the way for further development of setmelanotide as a potential therapy for Prader-Willi syndrome, addressing the critical needs of patients with this rare neuroendocrine disorder.