- New data shows setmelanotide leads to significant weight reduction in hypothalamic obesity patients.
- Rhythm Pharmaceuticals (RYTM, Financial) plans regulatory applications for acquired hypothalamic obesity by Q3 2025.
- Setmelanotide demonstrated a consistent safety profile with manageable side effects.
Rhythm Pharmaceuticals (RYTM) presented compelling real-world data demonstrating the effectiveness of setmelanotide in treating hypothalamic obesity. The data, presented at major European endocrinology conferences, involved 35 patients who participated in French early-access programs. Adult patients with acquired hypothalamic obesity (n=20) achieved a remarkable -23.0% mean BMI change at nine months, while pediatric patients displayed clinically meaningful reductions in BMI z-scores.
The study showed that setmelanotide was generally well-tolerated. The most common adverse effects were injection site reactions and skin hyperpigmentation, which align with the drug's established safety profile. Importantly, no new safety concerns were identified.
Building on the success of its pivotal Phase 3 trial, which showed a -19.8% placebo-adjusted BMI reduction, Rhythm Pharmaceuticals aims to submit regulatory applications in the third quarter of 2025 for setmelanotide's use in treating acquired hypothalamic obesity. The company is also progressing with its Phase 3 congenital hypothalamic obesity substudy, with enrollment expected to complete in the latter half of 2025.
These findings reinforce setmelanotide's potential as a viable treatment option for patients with significant unmet needs in managing hypothalamic obesity, a condition characterized by severe weight gain and intense hunger, underscoring the importance of Rhythm's strategy to expand its indications.
