Breakthrough Obesity Drug Achieves Phase 3 Success in Treating Hypothalamic Obesity” ###

Context and Indication:
Rhythm Pharmaceuticals' experimental drug, setmelanotide (brand name Imcivree), demonstrated success in a Phase 3 trial targeting acquired hypothalamic obesity, a rare disorder caused by damage to the hypothalamus, often from brain tumors or their treatment1 2 4.

Trial Results:

The 52-week TRANSCEND trial reduced body mass index (BMI) by an average of 16.5% in patients using setmelanotide, compared to a 3.3% BMI increase in the placebo group, resulting in a 19.8% placebo-adjusted reduction4 9.
80% of patients on the drug achieved a BMI reduction of at least 5%4 9.
Both adults and pediatric patients showed significant improvements, with mean placebo-adjusted BMI reductions of 19.2% in adults and 20.2% in pediatric participants4 10.

Safety Profile:

Setmelanotide was well-tolerated with no new safety signals observed.
Common side effects included nausea, vomiting, diarrhea, injection site reactions, and headache9.

Market Potential:

Currently, setmelanotide is FDA-approved for treating genetic obesity in certain conditions, and this trial supports its potential label expansion to hypothalamic obesity.
Approximately 5,000-10,000 individuals in the United States may suffer from acquired hypothalamic obesity, representing a significant unmet medical need2 9.

Future Developments:

Regulatory submissions to the FDA and European Medicines Agency (EMA) are planned for the third quarter of 2025 to expand setmelanotide's approved indications4 9.

Implications:
This breakthrough positions Rhythm Pharmaceuticals as a pioneer in addressing rare obesity disorders, combining robust clinical efficacy with potential to fulfill an underserved medical need in both adult and pediatric populations9 10.