Setmelanotide Shows 16.4% BMI Cut and 71.1% Weight-Category Gains in Pediatric Trial

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Ten pediatric patients with acquired hypothalamic obesity on setmelanotide for 2.5 years achieved BMI reductions of 16.4%, a 1.6-point drop in BMI Z-score and a 34.2-point decline in %BMI95. In Phase 3 TRANSCEND, 71.1% improved at least one weight category after one year and 44.4% reached healthy weight or overweight.

1. Long-Term Efficacy Data

In a cohort of 10 pediatric patients with acquired hypothalamic obesity, 2.5 years of setmelanotide therapy produced mean reductions of 16.4% in BMI, a 1.6-point decrease in BMI Z-score and a 34.2-point drop in %BMI95, demonstrating sustained weight-loss effects beyond the initial 16-week trial.

2. Phase 3 TRANSCEND Weight Category Improvements

Among pediatric participants in the TRANSCEND trial, 59.7% began with class II or III obesity; after one year of setmelanotide, 71.1% improved by at least one weight category and 44.4% by two categories, yielding 44.4% at healthy or overweight status versus 13.6% and 0% respectively in placebo.

3. Safety Profile Observations

Adverse events over both studies aligned with the known safety profile of setmelanotide, with skin hyperpigmentation, nausea, vomiting, headache and injection site reactions reported most frequently and no new safety signals observed.

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