Rhythm Pharmaceuticals Announces Major Breakthrough in Phase 3 Trial for Rare Form of Obesity

$RYTM

Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company focused on rare neuroendocrine diseases, has shared exciting news from its Phase 3 TRANSCEND trial. The trial evaluated the effectiveness of setmelanotide, a melanocortin-4 receptor (MC4R) agonist, in treating acquired hypothalamic obesity—a rare and challenging condition. The results are promising, with the study meeting its primary goal.

The trial was global, double-blind, and placebo-controlled, involving 120 participants over 52 weeks. Patients treated with setmelanotide saw a 16.5% reduction in Body Mass Index (BMI), while those in the placebo group experienced a 3.3% increase. This translates to a placebo-adjusted BMI reduction of nearly 20%, a significant outcome.

Beyond weight loss, the study also showed improvements in related symptoms like daily hunger and hyperphagia, the latter being a persistent, intense hunger that often leads to overeating. Notably, 83% of participants—both adults and those under 18—achieved either a 5% or greater reduction in BMI or a meaningful improvement in BMI Z-score.

Setmelanotide was generally well-tolerated. The most common side effects were mild to moderate, including nausea, vomiting, and injection site reactions, consistent with its known safety profile from previous trials. Rhythm Pharmaceuticals plans to submit regulatory applications for approval of setmelanotide in the U.S. and Europe by Q3 2025. If approved, it would become the first approved treatment specifically for acquired hypothalamic obesity.

These results also support the development of Rhythm’s next-generation MC4R agonists, which are currently in early-phase clinical trials. The company remains focused on advancing innovative treatments for people living with rare neuroendocrine conditions It plans to present the full TRANSCEND trial results at an upcoming medical conference, aiming to contribute valuable insights to the medical community and improve treatment options for patients with this rare disease.

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