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Investing.com -- Shares of Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) rose 9% following the announcement of positive results from its Phase 3 TRANSCEND trial, which met its primary endpoint with a significant reduction in body mass index (BMI) for patients with acquired hypothalamic obesity. The study demonstrated a -19.8% placebo-adjusted BMI reduction in patients treated with setmelanotide, the company’s investigational therapy.
The Boston-based biopharmaceutical company reported that patients on setmelanotide therapy achieved a mean BMI change of -16.5% compared with a +3.3% change for those on placebo over a 52-week period. These results were particularly notable in both adult and pediatric patient groups, with -19.2% and -20.2% placebo-adjusted BMI reductions, respectively.
The data, deemed highly clinically meaningful, offer hope for a new targeted therapy for both adults and children living with this serious disease. Acquired hypothalamic obesity typically follows damage to the hypothalamus from brain tumors, their treatment, or other injuries, leading to rapid weight gain and excessive hunger.
Stifel analyst Paul Matteis commented on the trial’s outcome, stating, "we believe efficacy here (~20% placebo-adjusted BMI reduction) is outstanding in a highly difficult-to-treat patient population." Matteis reiterated a Buy rating for Rhythm Pharmaceuticals, highlighting the potential for the drug to be a blockbuster opportunity with a fast ramp, according to Key Opinion Leaders (KOLs).
The TRANSCEND trial is said to be the largest and longest placebo-controlled study evaluating a therapy for patients with this condition. Rhythm Pharmaceuticals plans to submit a supplemental New Drug Application to the FDA and a Type II variation request to the European Medicines Agency in the third quarter of 2025, aiming to make setmelanotide the first approved therapy for patients with acquired hypothalamic obesity.
No new safety signals were observed during the trial, and setmelanotide was generally well tolerated. The most common treatment-emergent adverse events included nausea, vomiting, diarrhea, and injection site reactions.
The company is preparing to present the full data from the TRANSCEND study at an upcoming medical meeting and anticipates the completion of an additional cohort of Japanese patients with topline data expected in the first quarter of 2026.
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