(RTTNews) - Rhythm Pharmaceuticals Inc. (RYTM), a commercial-stage biopharmaceutical company, on Sunday reported encouraging preliminary six-month results from a Phase 2 trial for Setmelanotide in treating Prader-Willi syndrome. The data was resented at the Endocrine Society's Annual Meeting (ENDO 2026).
Prader-Willi syndrome (PWS) is a rare genetic disorder that leads to certain mental and behavioral problems, primarily causing a constant sense of hunger. There are an estimated 12,500-16,000 patients of PWS living in the U.S. and Europe, with about 80%-90% facing hyperphagia or obesity. Setmelanotide is a melanocortin-4 receptor agonist used to treat excess body weight and hypothalamic obesity, that received FDA approval in 2020 for the same. The drug is also approved for use in the European Union and the UK.
The ongoing Phase 2 trial for setmelanotide enrolled 18 patients with PWS, with a treatment period of 52 weeks.
Pediatric and adult patients showed a 3.60% mean reduction in body-mass index (BMI). Patients also showed a 0.35 mean reduction in BMI z-score from the baseline. Additionally, patients on treatment demonstrated a 0.74 mean gain in lean mass, and a 4.19 mean loss in fat mass, measured by a DEXA scan to determine bone mineral density. Hyperphagia was reduced in patients assessed by a Hyperphagia Questionnaire for Clinical Trials (HQ-CT), yielding a decrease of 7 or more points in moderate to severe hyperphagia. A decrease in anxiety was also observed in treated patients, determined by a PWS Anxiousness and Distress Behaviors Questionnaire (PADQ). The company stated that the strength of the Phase 2 results would support advancement into an upcoming Phase 3 trial for setmelanotide in PWS.
RYTM closed Friday at $88.38, up 0.82%. In the pre-market, shares are trading at $90.86, up 2.81%.
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