Rhythm Pharmaceuticals, Inc. has released additional positive data from its global phase 3 trial of Setmelanotide in patients with acquired hypothalamic obesity (HO). The new data set includes 12 patients from a Japanese cohort and 10 supplemental patients who were enrolled in addition to the primary 120-patient pivotal cohort.
The key highlights from the 52-week data include an -18.8% placebo-adjusted difference in BMI reduction for all patients, with a primary endpoint of mean BMI reduction of -16.4% from baseline for all patients on Setmelanotide therapy compared with a +2.4% BMI change for patients on placebo at 52 weeks. This data suggests a substantial improvement in BMI reduction for patients on Setmelanotide therapy compared to those on placebo.
Among patients aged 12 and older, the Setmelanotide group showed an average weekly reduction of 2.5 points in the weekly average most hunger score compared with a 1.3-point reduction in the placebo group.
These results further support Setmelanotide's potential to become the first therapy approved for patients living with the symptoms of acquired HO, including hunger, reduced energy expenditure, accelerated weight gain, and obesity.
Rhythm Pharmaceuticals is expecting a PDUFA goal date of March 20, 2026, for the supplemental new drug application (SNDA) for Setmelanotide in acquired HO. The company is preparing to bring Setmelanotide to patients pending U.S. Food and Drug Administration (FDA) approval.
The European Medicines Agency (EMA) is also reviewing Rhythm's submission for marketing authorization of Setmelanotide for the same indication. The company anticipates potential marketing authorization in the second half of 2026 in the European Union and the United Kingdom.
Acquired HO is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus. Rhythm estimates that there are approximately 10,000 patients living with acquired HO in the United States, 10,000 patients in Europe, and between 5,000 to 8,000 people living with acquired HO in Japan.
Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases. Setmelanotide is Rhythm's lead asset, approved by the FDA to reduce excess body weight and maintain long-term weight reduction in patients with syndromic or monogenic obesity.
As a result of these announcements, the company's shares have moved -4.18% on the market, and are now trading at a price of $101.63. Check out the company's full 8-K submission here.
