Rhythm Reports Expanded Phase 3 TRANSCEND Data Showing Significant BMI And Hunger Reduction With Setmelanotide

Rhythm Pharmaceuticals, Inc. has reported additional positive 52-week data from its global Phase 3 TRANSCEND trial evaluating setmelanotide in patients with acquired hypothalamic obesity (HO). The expanded dataset includes 12 patients from a Japanese cohort and 10 supplemental participants enrolled beyond the original 120-patient pivotal group, bringing the total analysed population to 142 patients.

Results showed an 18.8% placebo-adjusted difference in body mass index (BMI) reduction. Patients receiving setmelanotide achieved a mean BMI reduction of 16.4% from baseline (n=94), compared with a 2.4% increase in BMI among placebo-treated patients (n=48), and the primary endpoint reached statistical significance (p<0.0001). Among patients aged 12 years and older, those treated with setmelanotide experienced an average weekly reduction of 2.5 points in hunger scores, compared with 1.3 points in the placebo group (p=0.0015).

“Building off our strong pivotal data, these efficacy data further support setmelanotide’s potential to become the first therapy approved for patients living with the hunger, reduced energy expenditure, accelerated weight gain, and obesity of acquired hypothalamic obesity,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “We look forward to continuing our positive dialogue with regulators, and we are well prepared to bring setmelanotide to patients with acquired HO, pending U.S. Food and Drug Administration (FDA) approval."

Rhythm previously announced that TRANSCEND met its primary and key secondary endpoints based on topline data released in April 2025. The company’s supplemental New Drug Application (sNDA) is currently under review by the U.S. Food and Drug Administration, with a PDUFA target action date of March 20, 2026. Rhythm plans to submit the final supplemental data package ahead of the agreed deadline on March 2, 2026.

Regulatory review is also ongoing in Europe and Japan. The European Medicines Agency is assessing a Type II variation to the Marketing Authorisation Application for the same indication, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected in the second quarter of 2026 and potential European Commission approval later in the year. Additionally, Rhythm intends to submit the full data package to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) to seek marketing authorisation for acquired hypothalamic obesity in that market.