Rhythm Pharmaceuticals, Inc. (RYTM) announced positive results from its global Phase 3 TRANSCEND trial of Setmelanotide in patients with acquired hypothalamic obesity (HO).
At 52 weeks, the study demonstrated an 18.8% placebo-adjusted difference in BMI reduction across all 142 patients, including those from a Japanese cohort and supplemental participants.
Acquired hypothalamic obesity is a rare condition caused by injury to the hypothalamus, often following brain tumors, trauma, or stroke. It leads to accelerated weight gain, excessive hunger, and reduced energy expenditure, severely impacting quality of life.
In the trial, patients receiving Setmelanotide achieved a mean BMI reduction of 16.4% from baseline, compared with a +2.4% BMI increase in the placebo group. Among patients aged 12 and older, Setmelanotide also produced greater improvements in hunger scores. The therapy was generally well tolerated, with a safety profile consistent with earlier studies.
Rhythm has submitted a supplemental New Drug Application (sNDA) to the U.S. FDA, with a PDUFA goal date of March 20, 2026. The company is also pursuing regulatory reviews in Europe and Japan, with potential approvals expected later this year.
Setmelanotide, marketed as IMCIVREE, is already approved for certain rare genetic forms of obesity, including Bardet-Biedl syndrome (BBS) and POMC, PCSK1, or LEPR deficiencies. The TRANSCEND trial represents a major step toward expanding its use to patients with acquired hypothalamic obesity.
RYTM has traded between $45.90 and $122.20 over the past year. The stock closed Friday's session at $92.73, down 5.45%. In overnight trading Friday the stock rose 0.08% at $92.66.
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