RYTM
Published on 06/25/2025 at 14:20
Rhythm Pharmaceuticals, Inc. announced that three late-breaking abstracts have been accepted for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) taking place July 12-15 in San Francisco, CA. In a live oral presentation, Susan Phillips, M.D., Pediatric Endocrinology, University of California San Diego/Rady Children's Hospital, San Diego, will present data from Rhythm's pivotal Phase 3 TRANSCEND trial evaluating setmelanotide in the largest randomized, placebo-controlled trial in acquired hypothalamic obesity to date. Efficacy and Safety of Setmelanotide in Acquired Hypothalamic obesity: Results from a Double-Blind, Multicenter, Placebo-Controlled, Randomized Phase 3 Trial.
In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign. Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity.