Rhythm Pharmaceuticals, Inc. develops and commercializes therapies for rare neuroendocrine diseases, centered on IMCIVREE (setmelanotide), an MC4R agonist for hyperphagia and severe obesity linked to rare MC4R pathway disorders. Company updates commonly cover U.S. and international commercialization of setmelanotide, regulatory authorizations and label expansions for acquired hypothalamic obesity, Bardet-Biedl syndrome, POMC/PCSK1 deficiency and LEPR deficiency, and safety information tied to approved uses.
Rhythm news also follows clinical data from setmelanotide studies, including acquired hypothalamic obesity and genetically caused MC4R pathway diseases, along with development of investigational MC4R agonists bivamelagon and RM-718 and preclinical small molecules for congenital hyperinsulinism. Recurring corporate items include financial results, specialty-pharmacy access arrangements, healthcare conference presentations and board governance changes.
Rhythm Pharmaceuticals announced promising results from its Phase 2 study of setmelanotide, a treatment for obesity and hyperphagia in individuals with Bardet-Biedl syndrome (BBS). Published in Diabetes, Obesity and Metabolism, the study shows that setmelanotide effectively reduced body weight and hunger. This marks the first Phase 2 trial of any investigational drug showing such efficacy for BBS. The company prepares for topline data from its pivotal Phase 3 trial on setmelanotide, expected by early 2021, further advancing treatment options for this ultra-rare genetic disorder.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced promising interim data from a Phase 2 study of its once-weekly formulation of setmelanotide, aimed at treating rare genetic disorders of obesity. The study found that the weekly formulation achieved weight loss comparable to the daily formulation, with both being safe and well-tolerated. A total of 75 participants were included in the interim analysis, showing no serious adverse events. The company is continuing to analyze the data and aims to discuss registration steps with the FDA, targeting a more convenient treatment option for patients.
Rhythm Pharmaceuticals (Nasdaq: RYTM) announced that Hunter Smith, Interim CEO and CFO, and Dr. Murray Stewart, CMO, will participate in a fireside chat at the 2020 BofA Securities Health Care Conference on May 12, 2020, at 8:20 a.m. ET. A live audio webcast will be available on Rhythm's website, with a replay accessible for 30 days after the event. The company focuses on developing therapies for rare genetic disorders of obesity and is advancing its lead candidate, setmelanotide, through pivotal clinical trials, with topline data expected in late 2020 or early 2021.