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Rhythm Pharmaceuticals Announces Positive Topline Results from Pivotal Phase 3 Clinical Trial Evaluating Setmelanotide in Bardet-Biedl and Alström Syndromes
-- Study met primary endpoint and all key secondary endpoints with statistically significant and clinically meaningful reductions in weight and hunger -- --
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- Study met primary endpoint and all key secondary endpoints with statistically significant and clinically meaningful reductions in weight and hunger -- -- All primary endpoint responders were patients with BBS -- -- Plan to submit sNDA to FDA for BBS in the second half of 2021 -- -- Company to host conference call today at 8 a.m. ET -- BOSTON, Dec. 22, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced positive topline results from a pivotal Phase 3 clinical trial evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of insatiable hunger and severe obesity in individuals with Bardet-Biedl syndrome (BBS) or Alström syndrome. The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores. All primary endpoint responders were patients with BBS. There were three evaluable patients with Alström syndrome and none of them met the primary endpoint. “These Phase 3 results add to our growing understanding of setmelanotide’s potential to treat people living with rare genetic diseases of obesity,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We are pleased with the robust response observed in BBS patients, which supports our goal of delivering a precision medicine to this well-characterized patient population who suffer from insatiable hunger and severe, early-onset obesity. Although we are disappointed that none of the three evaluable Alström patients met the primary endpoint, we are encouraged by trends in hunger and weight reduction in some patients and look forward to evaluating the full data as we finalize our path forward in this indication.” Rhythm enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort for this Phase 3 trial. The primary analysis was conducted on 31 evaluable participants (28 with BBS and three with Alström syndrome) 12 years old and older. Five study participants (three with BBS and two with Alström syndrome) were younger than 12 years old at enrollment. The analysis of the primary endpoint shows: 11 of 31 or 34.5 percent1 of part...