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Rhythm Pharmaceuticals Completes Enrollment of Pivotal Cohort in Phase 3 Clinical Trial Evaluating Setmelanotide in Bardet-Biedl and Alström Syndromes
-- Topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 -- -- Supplemental cohort continues enrollment to meet patient
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- Topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --\n -- Supplemental cohort continues enrollment to meet patient demand -- BOSTON, Dec. 05, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced it completed enrollment of the pivotal cohort in its Phase 3 clinical trial evaluating setmelanotide for the treatment of insatiable hunger and severe obesity in individuals living with Bardet-Biedl syndrome (BBS) or Alström syndrome. The Company enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort, and it will continue to enroll patients in a supplemental cohort to meet demand and provide further data on the use of setmelanotide in people living with these conditions. “With no approved treatment options available, there is a pressing need for a new therapy that addresses the insatiable hunger and severe obesity that people living with BBS or Alström syndrome may experience,” said Murray Stewart, M.D., Chief Medical Officer of Rhythm. “We believe the significant demand among patients, families, caregivers and healthcare providers in the BBS community reflects this need for a therapy to address these conditions. We look forward to continuing to partner with the BBS and Alström syndrome communities as we work to identify patients, better understand the long-term burden of these disorders and advance this pivotal trial toward topline data in the fourth quarter of 2020 or early in the first quarter of 2021.” About Bardet-Biedl and Alström SyndromesBBS and Alström syndrome are ultra-rare genetic disorders that affect multiple organ systems. Clinical features of BBS may include cognitive impairment, polydactyly, renal dysfunction, hypogonadism and visual impairment. Clinical features of Alström syndrome may include progressive visual and auditory impairment, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy and short stature in adulthood. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be common in people living with either BBS or Alström syndrome. There is great variability in presentation and seve...