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 demand --
“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
About Bardet-Biedl and Alström Syndromes
BBS 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 severity of these symptoms across individuals with BBS or Alström syndrome. In
About the Pivotal Phase 3 Trial
The pivotal Phase 3 trial is designed to evaluate setmelanotide, a MC4R agonist, for the treatment of obesity and hyperphagia in people with BBS or Alström syndrome (NCT03746522). The trial is designed to enroll 30 patients, including at least 20 patients with BBS and at least six patients with Alström syndrome, aged six years and older. Participants were randomized to placebo or setmelanotide for 14 weeks followed by an open-label period on setmelanotide for 52 weeks. The primary endpoint of the trial is the proportion of participants (≥12 years of age) who achieve at least 10 percent reduction in body weight from baseline at approximately 52 weeks of therapy. Rhythm expects to report topline data from this trial in the fourth quarter of 2020 or early in the first quarter of 2021.
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide, its MC4R agonist, in people living with pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity, and plans to complete its first rolling New Drug Application (NDA) submission to the
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm’s anticipated timing for enrollment of patients in clinical trials and submission of an NDA, its expectations regarding addressable patient populations, its expectations regarding the timing of topline data from its clinical trials, and its ongoing efforts related the efficacy of setmelanotide in patients with POMC deficiency obesity, LEPR deficiency obesity, BBS syndrome and Alström syndrome. Statements using word such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward looking statements. Such statements are subject to numerous risks and uncertainties, including but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our sufficiency of cash and our expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the
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Source: Rhythm Pharmaceuticals, Inc.