Rhythm Pharmaceuticals, Inc. Announces Progress in Clinical Development of Setmelanotide for Treatment of Rare Genetic Disorders of Obesity
January 04, 2018 at 08:00 am
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Rhythm Pharmaceuticals, Inc. announced recent progress in the clinical development of the company’s lead product candidate, setmelanotide, a first-in-class melanocortin-4 receptor (MC4R) agonist. Following recent discussions with the U.S. Food & Drug Administration (FDA), Rhythm has finalized the protocol for its ongoing, open-label, single-arm, multinational pivotal Phase 3 clinical trial evaluating setmelanotide in patients with POMC deficiency obesity, an ultra-rare orphan disease that results in hyperphagia and severe, early-onset obesity. The FDA previously granted Breakthrough Therapy Designation for setmelanotide in this indication. The study originally positioned mean percentage change in weight from baseline as the primary endpoint, and a categorical analysis of responders for weight, defined as patients achieving a 10% change from baseline, as the first secondary endpoint. Following recent discussions with regulatory authorities, the study’s primary endpoint will now be the responder analysis, with mean percentage change in weight as the first secondary endpoint, in a change that increases the power of the trial. Other key secondary endpoints on hunger remain unchanged. The company confirms plans to file a New Drug Application (NDA) with the FDA based on one-year data from a cohort of 10 patients. The company currently has eight patients enrolled in its pivotal Phase 3 clinical trial, and expects to complete enrollment of the 10 required patients in the first half of 2018. In addition, the company plans to continue enrolling supplemental patients who may not complete one year of treatment at the time of NDA filing, including patients between six and 11 years of age under the implementation of a pediatric amendment, to provide additional important data regarding the use of setmelanotide in people living with POMC deficiency obesity. The company continues to expect to report initial data from this trial in the first half of 2019.
Rhythm Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on advancing its melanocortin-4 receptor (MC4R) agonists, including its lead asset, IMCIVREE, as a precision medicine designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases. IMCIVREE is approved by the Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients six years of age and older with monogenic or syndromic obesity due to proopiomelanocortin (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); or Bardet-Biedl syndrome (BBS). It is evaluating setmelanotide in Phase II and III trials for the treatment of obesity due to variants in one of numerous genes associated with the MC4R pathway.