Rhythm Pharmaceuticals Completes Pivotal Enrollment in Two Ongoing Phase 3 Clinical Trials Evaluating Setmelanotide in Rare Genetic Disorders of Obesity
Rhythm expects to report initial data from the Phase 3 trials of both POMC and LEPR deficiency obesity in the third quarter of 2019. Rhythm then plans to submit concurrent New Drug Application (NDA) filings to the
“Patients with MC4R pathway deficiencies represent an underdiagnosed population, whose early-onset obesity and excess hunger impairs their lives,” said
The open-label, single-arm, multinational Phase 3 trials evaluating the safety and efficacy of setmelanotide in POMC and LEPR deficiency obesity share the same trial design. The primary endpoint in both trials is a responder analysis for weight, defined as patients achieving a 10 percent change from baseline. The first secondary endpoint in both trials is the mean percentage change in weight. Hunger scores are also key secondary endpoints.
Rhythm is also evaluating setmelanotide in four additional rare genetic disorders of obesity. Rhythm expects to initiate a Phase 3 study evaluating setmelanotide in Bardet-Biedl Syndrome in 2018. The Company has treated a number of the first patients in a Phase 2 proof-of-concept basket study evaluating setmelanotide in Alström Syndrome, POMC epigenetic disorders and POMC heterozygous deficiency obesity and expects to announce initial data in each indication in the second quarter of 2018. In addition, Rhythm has launched efforts to build a patient registry, Tracing the Effect of the MC4R Pathway in Obesity (TEMPO), and is supporting
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the Company’s first-in-class melanocortin-4 receptor (MC4R) agonist, in Phase 3 studies in patients with pro-opiomelanocortin (POMC) deficiency obesity (which includes deficiencies in both the POMC and PCSK1 genes) and leptin receptor (LEPR) deficiency obesity. Rhythm also supports The Genetic Obesity Project (www.GeneticObesity.com), which is dedicated to improving the understanding of severe obesity that results from specific genetic disorders. The company is based in Boston, MA.
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 expectations regarding its preparation of an NDA filing with the
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