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Rhythm Pharmaceuticals Optimizes Design of EMANATE and DAYBREAK Clinical Trials to Advance Setmelanotide for Rare Genetic Diseases of Obesity
– Phase 3 EMANATE trial to focus on rare patient populations with highest likelihood for success – – First patient enrolled in EMANATE – – Ongoing Phase 2
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"– Phase 3 EMANATE trial to focus on rare patient populations with highest likelihood for success – – First patient enrolled in EMANATE – – Ongoing Phase 2 DAYBREAK trial to focus initially on 10 genes with strongest relevance to the MC4R pathway – – Trial and other program changes extend cash runway into 4Q2023 – BOSTON, April 06, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced modifications intended to optimize its Phase 3 EMANATE and ongoing Phase 2 DAYBREAK trials to focus on rare patient populations, which the Company believes have the highest likelihood of success. These changes are intended to optimize the design of each clinical trial, with the goal of most efficiently advancing setmelanotide as a precision medicine for patients with rare genetic diseases of obesity. The EMANATE trial now includes four independent sub-studies evaluating setmelanotide, the Company’s melanocortin-4 receptor (MC4R) agonist, in patients with obesity due to a heterozygous variant of the POMC/PCSK1 genes, the LEPR gene, the SRC1 gene and the SH2B1 gene. Rhythm estimates that patients with rare variants in these genes represent a potential addressable U.S. population of approximately 53,000, based on internal genetic sequencing data. The Company also announced today that the first patient has been enrolled in the EMANATE trial. Recent feedback from the U.S. Food and Drug Administration (FDA) indicated that additional clinical trials to support potential registration for non-rare patient populations would likely be required. As a result, Rhythm eliminated a fifth sub-study, intended to evaluate setmelanotide in patients with a PCSK1 N221D variant. In the POMC/PCSK1 and LEPR sub-studies, the Company will focus enrollment on heterozygous variants classified as pathogenic and likely pathogenic, as initially planned. In addition, instead of enrolling across the entire spectrum of variants of uncertain significance (VUS), Rhythm will focus on patients with suspected pathogenic variants, or a subset of VUS, which are most likely to impair MC4R pathway function. “We believe these modifications improve the likelihood of success for EMANATE’s independent sub-studies by focusing exclus...