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Rhythm Pharmaceuticals Announces Comprehensive Expansion of Clinical Development Program with Five New Phase 2 and 3 Trials Planned to Evaluate Setmelanotide in Rare Genetic Diseases of Obesity
-- Pivotal Phase 3 EMANATE trial planned in patients with heterozygous POMC, PCSK1 or LEPR, and SRC1 or SH2B1 deficiency obesities ---- EMANATE, as well as
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- Pivotal Phase 3 EMANATE trial planned in patients with heterozygous POMC, PCSK1 or LEPR, and SRC1 or SH2B1 deficiency obesities ---- EMANATE, as well as Phase 2 DAYBREAK trial evaluating setmelanotide in 31 new genes, Phase 3 pediatrics trial and Phase 3 trials of weekly setmelanotide formulation all expected to initiate in 2H 2021 ---- Updated Uncovering Rare Obesity genetic test with expanded gene panel launched in July -- -- Company to host conference call at 8 a.m. ET today -- CAMBRIDGE, Mass., Aug. 03, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced a significant expansion of its clinical development program for setmelanotide with five new planned Phase 2 and 3 clinical trials. Rhythm will review these updates on its second quarter 2021 financial results and business update conference call today at 8 a.m. ET. New trials include the pivotal EMANATE Phase 3 trial and the DAYBREAK Phase 2 trial, which together will evaluate setmelanotide in people with variants in one of 36 genes in the melanocortin-4 receptor (MC4R) pathway. Based on genetic sequencing data and setmelanotide response rates achieved in a phase 2 trial, Rhythm estimates that the five genetic indications being studied in the EMANATE trial represent a potential addressable patient population of approximately 100,000-200,000 people in the United States. In addition, Rhythm plans to initiate a Phase 3 trial in pediatric patients ages 2 to 6 years old, as these genetic diseases often present with severe obesity very early in life. The weekly formulation of setmelanotide, which is designed to improve compliance and adherence, will be studied in two phase 3 trials, including a switch study of patients currently on setmelanotide therapy and a de novo trial in patients with BBS. The company expects to initiate all five studies in the second half of 2021. “We are excited to announce the significant expansion of our development program for setmelanotide. With these five clinical trials, we will explore opportunities to extend the reach of our precision medicine to address the needs of many more patients suffering from a rare genetic disease of obesity, as well as the potential to improve convenience f...