BOSTON - Rhythm Pharmaceuticals Inc. (NASDAQ:RYTM), a biopharmaceutical company specializing in rare neuroendocrine diseases, has announced the inclusion of a Japanese patient cohort in its global Phase 3 clinical trial for setmelanotide, aimed at treating hypothalamic obesity. The trial extension follows discussions with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and is expected to begin dosing in the third quarter of 2024.
The decision to add 12 Japanese patients to the study comes in response to the significant unmet medical need in Japan, where an estimated 5,000 to 8,000 individuals suffer from hypothalamic obesity, a condition characterized by severe obesity and hyperphagia following damage to the hypothalamus. This rare form of obesity often results from the treatment of brain tumors such as craniopharyngioma and astrocytoma.
David Meeker, M.D., Chair, President, and Chief Executive Officer of Rhythm, expressed optimism about the plan, which aims to expedite the approval process for setmelanotide in Japan. The company has completed enrollment for its pivotal 120-patient cohort in the global Phase 3 trial, with results expected in the first half of 2025. The additional Japanese patients will not impact the timing for regulatory submissions in the United States or Europe.
Setmelanotide, Rhythm's lead asset, is an MC4R agonist approved in the United States for chronic weight management in patients with certain genetic disorders leading to obesity. It is also authorized in the European Union for obesity treatment associated with genetic conditions.
This expansion into the Japanese market represents a strategic move for Rhythm Pharmaceuticals, as it continues to develop and commercialize treatments for rare diseases with high unmet medical needs. This initiative is based on a press release statement from the company.
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