Rhythm Pharmaceuticals Announces Clinical Development Plan of Setmelanotide for Hypothalamic Obesity in Japan

-- Supplemental cohort of 12 Japanese patients to be added to global Phase 3 hypothalamic obesity trial, with dosing expected to begin in third quarter of 2024 --

-- Significant unmet need with 5,000 to 8,000 patients with hypothalamic obesity estimated to be living in Japan --

BOSTON, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced plans to add a cohort of patients with hypothalamic obesity in Japan to its ongoing global Phase 3 clinical trial of setmelanotide, with dosing expected to begin in the third quarter of 2024.

“Following constructive discussions with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), we are pleased to have developed a clear and efficient plan to support the potential approval of setmelanotide for hypothalamic obesity in Japan,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “With data showing a higher per-capita incidence and prevalence of patients with hypothalamic obesity in Japan compared to the United States and Europe and no approved therapeutic options, there is a major unmet need for a therapy to treat the rapid onset of severe obesity and hyperphagia of hypothalamic obesity.”

Rhythm estimates that there are approximately 5,000 to 8,000 patients in Japan with hypothalamic obesity, a rare form of obesity that occurs following damage to the hypothalamic region of the brain, which includes the melanocortin-4 receptor (MC4R) pathway and is responsible for controlling physiological functions such as hunger and weight regulation. The condition most frequently follows the growth and surgical removal or other treatment of craniopharyngioma, astrocytoma, or other rare brain tumors. These individuals often experience rapid weight gain, a reduction in energy expenditure, and an increase in hunger leading to severe obesity within six to 12 months following the hypothalamic lesions.

The Company and Japan’s PMDA agreed to add a cohort of 12 Japanese patients to the ongoing trial and, pending completion of the trial, to use these data as part of the Company’s registration package to seek approval from Japan’s Ministry of Health, Labor and Welfare. In addition to efficacy data, Rhythm will collect and submit pharmacokinetics (PK) data from Japanese patients, expediting the typical pathway of collecting such data from an earlier-stage trial in Japanese subjects.