Rhythm Announces Expansion in Setmelanotide Trials

Rhythm Pharmaceuticals Reveals Clinical Development Strategy for Setmelanotide in Addressing Hypothalamic Obesity in Japan

Rythm Pharmaceuticals, Inc.  a global commercial-stage biopharmaceutical company specializing in rare neuroendocrine diseases, has announced plans to expand its ongoing global Phase 3 clinical trial of setmelanotide to include patients with hypothalamic obesity in Japan. This expansion is scheduled to begin dosing in the third quarter of 2024.

Rhythm Plan for Setmelanotide

• Rhythm, expressed satisfaction with productive discussions with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), highlighting the company's clear plan to support potential approval of setmelanotide for hypothalamic obesity in Japan. 
• Given the higher incidence and prevalence of patients with this condition in Japan compared to the United States and Europe and the lack of approved therapeutic options, there is a significant unmet need for effective treatment.

Obesity facts & causes

• Rhythm estimates that approximately 5,000 to 8,000 individuals in Japan suffer from hypothalamic obesity, a rare condition resulting from damage to the hypothalamic region of the brain, particularly the melanocortin-4 receptor (MC4R) pathway responsible for regulating hunger and weight. 
• Hypothalamic obesity often occurs following the growth, surgical removal, or treatment of craniopharyngioma, astrocytoma, or other rare brain tumors, leading to rapid weight gain, reduced energy expenditure, and heightened hunger within six to twelve months post-lesion.

Upcoming Regulatory Documentation

• In collaboration with Japan's PMDA, Rhythm plans to include a cohort of 12 Japanese patients in the ongoing trial. 
• Pending trial completion, data from these patients will be part of the company's registration dossier submitted to Japan's Ministry of Health, Labor, and Welfare. 
• Additionally, Rhythm will collect and submit pharmacokinetics (PK) data from Japanese participants, streamlining the typical process of obtaining such data from an earlier-stage trial involving Japanese subjects.

Trials

• Furthermore, Rhythm has successfully enrolled patients in the pivotal 120-patient cohort of its global Phase 3 trial of setmelanotide in hypothalamic obesity, comprising individuals aged 4 years and older. 
• Participants will be randomized 2:1 to receive either setmelanotide therapy or placebo over a total period of 60 weeks, including up to eight weeks for dose titration. 
• Regulatory submissions to the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) will be based on data from this cohort, in accordance with agreements. 
• The inclusion of the additional 12-patient Japanese cohort will not impact the timing of regulatory submissions in the United States or Europe. 
• Rhythm remains on schedule to unveil top-line study results in the first half of 2025.

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