Rhythm Pharma Expands Setmelanotide Trials for Hypothalamic Obesity in Japan

Rhythm Pharmaceuticals, a biopharmaceutical firm specializing in rare neuroendocrine disorders, has announced the expansion of its global Phase 3 clinical trial for setmelanotide to include a Japanese cohort suffering from hypothalamic obesity. The company aims to address the significant need for treatment in Japan, where an estimated 5,000 to 8,000 individuals are affected by this rare condition, which often results from brain tumor treatments and is characterized by rapid weight gain and increased hunger.
The decision to include the Japanese cohort follows positive discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) and is expected to commence dosing in the third quarter of 2024. David Meeker, Rhythm's CEO, highlighted the lack of approved therapies and the higher incidence of the disease in Japan, emphasizing the urgency for a new treatment option.
The trial will incorporate 12 Japanese patients and utilize the data for regulatory approval from Japan's Ministry of Health, Labor and Welfare. Rhythm will also collect pharmacokinetics data from these patients, which will help expedite the approval process. The company has completed enrollment for the primary 120-patient cohort of the trial, which is focused on individuals aged four and above with hypothalamic obesity. The study is designed to assess the efficacy and safety of setmelanotide over a 60-week period, with regulatory submissions in the US and Europe based on these results.
Setmelanotide, Rhythm's lead product, is an MC4R agonist that has been approved in the US for chronic weight management in patients with specific genetic forms of obesity. It is also authorized in the European Union for the treatment of obesity associated with certain genetic conditions. The drug is not indicated for all types of obesity and has contraindications, including serious hypersensitivity reactions.
Rhythm is also developing setmelanotide for other rare diseases and has a pipeline of small molecules for congenital hyperinsulinism. The company's headquarters are located in Boston, Massachusetts, and it is committed to improving the lives of patients with rare neuroendocrine diseases.