Rhythm Pharmaceuticals, Inc. has released positive topline results from its phase 2 trial evaluating Bivamelagon, an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity. The trial showed statistically significant and clinically meaningful reductions in body mass index (BMI) at 14 weeks of treatment. In the 600mg cohort, Bivamelagon achieved a BMI reduction of -9.3% from baseline, while in the 400mg cohort, it achieved a reduction of -7.7%. Patients in the placebo cohort experienced an increase in BMI by 2.2% over the same period.
Comparing the results of Bivamelagon with prior Setmelanotide trials, a post-hoc analysis demonstrated consistent BMI reductions. Patients in the 600mg and 400mg cohorts achieved a mean reduction greater than 2.8 points in their ‘most’ hunger scores measured on a 10-point scale at 14 weeks of Bivamelagon therapy. However, there were limited instances of localized hyperpigmentation observed during the trial.
Rhythm Pharmaceuticals plans to request an end-of-phase 2 meeting with the U.S. Food and Drug Administration (FDA) and seek scientific advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to advance Bivamelagon in acquired hypothalamic obesity. The company is also refining the formulation of Bivamelagon potentially to improve tolerability ahead of initiating a phase 3 trial.
The phase 2 trial was a randomized, placebo-controlled, double-blind study involving patients 12 years of age and older. The patients took an oral daily dose of either Bivamelagon (in 200mg, 400mg, or 600mg doses) or placebo for 14 weeks. Patients may continue on therapy in the open-label portion for up to 52 weeks.
Acquired hypothalamic obesity is a rare form of obesity that occurs following damage to the hypothalamic region of the brain, responsible for controlling physiological functions such as hunger and weight regulation. Rhythm estimates there are 5,000 to 10,000 people living with hypothalamic obesity in the U.S., 5,000 to 8,000 people living with hypothalamic obesity in Japan, and 3,500 to 10,000 people living with hypothalamic obesity in the E.U.
Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company with a focus on transforming the lives of patients and their families living with rare neuroendocrine diseases. Its lead asset, Imcivree (Setmelanotide), is an MC4R agonist designed to treat hyperphagia and severe obesity, and is approved by the U.S. FDA.
The company is now poised to advance Bivamelagon in acquired hypothalamic obesity and is looking forward to engaging with regulatory authorities for the next phase of the drug's development. As a result of these announcements, the company's shares have moved 36.63% on the market, and are now trading at a price of $89.00. If you want to know more, read the company's complete 8-K report here.
