Rhythm Pharmaceuticals presented data from its clinical programs for acquired hypothalamic obesity at the Endocrine Society’s Annual Meeting (ENDO 2025). The studies focused on the efficacy and safety of bivamelagon and setmelanotide, both MC4R agonists designed to address this rare condition caused by damage to the hypothalamus. These presentations detailed positive clinical trial results showing significant weight loss in patients.
This research is crucial because acquired hypothalamic obesity, resulting from brain tumors or their treatment, currently lacks effective treatments. The rapid weight gain, excessive hunger, and reduced energy expenditure associated with this condition significantly impact patients’ quality of life. Positive clinical data for new therapies offer hope for improved management of this challenging condition. The potential for these therapies to become standard of care represents a major step forward for patients.
Phase 2 trial data for bivamelagon revealed statistically significant BMI reductions: 9.3% in the 600mg cohort, 7.7% in the 400mg cohort, and 2.7% in the 200mg cohort, over 14 weeks. Meanwhile, the Phase 3 TRANSCEND trial of setmelanotide demonstrated a remarkable 19.8% placebo-adjusted difference in BMI reduction over 52 weeks. Importantly, significant BMI reductions were observed across various age and sex subgroups, and even in patients with prior or current use of GLP-1s.
The positive results from both bivamelagon and setmelanotide trials suggest a promising future for individuals with acquired hypothalamic obesity. The data support the potential of these MC4R agonists to address the unmet medical needs of this patient population, offering hope for improved health outcomes and quality of life. Further research and regulatory progress will be critical in determining the ultimate role of these therapies in managing this rare and serious condition.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
