RYTM News & Events

Reports Q1 revenue $60.11M, consensus $56.07M. "The U.S and European approvals of IMCIVREE mark a transformational milestone for patients with acquired hypothalamic obesity who, until now, had no approved therapies for this devastating disease," said David Meeker, Chairman, Chief Executive Officer and President of Rhythm Pharmaceuticals. "The U.S. launch is off to a strong start, as patient demand and physician engagement in this early stage of the launch reinforce the significant unmet need and long-term opportunity in acquired HO. With European Commission authorization for acquired HO earlier than expected, we look forward to engaging with country-level officials with launches anticipated in 2027. We are progressing rapidly in Japan for acquired HO, as well; regulatory review is underway, and if approved, we anticipate launching there in the second half of 2026."

Meeker added, "In addition, we continue to advance our MC4R agonism pipeline with upcoming data readouts from ongoing trials of setmelanotide in Prader-Willi syndrome and our weekly injectable, RM-718, in acquired HO. Our ongoing global commercial efforts and clinical development pipeline position Rhythm to achieve long-term, sustained growth."

Rhythm Pharmaceuticals announced the presentation of new data delivered by Rhythm and its partners at The Pediatric Endocrine Society Annual Meeting. "The data presented provide important longer-term insights into treatment response for pediatric patients treated with setmelanotide, including reductions across multiple weight-related measures," said Jennifer Miller, professor of pediatrics, pediatric endocrinology, University of Florida College of Medicine. Pediatric patients with acquired hypothalamic obesity who received setmelanotide for up to 2.5 years achieved sustained reductions across multiple age-adjusted weight-related measures. Among pediatric participants who completed 2.5 years of treatment, mean changes from baseline included a 16.4% change in BMI. Furthermore, data from the Phase 3 TRANSCEND trial showed that after one year of treatment, 71.1% of pediatric patients treated with setmelanotide improved by at least one weight category.

Rhythm Pharmaceuticals announced the European Commission, or EC, has expanded the marketing authorization for Imcivree - setmelanotide - to include the treatment of obesity and control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity, or HO, due to hypothalamic injury or impairment. Acquired HO is a rare neuroendocrine disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or impairment. Rhythm estimates there are approximately 10,000 people living with acquired HO in Europe. The EC approval is based on results from the Phase 3 TRANSCEND trial of setmelanotide in acquired HO. The global study met its primary endpoint. Setmelanotide was generally well tolerated in the TRANSCEND study. With EU authorization in place, The company will now work to secure access on a country-by-country basis across Europe. Commercial launches are anticipated in 2027. The company's supplemental New Drug Application was approved by the FDA on March 19 for Imcivree to treat patients in the U.S. living with acquired HO.