Rhythm Pharmaceuticals Inc. (NASDAQ: RYTM) shares jumped on Wednesday after the company announced positive results from two of its late-stage obesity clinical trials. Specifically, the data come from two Phase 3 trials evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities.
Both studies met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.
In terms of the results, eight of 10 patients with POMC deficiency obesity achieved the primary endpoint of greater than 10% weight loss over roughly one year. Additionally, 50% of the POMC deficiency obesity patients in the trial met or exceeded a 25% improvement in self-reported hunger scores.
Five of 11 patients with LEPR deficiency obesity achieved the primary endpoint of greater than 10% weight loss over one year. At the same time, 72.7% of the LEPR deficiency obesity patients in the trial met or exceeded a 25% improvement in self-reported hunger scores.
Keith Gottesdiener, M.D., CEO of Rhythm, commented:
We believe these statistically significant data demonstrate setmelanotide’s ability to induce marked weight loss and substantially reduce hunger and we are excited about the potential difference we can make in the lives of people with rare genetic disorders of obesity. We believe these pivotal data are the first step towards making a positive impact for people affected by rare genetic disorders of obesity who have grown up with insatiable hunger and early-onset, rapid weight gain that often leads to debilitating comorbidities. We believe this milestone moves us closer to delivering a treatment for numerous MC4R pathway-driven disorders of obesity. We are working to advance setmelanotide to its first regulatory submission in POMC and LEPR deficiency obesities.
Shares of Rhythm Pharma traded up about 20% Wednesday to $22.92, in a 52-week range of $18.00 to $34.97. The consensus price target is $37.14.