Rhythm says setmelanotide demonstrated clinically meaningful reductions in BMI

Rhythm Pharmaceuticals hosted an R&D event for investors and analysts, “Rhythm Update on MC4R Pathway Programs.” During the event, the Company announced new topline data from its ongoing Phase 3, open-label pediatrics trial evaluating one year of setmelanotide therapy in patients between the ages of 2 and younger than 6 years with Bardet-Biedl syndrome or proopiomelanocortin or leptin receptor deficiency obesity and new preliminary data from the first stage of its exploratory Phase 2 DAYBREAK trial. Rhythm also presented pre-clinical data on its new drug candidate, RM-718, which is designed to be a more selective MC4R agonist with weekly administration, and its plans for Phase 1 clinical development. The Company hosted Amy Wood, Executive Director and Co-Founder of the Raymond A. Wood Foundation, and Jennifer Miller, MD, Professor of Pediatric Endocrinology, University of Florida, who shared insights on hypothalamic obesity and the severe impact of the disease on patient quality of life. Rhythm continues to advance the development of setmelanotide in patients with hypothalamic obesity and remains on track to complete Phase 3 study enrollment by the end of 2023. Setmelanotide demonstrated clinically meaningful reductions in BMI and BMI-Z score in patients aged 2 to less than6. Rhythm presented new data from its 52-week, Phase 3 pediatrics trial in patients between 2 and younger than 6 years old. The hyperphagia and severe obesity of rare genetically-caused MC4R pathway diseases can present early in life, and these data show potential efficacy in patients younger than 6. This trial is a multi-center, one-year, open-label trial in pediatric patients with obesity due to biallelic POMC, PCSK1 or LEPR deficiency or a clinical diagnosis of BBS with genetic confirmation. The primary efficacy endpoint is a responder analysis, based on the proportion of patients who experience a decrease from baseline in BMI-Z score of greater than or equal to0.2. Highlights from the data include: 83.3% of all patients achieved greater than or equal to 0.2 reduction in BMI-Z score from baseline to week 52; 18.4 percent mean reduction from baseline in BMI at week 52; 3.04 mean reduction from baseline in BMI-Z score at week 52; 11 patients completed the trial, and all remain on therapy, as of Dec. 5, 2023; one patient discontinued and was lost to follow-up; and The safety profile is consistent with past trials evaluating setmelanotide. Rhythm also announced it has submitted a Type II variation application to the European Medicines Agency seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hunger in pediatric patients between 2 and younger than 6 years old with BBS or POMC, PCSK1 or LEPR deficiency in the European Union. The Company anticipates submitting a supplementary New Drug Application to the U.S. Food and Drug Administration in the first half of 2024 seeking a similar label expansion.