Rhythm Pharmaceuticals reports positive data for setmelanotide in obesity

Rhythm Pharmaceuticals announced positive proof-of-concept data from multiple cohorts in its Phase 2 Basket Study evaluating setmelanotide in patients with severe obesity due to genetic variants in the melanocortin-4 receptor pathway, and provided an update on its genetic sequencing efforts. The company also discussed plans to initiate a potentially registration-enabling Phase 3 trial evaluating setmelanotide in patients with MC4R pathway deficiencies due to a variant in one of the two alleles in the POMC, PCSK1, or LEPR genes, as well as the SRC1 and SH2B1 genes. Additionally, Rhythm provided an update on its sequencing efforts, now comprised of samples from approximately 37,500 individuals with severe obesity, and detailed an additional planned exploratory study to evaluate setmelanotide for the treatment of obesity due to a deficiency in one of 31 additional genes associated with the MC4R pathway. Rhythm estimates that there are between 100,000 and 200,000 people with HET obesity or SRC1 or SH2B1 deficiency obesity in the U.S. who could potentially benefit from setmelanotide therapy. Clinical data from Phase 2 Basket Study including five cohorts totaling 65 patients demonstrated proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, SRC1 and SH2B1 deficiency obesities. New data from pivotal Phase 3 trial in Bardet-Biedl syndrome showed statistically significant and clinically meaningful improvements in pediatric BMI-Z scores.