Rhythm Pharmaceuticals reported its Q4 and full year 2019 financial results, highlighting progress in developing therapies for rare genetic disorders of obesity and preparing for the potential launch of setmelanotide.
On track to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020.
Granted orphan drug designation by the European Medicines Agency for setmelanotide in Alström syndrome.
Completed full enrollment in Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in December; topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021.
Rhythm Phase 2 Basket study actively enrolling multiple cohorts across five rare MC4R pathway-related disorders with potential U.S. prevalence greater than 80,000 patients; data updates expected in 2020.
Rhythm Pharmaceuticals is focused on advancing the development of setmelanotide and building a community to improve the understanding and treatment of rare genetic disorders of obesity.