Business
Rhythm Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results
-- 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
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- On track to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020 --\n -- 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 -- -- Ongoing genetic sequencing efforts have sequenced more than 25,000 people with early-onset, severe obesity -- BOSTON, March 02, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2019. “We believe Rhythm is entering 2020 in a position of strength as we continue to make significant progress in the science and understanding of rare genetic disorders of obesity and prepare to deliver setmelanotide as the first therapeutic option for people living with these conditions,” said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm. Murray Stewart, M.D., Chief Medical Officer, added, “We expect to submit our first New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming weeks. In parallel, we are focused on advancing the development of setmelanotide in five additional melanocortin-4 receptor (MC4R) pathway-related disorders in our Phase 2 Basket study. We are also working to build a robust community of patients, caregivers and healthcare providers in order to drive patient identification, increase genetic testing and improve the understanding of these conditions. These efforts are critically important to our progress in Bardet-Biedl syndrome (BBS) and Alström syndrome, for which we expect topline pivotal data in the fourth quarter of 2020 or early in the first quarter of 2021. Lastly, our ongoing sequencing efforts continue to demonstrate that a significant pe...