Rhythm Pharmaceuticals, Inc. announced that the company will outline expected 2019 milestones and review updated clinical data from its Phase 2 basket studies of setmelanotide in patients with BBS in a presentation at the 37th Annual J.P. Morgan Healthcare Conference on January 10, 2019. Setmelanotide is a first-in-class melanocortin-4 receptor (MC4R) agonist. The company announced updated clinical data from two adolescent patients in its Phase 2 basket studies evaluating setmelanotide for the treatment of BBS who previously had only short-term results (18 and 15 weeks of total treatment). Data is now available for 47 and 41 weeks of treatment. These two patients have lost 11.2% (-13.7 kg) and 15.5% (-13.7 kg) of their body weight and experienced hunger score reductions of 66% and 21%, respectively. Treatment with setmelanotide continues to be well tolerated and safety data were consistent with previous clinical studies. In total, six out of nine BBS patients enrolled in the Phase 2 basket studies have now achieved a clinically meaningful weight loss of 10% change from baseline, which is the primary endpoint for the company’s pivotal Phase 3 clinical trial, and, as previously reported, one additional patient with Type-1 diabetes responded with marked improvements in hunger score and blood sugar levels. Rhythm’s ongoing, separate pivotal Phase 3 clinical trials evaluating setmelanotide in POMC and LEPR deficiency obesity continue to progress, with topline data expected in the third quarter of 2019. Pending positive results, the company expects to submit an NDA filing for each of POMC and LEPR deficiency obesity to the U.S. Food and Drug Administration (FDA) in late 2019 or early 2020. Rhythm continues to evaluate setmelanotide for the treatment of additional rare genetic disorders of obesity in its Phase 2 basket studies, including POMC and other MC4R pathway deficiency heterozygous obesities, as well as POMC epigenetic disorders. The company expects to announce updated interim data from these disorders in the first quarter of 2019. In addition, Rhythm is continuing to expand the evaluation of setmelanotide into additional MC4R pathway disorders in its Phase 2 basket studies. Setmelanotide is a potent, first-in-class, MC4R agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide activates MC4R, part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes within the MC4R pathway are associated with unrelenting hunger and severe, early-onset obesity. Rhythm is currently developing setmelanotide as a replacement therapy for patients with monogenic defects upstream of MC4R, for whom there are no effective or approved therapies. The FDA has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström Syndrome. The European Medicines Agency has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway.