Market Assessment
Rhythm Pharmaceuticals (RYTM) is a small-cap ($695M) biopharma developing anti-obesity therapeutics for rare and ultra-rare genetic obesity disorders. The lead drug candidate, setmelanotide (formerly RM-493), is a melanocortin-4 receptor (MC4R) agonist peptide in clinical trials for rare genetic obesity disorders associated with pro-opiomelanocortin ((POMC)), leptin receptor ((LEPR)) deficiency as well as Bardet-Biedl syndrome ((BBS)) and Alström syndrome. The global prevalence of rare genetic obesity is not fully known, but misdiagnosis and/or underdiagnosis could be contributing factors.
In Q3/2019, Rhythm reported that setmelanotide was clinically successful in a Phase 3 study in