Rhythm Pharmaceuticals (NasdaqGM:RYTM) Earnings Call Presentation

Clinical Trial Results & Regulatory - Setmelanotide achieved a 19.8% placebo-adjusted difference in BMI reduction from baseline in the Phase 3 acquired hypothalamic obesity (HO) trial (P<0.0001)[33] - In the setmelanotide arm of the Phase 3 trial, patients experienced a -16.5% change in BMI from baseline[32] - A statistically significant reduction in Most Hunger Score was observed with setmelanotide vs placebo in participants aged ≥12 years (P=0.0086)[40] - Setmelanotide was generally well-tolerated in the Phase 3 trial, with most common adverse events including skin hyperpigmentation (55.6%), nausea (50.6%), and headache (38.3%)[45] Market Opportunity & Commercial Strategy - The estimated U S prevalence of acquired HO is approximately 10,000 individuals[48] - The estimated prevalence in Europe is also around 10,000 individuals[48] - The estimated prevalence in Japan is between 5,000 and 8,000 individuals[48] - Rhythm anticipates strong commercial and Medicaid coverage for setmelanotide in HO, similar to or better than coverage for Bardet-Biedl syndrome (BBS)[82] Company Readiness - Rhythm has established a solid global foundation with IMCIVREE available in >25 countries and >350 employees in 15 different countries[23] - The company has an experienced rare disease team in place to support ongoing growth in BBS[72]