Rhythm Pharmaceuticals (RYTM) Corporate presentation summary

Strategic focus and pipeline development

• Advancing MC4R agonist therapies across genetic, acquired, and next-generation indications, including setmelanotide, bivamelagon, and RM-718 for rare neuroendocrine diseases.

• IMCIVREE (setmelanotide) is approved for acquired hypothalamic obesity (HO) in the US and EU, with launches in additional countries planned.

• Pipeline includes ongoing and planned trials in congenital HO, Prader-Willi syndrome, and next-generation oral and injectable MC4R agonists.

• U.S. patent protection for next-generation assets extends into the 2040s.

Market opportunity and commercial execution

• Estimated prevalence for acquired HO: ~10,000 in both the US and Europe, 5,000–8,000 in Japan; Prader-Willi syndrome: ~20,000 in the US, ~400,000 worldwide.

• IMCIVREE is available in over 25 countries, with more than 100 employees across 13 countries and eight new country launches in 2025.

• Net product revenues have shown steady growth since the BBS launch, reaching $60.1M in Q1 2026.

• U.S. launch for acquired HO is off to a strong start, with ~110 patient start forms and >150 unique prescribers as of May 2026.

Clinical efficacy and safety data

• Phase 3 trial in acquired HO showed a -18.8% placebo-adjusted BMI reduction at 52 weeks (p<0.0001), with consistent efficacy across age groups and in patients with prior or concomitant GLP-1 use.

• Real-world and long-term extension data confirm sustained BMI reductions and improvements in hunger and body composition.

• Bivamelagon achieved statistically significant BMI reductions at all doses in a Phase 2 trial, with up to -9.3% mean BMI reduction at 600mg.

• Both setmelanotide and bivamelagon demonstrated meaningful reductions in hunger scores.