Rhythm Pharmaceuticals (RYTM) Corporate presentation summary

Strategic positioning and growth

• Global commercial infrastructure supports IMCIVREE availability in over 25 countries, targeting rare genetic obesity disorders and acquired hypothalamic obesity (HO).

• IMCIVREE received FDA approval for acquired HO in March 2026, with expansion opportunities in Europe and Japan.

• Next-generation MC4R agonists, RM-718 (weekly) and oral bivamelagon, have patent protection into the 2040s.

• Cash reserves of $388.9M as of December 2025 are expected to fund operations for at least 24 months.

• Leadership team brings extensive experience in rare disease drug development and commercialization.

Product pipeline and clinical milestones

• IMCIVREE is approved for acquired HO, Bardet-Biedl syndrome (BBS), and POMC, PCSK1, or LEPR deficiencies.

• Multiple clinical trials are ongoing or planned for setmelanotide, bivamelagon, and RM-718 in rare neuroendocrine diseases, including Prader-Willi syndrome (PWS) and congenital HO.

• Key 2026 milestones include European Commission decision on IMCIVREE for HO, Phase 3 trial initiations, and data disclosures for PWS and congenital HO studies.

• Bivamelagon achieved statistically significant BMI reductions at all doses in Phase 2 trials for HO.

• Real-world and clinical data support efficacy and safety of setmelanotide and bivamelagon in reducing BMI and hunger in HO and related disorders.

Market opportunity and commercial performance

• Estimated U.S. prevalence: acquired HO (~10,000), BBS (4,000–5,000), POMC/PCSK1/LEPR deficiencies (600–2,500), and PWS (~20,000).

• IMCIVREE net product revenues have shown steady growth since BBS launch, with global sales increasing each quarter.

• IMCIVREE is available in more than 25 countries, with over 100 employees across 13 countries and continued expansion.

• Patent protection for next-generation assets extends into the 2040s, supporting long-term commercial potential.