Rhythm Pharmaceuticals (RYTM) Canaccord Genuity's 46th Annual Growth Conference summary

Company overview and product pipeline

• Operates in over 24 countries with primary therapy setmelanotide (IMCIVREE), the first MC4R agonist approved for rare genetic obesity disorders.

• Initial approvals targeted ultra-orphan biallelic obesity forms, later expanding to Bardet-Biedl syndrome (BBS) and acquired hypothalamic obesity (HO).

• Achieved global launch after 2022 BBS approval, with Q1 revenue run rate around $60 million.

• Recent phase III data in acquired HO showed 18.8% body weight loss, supporting further regulatory filings.

• Pipeline includes oral MC4R agonist bivamelagon (phase III planned by year-end) and weekly injectable RM-718, both showing proof of concept in HO.

Commercial launch and market expansion

• Over 400 start forms for IMCIVREE in AHO within 14 weeks, written by 300+ physicians, indicating broad adoption.

• Two-thirds of approved scripts received prior authorization; payer coverage progressing faster than for BBS.

• Dedicated BBS field team established to support ongoing growth in that segment.

• U.S. AHO market estimated at 10,000 patients, with confidence in upper range due to broader tumor types and improved survival.

• No evidence of a bolus effect; new patient starts remain steady and market penetration is still early.

International strategy and regulatory progress

• Japan HO prevalence estimated at 50–80% of U.S. levels; regulatory process streamlined with PMDA allowing a small local cohort for registration.

• Japanese launch expected around year-end, with full staffing and patient identification underway.

• U.S. launch pace influenced by endocrinologist capacity and payer processes; Japan offers national reimbursement at approval and stable pricing.

• Early access programs in France and Italy have enabled treatment of HO patients pre-approval; European launches depend on country-by-country reimbursement.

• Germany expected to grant reimbursement exemption by year-end, enabling Q1 launch.