Rhythm Pharmaceuticals (RYTM) Jefferies Global Healthcare Conference 2026 summary

Business and product updates

• Recent U.S. launch of IMCIVREE for hypothalamic obesity (HO) showed over 150 new patient start forms in six weeks, with 80% of prescribing physicians new to the product and about 40 clinical trial patient conversions.

• Anticipates near-term approval and self-launch of IMCIVREE in Japan by year-end, and a potential launch in Germany pending G-BA exemption, either late this year or early next.

• Early launch feedback indicates strong physician interest, with a broadened sales force targeting 5,000 potential prescribers and significant engagement from both specialty centers and community endocrinologists.

• Initial patient mix is more pediatric, expected to normalize over time as care transitions from specialty centers to broader endocrinology practices.

• Reimbursement processes are progressing, with early positive payer responses and expectations for faster policy adoption compared to previous indications.

Clinical development and data updates

• Updated phase II data for IMCIVREE in Prader-Willi syndrome (PWS) to be presented at ENDO, focusing on BMI change, quality of life, and body fat percentage over six months in 17 patients.

• Phase III study for oral bivamelagon in HO expected to start before year-end, with positive phase II results showing up to 16.6% weight loss at highest dose over 12 months and strong patient persistence.

• RM-718, a weekly injectable, is in multiple ascending dose studies for HO and PWS, with phase I-B/Part C data expected in the second half of 2026; efficacy is anticipated to be comparable to other therapies.

• Product selection for indications will consider disease responsiveness, patient preference, and compliance, with a hypothesis that weekly injectables may be preferable in PWS due to behavioral factors.

Market opportunity and strategy

• HO diagnosis rates are higher than BBS, with patients more concentrated in endocrinology care, supporting a more favorable launch trajectory.

• Discontinuation in BBS is primarily due to hyperpigmentation; oral or MC1-sparing therapies could improve persistence and expand the market.

• Real-world BBS discontinuation rate is about 30% at one year, higher in the U.S. than in Europe; persistence improves after the first year.

• Identified HO patient pool in the U.S. is growing, with confidence in reaching a 10,000-patient prevalence as awareness and diagnosis improve through expanded field force and education.