Rhythm Pharmaceuticals (RYTM) 25th Annual Needham Virtual Healthcare Conference summary

Strategic priorities and product launches

• Launch of IMCIVREE for acquired hypothalamic obesity (HO) in the U.S. is the top priority, with a significant sales force expansion and patient support network buildout.

• Expansion plans for IMCIVREE in Europe and Japan are underway, with a positive CHMP opinion in Q2 and a targeted European launch in 2027.

• Initiation of a phase III study for bivamelagon in AHO and expected data for RM-718 in HO later this year.

• Ongoing development in Prader-Willi syndrome (PWS) with a mid-year data update planned and a phase II study for RM-718 in PWS patients.

• Continued focus on genetic disorders within the MC4R franchise, despite setbacks in the MASH study.

Commercial opportunity and market dynamics

• HO prevalence in the U.S. is estimated at over 10,000 patients, with a higher baseline diagnosis rate compared to other rare diseases.

• Early launch reception for IMCIVREE in HO is positive, with efforts focused on endocrinologist education and patient identification.

• Comparison with BBS launch highlights faster uptake potential in HO due to higher diagnosis rates and more concentrated specialty care.

• Revenue reporting will not be split by indication, but HO-specific script data and launch metrics will be provided.

• European pricing is lower than the U.S., but all patients are reimbursed, unlike the U.S. where some receive free drug.

Clinical development and data readouts

• Mid-year PWS data readout will include 17 patients with six months of therapy, focusing on BMI, hyperphagia scores, and DEXA data.

• A 5% placebo-adjusted BMI reduction over 52 weeks is considered clinically meaningful for PWS.

• Phase III PWS trial preparation is underway, with endpoints likely to include BMI change, HQ-CT improvement, and fat mass change.

• RM-718, a weekly injectable, may offer advantages over setmelanotide due to MC1R sparing and improved administration.