Rhythm Pharmaceuticals (RYTM) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company overview and product focus

• Focused on therapies targeting the melanocortin-4 (MC4) pathway, crucial for hunger and energy balance.

• Initial approvals for setmelanotide (Imcivree) in ultra-rare POMC and leptin receptor deficiencies in 2020, followed by Bardet-Biedl syndrome (BBS) in 2022.

• Hypothalamic obesity (HO) program gained attention after positive phase II data in 2022.

Clinical development and regulatory progress

• Phase III HO trial fully enrolled with 131 patients; readout expected in first half of 2025.

• Positive regulatory interactions in Japan led to trial expansion without delaying US/EU filings.

• Phase III trial design includes broad inclusion criteria and allows stable GLP use; over 10% of patients are on GLPs.

Efficacy and rationale for MC4 targeting

• Setmelanotide showed consistent, robust BMI reduction in HO: 14% at 16 weeks, 25% at 12 months.

• MC4 pathway deficit is fundamental in HO; GLP-1 agonists show limited, short-lived benefit in this population.