Rhythm Pharmaceuticals' Q2 2025: Navigating Key Contradictions in Trial Design, Stock Compensation, and Market Dynamics

Prader-Willi syndrome trial design and goals, stock compensation guidance, patient enrollment and study design for the Prader-Willi study, efficacy expectations for RM-718, and U.S. market dynamics and off-label use are the key contradictions discussed in Rhythm Pharmaceuticals' latest 2025Q2 earnings call.



Revenue Growth and International Expansion:
- Rhythm Pharmaceuticals reported global revenue of $48.5 million for Q2, up 29% quarter-over-quarter, with 66% of revenue generated in the U.S. and 34% outside the U.S.
- The growth was driven by increased patient numbers, particularly in international markets, and a 12% increase in the number of reimbursed patients globally.

U.S. Revenue and Patient Growth:
- U.S. revenue increased by $7.6 million or 31% over the prior quarter, with the number of reimbursed patients on therapy growing at mid-single-digit percentage rates.
- The increase was due to improved demand and inventory factors, with a positive effect from product shipped exceeding product dispensed to patients.

International Market Growth:
- International region revenue saw approximately 24% growth, driven by increased sales in approved indications like BBS and POMC LEPR deficiencies, and early access programs for hypothalamic obesity in France and Italy.
- Growth was attributed to reimbursed hypothalamic obesity patients accounting for a meaningful percentage of total international patients and new patient starts in countries like Poland and the Czech Republic.

Clinical Trial and Regulatory Milestones:
- The company is on track to complete U.S. and European regulatory filings for setmelanotide in acquired hypothalamic obesity in Q3, with plans to update upon acceptance of filings.
- The progress was driven by productive meetings with regulatory bodies and positive Phase III trial results in acquired hypothalamic obesity.