RHYTHM PHARMACEUTICALS, INC. Q1 FY2025 earnings call

Management Statement and Operational Highlights:

• David Meeker discussed the Phase 3 trial for HO on track for Q3 filing, in-person FDA meeting, and consistent response across age groups in the trial.
• Jennifer Lee highlighted strong demand for IMCIVREE in Q1, increased prescribers for BBS, and improved Medicaid access.
• Yann Mazabraud spoke about global growth in BBS patients and upcoming European conferences with data presentations.
• Financials: Q1 2025 net product revenues up 45% vs Q1 2024, R&D expenses down, SG&A expenses up, cash on hand $314.5 million with cash runway into 2027.

Segment Performance:

• IMCIVREE revenue in Q1 2025 was $37.7 million. US revenues were affected by an inventory swing, with the number of patients on reimbursed therapy increasing 14% globally. Outside the US, sequential revenue growth was strong at $3.1 million, driven by France, Germany, and Italy.
• For acquired hypothalamic obesity (HO), the Phase 3 trial is on track for Q3 filing with an in-person Type D meeting scheduled with the FDA. HO contribution to revenues from France and Italy continues to grow.

Guidance:

• Projected cash runway into 2027.
• OpEx guidance unchanged at $285 million to $315 million non-GAAP OpEx.
• Bivamelagon Phase 2 readout in Q3, HO study updates by end of year.

Risks:

• Inventory swings affecting US revenue.
• Compliance and discontinuation rates in patient populations.
• Uncertainties in FDA label approval and payer mix for HO.

Q: Jon Wolleben asked about non-responders and real-world persistence.

A: David Meeker discussed reasons for discontinuations, compliance, and expectations for lower discontinuations in HO.

Q: Derek Archila asked about RM-718 efficacy data and compliance.

A: David Meeker and Hunter Smith talked about RM-718 Phase 2 readout timing and compliance returning to normalized levels.

Q: Tazeen Ahmad asked about data updates at conferences.

A: David Meeker discussed importance of incremental patient data and small numbers in early updates.

Q: Seamus Fernandez asked about bivamelagon and PWS.

A: David Meeker talked about bivamelagon's efficacy threshold and PWS opportunity.

Q: Whitney Ijem asked about HO launch trajectory and label.

A: David Meeker discussed HO launch similarities/differences to BBS and label uncertainties.

Q: Dennis Ding asked about HO revenue ramp.

A: David Meeker discussed different ramp for HO due to concentrated endocrinology focus.

Q: Leland Gershell asked about inventory lumpiness and repeat prescribers.

A: Hunter Smith and Jennifer Lee talked about inventory timing and repeat prescribers in BBS vs HO.

Q: Ram Selvaraju asked about China strategy and HO commercial rollout.

A: David Meeker and Jennifer Lee discussed China strategy and marketing differences in HO vs BBS.

Q: Paul Matteis asked about bivamelagon safety and bar for Phase 2.

A: David Meeker talked about bivamelagon safety and context for efficacy bar in shorter trial