RHYTHM PHARMACEUTICALS, INC. Q4 FY2024 earnings call

• Preannounced strong Q4 earnings and raised $75 million in gross proceeds from ATM program. - Completed enrollment in Phase II daily oral bivamelagon study and 718 program enrollment beginning. - Phase III trial of setmelanotide in acquired HO to read out in second quarter. - FDA approved label expansion for IMCIVREE to include children as young as 2 years old. - International expansion with access in over 15 countries, new partnership in Turkey, and Japan operations building. - Q4 revenue from IMCIVREE global sales was $41.8 million, with U.S. revenue accounting for 76% of Q4 revenue.

Global sales of IMCIVREE in Q4 2024 were $41.8 million. U.S. revenue in Q4 was $31.7 million, accounting for 76% of the quarter's revenue. For the year, U.S. sales made up 74% of revenue. Cost of sales for Q4 was $3.8 million (9% of net product revenue). R&D expenses were $41.2 million in Q4 2024, up from $29.9 million in Q4 2023. SG&A expenses were $38.1 million in Q4 2024, up from $32.4 million in Q4 2023. Cash on hand ended 2024 at $320.6 million.

• For 2025, anticipate non-GAAP OpEx of $285 million to $315 million, including non-GAAP SG&A of $135 million to $145 million and non-GAAP R&D of $150 million to $170 million. - Cash on hand of $320.6 million expected to fund operations into 2027, past key milestones. - No revenue guidance provided, but guidance on non-GAAP operating expenses.

• Uncertainty in clinical trial outcomes and regulatory approvals. - Market competition and challenges in reimbursement and uptake of products. - Potential impact of external factors on financial performance and operational plans.

Q: Could you comment on whether you're going to share the SAD MAD portion of the RM-718 study separately?

A: Likely won't present it as an independent breakout, will do in concert with Part C of the trial.

Q: Interested to get a sense of uptake in HO population. How identifiable is it compared to BBS?

A: HO population has a significant unmet need, concentrated in endocrinology call points but has an undiagnosed pool; uptake will face reimbursement challenges but initial payer feedback is positive.

Q: What does management think well is in terms of percent change in BMI for HO Phase III trial?

A: 5% is regulatory hurdle, would be disappointed with less than 10%, but comparisons to GLP-1 trials are challenging due to different biology.

Q: Is there likely to be a modestly down quarter-over-quarter in Q1 due to inventory destocking?

A: Anticipate destocking in Q1, Q1 can be 'lunky' for companies like Rhythm.

Q: Comment on impact of label expansion for IMCIVREE on U.S. market dynamics.

A: Label expansion helps differentiate patient population, but impact on revenues not significant yet; supports long-term benefit for patients.

Q: How is the Trispera partnership likely to inform future ex-U.S. BD activities?

A: Partnership in Turkey is country-specific but indicative of country-by-country approach to distributorships, with success in other partnerships like Israel and Middle East.