Rhythm Pharmaceuticals, Inc. Q2 FY2025 earnings call

• BBS sales continued to grow, with 3 years post-launch and North American/international teams engaging in rare disease communities. - Phase III readouts of setmelanotide in acquired hypothalamic obesity and next-gen compounds set the stage for growth. - On track for U.S. and European regulatory filings for setmelanotide in HO in Q3. - International expansion of IMCIVREE with availability in over 20 countries and early access programs for HO in France/Italy. - Pending launch of IMCIVREE in hypothalamic obesity upon FDA approval. - Upcoming milestones include U.S. and EMEA filings for HO, Prader-Willi data by end of year, and enrollment timelines for various trials.

Global revenue for the second quarter was $48.5 million, an increase of 29% quarter-over-quarter. 66% of Q2 revenue ($32 million) was generated in the United States and 34% ($16.5 million) outside the United States. Quarter-over-quarter, the global number of patients on therapy increased by approximately 12%. BBS sales showed steady growth. International region saw IMCIVREE sales in approved indications (BBS, POMC/LEPR deficiencies) and paid early access for HO patients in France and Italy driving growth.

• Full-year non-GAAP operating expenses expected $285 million to $315 million, with non-GAAP SG&A expenses $135 million to $145 million and non-GAAP R&D expenses $150 million to $170 million. - Aim to disclose preliminary results from the Phase II Prader-Willi trial before end of 2025, complete enrollment of 718 weekly Phase II study in HO patients in 2026, release top-line data from Japanese cohort and EMANATE trial in Q1 2026, and initiate Phase III study with bivamelagon in acquired HO in 2026.

• Clinical trial outcomes may not meet expectations. - Regulatory hurdles in obtaining approvals for new indications. - Market adoption challenges for rare disease therapies. - Currency fluctuations impacting international revenue.

Q: Congrats on a good quarter. Could you frame the Prader-Willi study? Is it exploratory and what's needed for good data?

A: It's exploratory. Goal is to have north of 10 patients in open-label trial, looking for at least 5% decrease in BMI as a target. Patients on trial will continue beyond 6 months.

Q: Assuming positive Prader-Willi data, how about next-gen MC4Rs like bivamelagon?

A: Historically, subsequent development would be with next gen, but if data on setmelanotide is compelling, could go with setmelanotide initially, then decide on next gen.

Q: Will you provide updated HO prevalence estimates during the Commercial Day?

A: Not sure, but will reconfirm current understanding and share field team learnings.

Q: On 718 study, what's expected for patient mix and efficacy gauge?

A: Similar to other trials, 12 and older, expect data in similar range, but small numbers and short duration may cause noise. Key is right dose for weekly pharmacokinetic profile.

Q: On Prader-Willi, given VYKAT availability and single-center study, who's enrolled and impact on setmelanotide efficacy?

A: Inclusion criteria is Prader-Willi patients 6 and above, no exclusion for VYKAT use. Some patients on VYKAT will be enrolled, which may include more challenging patients like diabetics, making efficacy analysis more complex.