Rhythm Pharmaceuticals, Inc. Q3 FY2025 earnings call

• Rhythm delivered strong growth in Q3 2025, preparing to launch IMCIVREE in acquired hypothalamic obesity pending FDA approval. - Finished 2025 strong with robust Phase III data for setmelanotide and HO, Phase II data for bivamelagon, and a $189 million equity offering in July. - Global IMCIVREE revenue grew, with $51.3 million in sales in Q3, driven by BBS, and 10% growth in the number of patients on reimbursed therapy. - Regulatory progress: FDA and EMEA accepted regulatory filings for HO, with PDUFA date in December 20 and potential European approval in H2 2026. - German study showed setmelanotide improved steatotic liver disease and kidney function in BBS patients. - Preparations for HO launch: Hired experienced professionals, engaged with customers, analyzed claims data to target physicians, and focused on educating physicians and payers. - International efforts: Reached reimbursement pricing agreement in France for IMCIVREE, expanded EMA submission for HO, and built local team in Japan with plans for top-line data in Q1 2026 and potential launches in 2027.

Global revenue for Q3 2025 was $51.3 million, a sequential 6% increase from Q2 2025. $38.2 million (74%) of Q3 net revenue was generated in the United States, and $13.1 million (26%) was generated outside the United States. The number of patients on reimbursed therapy increased by 10% globally during the quarter. U.S. revenue was buoyed by a high single-digit percentage increase in the number of reimbursed patients, with approximately $3.7 million of the quarter-over-quarter revenue increase driven by more IMCIVREE dispensed to patients. Outside the U.S., patients on reimbursed therapy increased at a low double-digit percentage, but international revenue decreased by $3.4 million quarter-over-quarter due to variability in ordering patterns for named patient sales in certain distributor markets.

• Tightened full-year 2025 operating expense guidance, anticipating $295 million to $315 million in non-GAAP operating expenses, with non-GAAP R&D expenses of $150 million to $165 million and non-GAAP SG&A expenses of $145 million to $150 million. - Upcoming milestones include PDUFA date for HO in December, preliminary results from Prader-Willi syndrome Phase II trial by end of 2025, completion of HO Phase II study enrollment in Q1 2026, top-line data from Japanese cohort of HO Phase III trial in Q1 2026, top-line data from EMANATE trial in Q1 2026, completion of congenital HO trial enrollment in H1 2026, and initiation of bivamelagon Phase III trial in acquired HO next year.

Q: Mike Ulz from Morgan Stanley asked about bivamelagon's Phase III HO study trial design and FDA feedback.

A: David Meeker responded that the HO trial will be a double-blind randomized controlled trial, they'll discuss with regulators the duration of the double-blind period, expecting a full year of data, and anticipate a post-Phase II meeting with the FDA in Q1 2026 for feedback.

Q: Phil Nadeau from TD Cowen asked about PWS efficacy endpoints.

A: David Meeker said the Prader-Willi Phase II trial will present patient-by-patient data, it's a judgment call, primary endpoint is BMI percent change, and improvement in hyperphagia is expected if BMI changes due to the drug's mechanism.

Q: Derek Archila from Wells Fargo asked about the variability trial extension for IMCIVREE and FDA discussions on HO indication statement.

A: David Meeker said the trial extension was housekeeping, allowing patients to continue beyond 6 months and adding a site if needed; on HO indication, regulatory interactions were routine with no specific indication statement discussions yet.

Q: Angela Qian from Canaccord Genuity asked about HO launch payer conversations and gross to net.

A: David Meeker and Jennifer Chien responded that they're engaging with payers, patients in trials stay on drug, teams work through reimbursement post-approval, and gross to net depends on Medicare/commercial mix and Medicaid share.

Q: Faisal Khurshid from Leerink Partners asked about HO launch curve relative to other launches.

A: Jennifer Chien said HO has a solid ground based on BBS launch, right team in place, but ramp may be affected by patient distribution and diagnosis process; David Meeker added PWS is different due to patient care in specialized centers vs HO patients in community/local endocrinologists.

Q: Erik Wong from Goldman Sachs asked about HO launch reimbursement process and gross to net relative to BBS.

A: David Meeker and Jennifer Chien discussed gross to net depending on Medicare/commercial mix and Medicaid share, and teams engage with payers pre-approval to educate on HO and work through reimbursement post-approval.

Q: Julian Pino from Stifel asked about Prader-Willi trial amendment and additive benefit of setmelanotide with VCA.

A: David Meeker said trial amendment was housekeeping, and setmelanotide and VCA are complementary with no overlapping toxicity.

Q: Evan Wang from Guggenheim Securities asked about Prader-Willi trial participation and international HO launch preparations.

A: David Meeker said Prader-Willi trial data will be out by end of 2025; Yann Mazabraud said international HO launch in Europe is expected in 2027, following BBS launch sequence, engaging with payers, and adding staff as needed.

Q: Georgia Ban from Jefferies asked about Prader-Willi initial data disclosure and Phase III decision.

A: David Meeker said initial data disclosure in December may lead to a go/no-go decision or waiting for longer follow-up.

Q: Raghuram Selvaraju from H.C. Wainwright asked about German study's impact on other indications.

A: David Meeker said the study showed setmelanotide improved liver function in BBS patients, highlighting more to learn about the mechanism beyond BMI change.

Q: Jonathan Wolleben from Citizens JMP Securities asked about the 2,000 potential HO patients.

A: Jennifer Chien said the 2,000 patients were identified through field organization discussions and physician interactions, with the process ongoing to get patients to diagnosis.