Sarepta Therapeutics, Inc. Q4 FY2025 earnings call

The company entered 2026 in a solid financial position with a large cash balance. The PMO therapies have stable revenues. ELEVIDYS had uncertainties resolved in 2025 with an updated label. There are exciting siRNA pipeline programs including neuromuscular, pulmonary, and CNS programs. There are new commercial initiatives for ELEVIDYS to address information deficits, update messaging with data, and expand field teams. The siRNA programs for DM1, FSHD, and Huntington's disease are advancing with expected updates this year.

For 2025, total revenues were $2.2 billion, with $1.86 billion in net product revenue and $334 million in collaboration, contract manufacturing and royalty revenue from Roche. Net product revenue for 2025 was $1.86 billion, consisting of $966 million from the PMO franchise and $899 million for ELEVIDYS. In the fourth quarter, PMO net product revenues totaled $259 million, with EXONDYS 51 at $148 million, VYONDYS 53 at $34 million, and AMONDYS 45 at $77 million. Fourth quarter ELEVIDYS revenues were $110 million. For 2026, the net product revenue guidance for approved therapies is in the range of $1.2 billion to $1.4 billion. The first quarter revenue is expected to be flat to perhaps down 15% from the prior quarter. Additionally, in 2026, it is anticipated to have a $40 million milestone payment from Roche in Q1 and $325 million in noncash collaboration revenue tied to Roche declining an option, with total collaboration, contract manufacturing and royalty revenues expected to be between $450 million and $550 million.

The net product revenue guidance for 2026 approved therapies is $1.2 billion to $1.4 billion. The first quarter revenue is expected to be flat to down 15% from the prior quarter. There is a $40 million milestone payment from Roche expected in Q1 2026 and $325 million in noncash collaboration revenue tied to Roche's option decline. Total collaboration, contract manufacturing and royalty revenues in 2026 are expected $450 million to $550 million. The 2026 non-GAAP expense outlook is reaffirmed as $800 million to $900 million.

Q: Anupam Rama inquired about internal versus external candidates for CEO succession.

A: Doug Ingram stated that the Board is looking at both internal and external candidates and will be thoughtful in choosing someone who can drive the plans and lead the team.

Q: Brian Abrahams asked about key metrics for ELEVIDYS initial receptivity.

A: Patrick Moss mentioned that efforts take time, promotional efforts are rooted in data, and a small number of patients can impact performance.

Q: Tejas Wein asked about siRNA readouts benchmarks.

A: Louise Rodino-Klapac said they are looking for muscle concentration and consistency with preclinical data.

Q: Lin Tsai asked about siRNA pivotal studies and accelerated approval.

A: Louise Rodino-Klapac said they are working towards Phase III and taking the fastest approach regardless of the approval pathway.

Q: Joseph Schwartz asked about clinicians risk stratifying ELEVIDYS patients.

A: Patrick Moss said each patient is unique and physicians consider various factors.

Q: John Kim asked about the Japanese market for ELEVIDYS.

A: Patrick Moss said to turn to Roche for details.

Q: Tommie Reerink asked about ELEVIDYS quarterly dynamics.

A: Patrick Moss said quarter-to-quarter dynamics are noisy and they focus on long-term performance.

Q: Tazeen Ahmad asked about PMO use after ELEVIDYS.

A: Patrick Moss said few patients go back on PMO and it is not materially in the forecast.

Q: Mitchell Kapoor asked about ELEVIDYS early trends.

A: Patrick Moss said there are early green shoots but the time from enrollment to infusion is long.

Q: Michael Ulz asked about DM1 and FSHD PD data.

A: Louise Rodino-Klapac said for DM1 there will be early DMPK knockdown data and for FSHD there will be DUX4 target gene correction data.

Q: Biren Amin asked about Cohort 8 and ENVISION.

A: Louise Rodino-Klapac said they are collecting liver parameters for Cohort 8 and expect Cohort 8 data to inform restarting ENVISION.

Q: Konstantinos Biliouris asked about DM1 splice correction.

A: Louise Rodino-Klapac said they are early on analyzing data to inform Phase III.

Q: Joshua Fleishman asked about ENDEAVOR Cohort 8 recruitment.

A: Louise Rodino-Klapac said there is significant interest and they are in the process of screening.

Q: William Pickering asked about DM1 disclosure and splicing follow-up.

A: Louise Rodino-Klapac said they have SAD cohort data, early MAD data, and splicing data will be shared in the second half.

Q: Luke asked about DM1 splice correction bar.

A: Louise Rodino-Klapac said they are early on analyzing to inform Phase III.

Q: Gil Blum asked about the LGMD program and sirolimus.

A: Louise Rodino-Klapac said they need to discuss with the agency and will have a conversation after Cohort 8 data.

Q: Samantha Corwin asked about PMO guidance and ENDEAVOR Cohort 8 enrollment.

A: Douglas Ingram said there is competition with Dyne's exon 51 therapy and Louise Rodino-Klapac said they are in the process of screening.

Q: Sean asked about DMD newborn screening impact.

A: Douglas Ingram said it takes time and is not near-term impact, and they need to adapt ELEVIDYS label.

Q: Brandon asked about Japan launch signs.

A: Douglas Ingram said Roche is best to provide details and he is excited about the launch