Sarepta Therapeutics, Inc. Q3 FY2024 earnings call

Management Statement and Operational Highlights

• Commercial Performance: ELEVIDYS launch remains strong with robust patient demand, ample site capacity, positive access/reimbursement trends, and consistent conversion rates. PMO franchise performance is solid with no significant cannibalization from ELEVIDYS.
• R&D Updates: Discontinued SRP-5051 development program after FDA dialogue and risk-benefit analysis. Progress in LGMD pipeline with SRP-9003, SRP-9004, and SRP-9005; SRP-9003 on track for BLA filing in 2025. Advancing ELEVIDYS clinical program with data presentations and post-marketing commitments. Progress with suspension manufacturing for ELEVIDYS. Discontinued PPMO franchise due to persistent hypomagnesemia and evolving Duchenne landscape.
• Financials: Total net product revenue was $430 million, growing 39% year-over-year. GAAP and non-GAAP profitable in Q3. Cash, cash equivalents, investments, and long-term restricted cash totaled approximately $1.4 billion as of September 30, 2024.

Segment Performance

• ELEVIDYS: Net product revenue was $181 million, growing nearly 50% quarter-over-quarter. Excluding royalty revenue from Roche's ex-US ELEVIDYS, it was $181 million; including royalty, it reached $190.5 million.
• PMO Franchise (EXONDYS 51, VYONDYS 53, AMONDYS 45): Achieved approximately $249 million in the third quarter. EXONDYS 51 led with $140.7 million, VYONDYS 53 at $32.2 million, and AMONDYS 45 at $75.9 million, showing about 4% growth from Q3 2023.

Guidance

• Q4 and 2025: Reiterated Q4 guidance in line with consensus. Long-term guidance of $3 billion by 2025, with two-thirds from ELEVIDYS and one-third from PMOs. Peak year sales are expected to be $5 billion or more.
• Pipeline: Anticipated BLA filing for SRP-9003 in 2025.

Risks

• PPMO Discontinuation: Risks associated with hypomagnesemia and long-term safety in chronic treatment setting led to discontinuing SRP-5051.
• Regulatory and Market Risks: Evolving Duchenne landscape and FDA requirements could impact pipeline progress and approvals.

Question and Answer

• Q: Clarify Q4 guidance after beating 3Q expectations A: Douglas Ingram stated they are comfortable with original guidance, confident in 2025 guidance of $3 billion, with two-thirds from ELEVIDYS.
• Q: Color on PPMO discontinuation, hypomagnesemia A: Louise Rodino-Klapac explained the discontinuation was due to FDA discussions, hypomagnesemia, and evolving Duchenne landscape; the entire PPMO franchise was halted.
• Q: Reconcile ELEVIDYS US patient count with sales A: Douglas Ingram noted focus on revenue metric, stating ELEVIDYS launch is going well with positive safety and efficacy.
• Q: Site capacity for ELEVIDYS A: Dallan Murray mentioned there are ~75 sites, with the vast majority dosing patients, and the focus remains on revenue guidance.
• Q: Manufacturing capacity for ELEVIDYS ex-US A: Douglas Ingram stated they are comfortable with manufacturing to meet demand, with suspension manufacturing in progress and a bridging study planned for 2025.