Sarepta Therapeutics, Inc. Q4 FY2024 earnings call

• Project Moonshot achieved in 2024: Obtained broad label for ELEVIDYS, LGMD franchise moving towards approvals, ELEVIDYS launched broadly with > all other in vivo gene therapies combined, became profitable and cash flow positive in 2024, secured Arrowhead collaboration adding siRNA platform. • 2024 performance: Exceptional Q4 and full year with net product revenue up, overperformed guidance. • ELEVIDYS launch: Q4 sales exceeded guidance, treated <5% of on-label addressable patient population, EMBARK study Part 2 data supports durability. • LGMD programs: Completed dosing for SRP-9003, BLA submission later in 2024; ongoing dosing for SRP-9004; initiated registration study for SRP-9005. • Arrowhead collaboration: Added siRNA platform with multiple clinical, preclinical, research programs; ARO-DUX4 and ARO-DM1 readouts expected in 2025. • R&D and tech ops: Milestones in 2025, planning suspension manufacturing, ENVISION study for ELEVIDYS in non-ambulatory.

For the fourth quarter, net product revenue was $638 million, growing 75% over the prior year. Full year net product revenue was $1.8 billion, up 56% year-over-year. ELEVIDYS had Q4 sales of $384 million, a 112% increase over the prior sequential quarter, representing less than 5% of the on-label addressable opportunity. The three PMOs (EXONDYS 51, AMONDYS 45, VYONDYS 53) achieved $254 million in the fourth quarter and $967 million for the full year. Individual PMO net product revenues in Q4 were $137.6 million for EXONDYS 51, $40.2 million for VYONDYS 53, and $76.2 million for AMONDYS 45.

• 2025 net product revenue guidance $2.9 billion to $3.1 billion, ~70% growth y-o-y, with ELEVIDYS ~160% growth y-o-y. • By 2030, projected $16 billion cumulative operating income and $13 billion free cash flow after Arrowhead investment. • 2025 R&D and SG&A expenses expected to be $1.2 billion to $1.3 billion, mostly from Arrowhead programs.

• Risks associated with forward-looking statements, actual results differing from projections. • Regulatory risks related to approvals and compliance. • Manufacturing risks in scaling production. • Market competition risks for gene therapy and siRNA programs. • Payer access and reimbursement challenges. • Clinical trial outcomes risks for pipeline programs.

Q: Tazeen Ahmad asked about 1Q cadence of patient onboarding.

A: Doug Ingram stated confidence in 2025 guidance, growth quarter-over-quarter as launch ramps up.

Q: Ellie Merle inquired about LGMD 2E prevalence and ambulatory/non-ambulatory split.

A: Doug Ingram said LGMD 2E is rare, ~25% of Duchenne ELEVIDYS opportunity; Louise Rodino-Klapac noted generally 50-50 split.

Q: Gena Wang asked about FSHD and DM1 data.

A: Doug Ingram and Louise Rodino-Klapac discussed SAD data, DUX4 assay for FSHD, and biomarkers for DM1.

Q: Brian Skorney asked about rate-limiting steps to ELEVIDYS revenue recognition.

A: Dallan Murray said it's a game of time with robust demand, site capacity, access, and reimbursement, with consistent conversion rates.

Q: Ry Forseth asked about competitive gene therapies and 2E program vector genomes.

A: Doug Ingram said no current competitive next-gen therapies, confident in expression and safety for 2E program; Dallan Murray emphasized incident population size.