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    Leo Watson • Mizuho

    Leo Watson's questions to Sarepta Therapeutics Inc (SRPT) leadership

    Leo Watson's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q4 2024

    Question

    Leo Watson, on for Uy Ear at Mizuho, asked for key learnings from the pre-BLA meeting with the FDA for SRP-9003, how they apply to other LGMD programs, and how FDA interactions might change going forward.

    Answer

    Chief Scientific Officer Dr. Louise Rodino-Klapac described the pre-BLA meeting as 'extremely positive,' with the FDA endorsing their clinical plan, CMC strategy, and the use of beta-sarcoglycan as a surrogate biomarker for accelerated approval. CEO Douglas Ingram credited the FDA, particularly leaders Dr. Marks and Dr. Verdun, for modernizing the approach to ultra-rare diseases, which bodes well for the entire LGMD platform and future programs.

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    Leo Watson's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q3 2024

    Question

    Leo Watson from Mizuho inquired about the competitive dynamics in the exon-skipping market following competitor data and the PPMO discontinuation, and also asked about long-term PMO cannibalization from ELEVIDYS beyond 2025.

    Answer

    President and CEO Doug Ingram stated that while some cannibalization is modeled post-2025, the PMO franchise is expected to be very durable. Regarding competition, he noted that no competitor is close, and the high bar for both efficacy and safety, combined with a long development path, makes the PMO franchise highly defensible.

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