Release Date: February 26, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q: Can you provide more details on the cadence of patient onboarding in Q1 and how it impacts your 2025 guidance? A: Douglas Ingram, President, CEO, and Director, emphasized confidence in the 2025 guidance, noting a strong track record of meeting or exceeding guidance. He mentioned that growth is expected quarter over quarter, with a projected total revenue of $3 billion, driven by ELEVIDYS and PMOs.
Q: What is the prevalence of limb-girdle muscular dystrophy type 2E, and how should we think about its revenue contribution? A: Douglas Ingram explained that type 2E is an ultra-rare disease, with all three sarcoglycan launches collectively representing about 25% of the Duchenne ELEVIDYS opportunity. Louise Rodino-Klapac, EVP, Chief Scientific Officer, noted a generally even split between ambulatory and non-ambulatory patients.
Q: What data will be shared later this year for FSHD and DM1, and how significant will it be? A: Louise Rodino-Klapac stated that both FSHD1 and DM1 will have single-ascending dose (SAD) data focusing on safety and early biomarker efficacy. Douglas Ingram highlighted the potential to show direct knockdown of DUX4 in FSHD, which would be a significant proof of biology.
Q: Can you discuss the potential for accelerated approval based on splicing biomarkers for FSHD and DM1? A: Louise Rodino-Klapac mentioned that FSHD has a unique opportunity with an assay to quantify DUX4 knockdown, which could be a potential biomarker. The Phase 1 studies aim to evaluate multiple biomarkers and functional outcomes to determine the best path forward.
Q: How is Sarepta modeling the impact of competitive gene therapies in DMD, and what are the expectations for the 2E program? A: Douglas Ingram noted that it's too early to model competition from early-stage therapies. For the 2E program, consistent expression levels with previous data are expected, and the regulatory submission will depend on achieving those levels and ensuring safety.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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