• Fourth Quarter Net Product Revenues: $638 million, a 75% increase over the same quarter prior year.
• Full-Year Net Product Revenues: $1.8 billion, representing a 56% year-over-year growth.
• ELEVIDYS Fourth Quarter Sales: $384 million, a 112% increase over the prior sequential quarter.
• ELEVIDYS Full-Year Sales: Over $1 billion since initial approval in 2023.
• PMO Fourth Quarter Revenues: $254 million.
• PMO Full-Year Revenues: $967 million.
• GAAP Net Income for Fourth Quarter: $159 million, or $1.65 per basic share and $1.50 per diluted share.
• Non-GAAP Net Income for Fourth Quarter: $206 million, or $1.90 per diluted share.
• Cash and Cash Equivalents: Approximately $1.5 billion as of December 31, 2024.
• 2025 Revenue Guidance: Between $2.9 billion and $3.1 billion, representing 70% growth over 2024.

• Warning! GuruFocus has detected 5 Warning Sign with SRPT.

Release Date: February 26, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Sarepta Therapeutics Inc (NASDAQ:SRPT) achieved all goals laid out in Project Moonshot, including obtaining approval for the broadest possible label for ELEVIDYS.
• The company reported exceptionally strong financial performance with fourth-quarter net product revenues of $638 million, a 75% increase over the same quarter the previous year.
• ELEVIDYS had the most successful gene therapy launch in history, with fourth-quarter sales of $384 million, representing a 112% increase over the prior sequential quarter.
• Sarepta Therapeutics Inc (NASDAQ:SRPT) became sustainably profitable and cash flow positive in 2024, projecting over $16 billion in cumulative operating income by 2030.
• The company has a robust pipeline with multiple potential blockbuster siRNA launches expected before the end of the decade, supported by the Arrowhead collaboration.

Negative Points

• The launch of ELEVIDYS, while successful, faces a natural cadence issue, taking about four months to get patients on therapy due to various administrative and logistical steps.
• Despite the success of ELEVIDYS, it has only treated less than 5% of the on-label addressable patient population, indicating a long road ahead for market penetration.
• The company faces challenges in expanding manufacturing capabilities, with plans to move to suspension manufacturing to improve costs and scalability.
• There is ongoing litigation and increased expenses related to professional services, impacting SG&A costs.
• The company acknowledges the risks and uncertainties associated with forward-looking statements, which could materially affect business operations and stock trading prices.

Q & A Highlights

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.