Sarepta Therapeutics(SRPT) - 2022 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Total revenue for Q3 2022 was approximately $230.3 million, with net product revenue of$ 207.8 million, representing nearly 25% growth compared to the same quarter last year [7][66] - The company maintained its full-year total revenue guidance of between $905 million and$ 920 million and net product revenue guidance of between $825 million and$ 840 million [8] - On a GAAP basis, the company reported a net loss of $257.7 million for Q3 2022, compared to a net loss of$ 48.1 million for the same period in 2021 [69] Business Line Data and Key Metrics Changes - Net product revenue for EXONDYS 51 was $122 million, for AMONDYS 45 was$ 55 million, and for VYONDYS 53 was $31 million in Q3 2022 [54][67] - EXONDYS 51 experienced nearly 6% growth year-over-year, while AMONDYS 45 and VYONDYS 53 grew significantly, with AMONDYS 45 impacted by a pull-forward effect [58][59] - The RNA-based PMO franchise continues to show strong performance, contributing to the overall revenue growth [54] Market Data and Key Metrics Changes - The company noted that ex-U.S. sales growth for EXONDYS 51 has become an important contributor to net product revenue growth, despite introducing some quarter-to-quarter fluctuations [56] - The company is focused on expanding access to the Duchenne population amenable to exon 45 skipping, indicating a strategic approach to market penetration [55] Company Strategy and Development Direction - The company is preparing for the launch of SRP-9001, a gene therapy for Duchenne muscular dystrophy, with plans for a mid-2023 launch following a BLA submission [9][10] - The company aims to expand the addressable population for SRP-9001 and is working on studies to include non-ambulatory patients and those with early exon mutations [16][17] - The company is committed to advancing its gene therapy platform and has announced collaborations to enhance its genetic medicine delivery systems [33] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency of bringing therapies to patients with Duchenne muscular dystrophy, highlighting the critical need for accelerated approval pathways [11][12] - The management expressed confidence in the upcoming BLA submission and the potential for priority review by the FDA, with a PDUFA date anticipated in May 2023 [79][82] - The company is well-capitalized with approximately$ 2.1 billion in cash and investments as of September 30, 2022, to support the launch of SRP-9001 if approved [77] Other Important Information - The company announced a management transition with the retirement of Bill Ciambrone, who played a key role in the development and manufacturing processes for SRP-9001 [23][29] - The company presented significant real-world evidence at the World Muscle Society Conference, demonstrating the survival benefits associated with its therapies [35][36] Q&A Session Summary Question: Concerns about SRP-9001 review process - Management confirmed that they have requested a priority review for the BLA submission and are planning for a May 2023 PDUFA date, with no current indications from the FDA suggesting otherwise [79] Question: Manufacturing commitments for SRP-9001 - Management stated they are in good shape regarding manufacturing commitments and have completed significant CMC work ahead of the EMBARK study [82] Question: Potential impact of EMBARK study results - Management expressed confidence in the EMBARK study's design and power, indicating they would consider the totality of evidence if the study did not meet statistical significance [99][100] Question: Real-world data and primary endpoint timing - Management addressed concerns about variability in patient responses and reiterated confidence in the study design and powering for the EMBARK trial [104][106]