Sarepta Therapeutics(SRPT) - 2022 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Fourth quarter total revenue was $258.4 million, with net product revenue at$235.9 million, representing a 32% increase year-over-year [6] - Full year total revenue reached $933 million, and net product revenue was$843.8 million, indicating a 38% year-over-year increase [6] - The company has maintained a consistent 40% compounded annual growth rate over the last five years, driven entirely by serving the Duchenne community without price increases [6] Business Line Data and Key Metrics Changes - For the fourth quarter of 2022, net product revenue for EXONDYS 51 was $146 million, reflecting a 22$28.5 million in net product revenue for Q4 2022, marking a 15% increase year-over-year [21] - AMONDYS 45 saw net product revenue of $61.4 million in Q4 2022, representing nearly 80$96.3 million for the full year 2022, accounting for approximately 11% of overall net product revenues [42] - Fourth quarter 2022 ex U.S. sales were strong at $36.9 million, exceeding expectations for the quarter [43] Company Strategy and Development Direction - The company is focused on advancing the Biologics License Application (BLA) for gene therapy SRP-9001, which is seen as a pivotal moment for both the company and the Duchenne community [3][4] - Plans include addressing any remaining FDA questions, preparing for preapproval inspections, building inventory for launch, and ensuring launch readiness [5] - The company aims to commence studies to broaden the label for SRP-9001 and has initiated a pilot study for SRP-604 to treat limb-girdle muscular dystrophies [11][14] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the FDA's acceptance of the BLA for SRP-9001 via the Accelerated Approval pathway, highlighting the potential clinical benefits for patients [8] - The company is well-capitalized with approximately$2 billion in cash and equivalents, positioning it to execute its plans effectively [27] - Management emphasized the urgency of providing therapies to patients with Duchenne muscular dystrophy, indicating that families are unlikely to wait for additional data before seeking treatment [115] Other Important Information - The company reported a GAAP net loss of $109.2 million for Q4 2022, compared to a loss of$122 million in Q4 2021 [45] - R&D expenses for Q4 2022 were $213.8 million, an increase from$197.3 million in the same quarter of 2021, primarily due to higher upfront and milestone expenses [25] - SG&A expenses on a GAAP basis were approximately $120.5 million for Q4 2022, up from$78.1 million in Q4 2021, driven by increased stock-based compensation [26] Q&A Session Summary Question: What was the FDA's commentary on the mid-cycle review regarding the external control arm? - Management confirmed that the FDA saw no significant safety issues with the filing for SRP-9001 and did not identify any major deficiencies in the clinical data set [30][51] Question: How confident is the company regarding the manufacturing inspections? - Management expressed confidence in the timing of the preapproval inspections and stated that they are well-prepared for them [32][73] Question: What is the expected patient supply at launch? - The company aims to ensure that all patients can access the therapy without back orders, with plans to have around 50 sites operational at launch [35][39] Question: How does the company view the payer pathway for SRP-9001? - Management believes that their extensive experience with the payer community will facilitate productive discussions regarding access and reimbursement for SRP-9001 [103]