Sarepta Therapeutics(SRPT) - 2023 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Total revenue for Q1 2023 was $253.5 million, an increase from $210.8 million in Q1 2022, reflecting a growth of $42.7 million [52] - Net product revenue was $231.5 million, representing a 23% increase compared to the same quarter of the previous year [8][52] - The company reported a GAAP net loss of $516.8 million for Q1 2023, primarily due to a non-cash loss on debt extinguishment of $387.3 million [35][56] - Non-GAAP net loss was $85.5 million, compared to a loss of $48.6 million in Q1 2022 [36] Business Line Data and Key Metrics Changes - EXONDYS 51 generated $132.6 million in revenue, up over 13% from Q1 2022 [47] - VYONDYS 53 sales were $33 million, growing approximately 18% year-over-year [47] - AMONDYS 45 sales totaled $65.9 million, representing more than 50% growth compared to Q1 2022 [47] Market Data and Key Metrics Changes - Total ex-U.S. net product revenue was approximately $31 million, reflecting a decrease from the previous quarter, which was anticipated [46] - The U.S. market has reached a mature phase for all three products, leading to expectations of more modest growth in new patient starts [30] Company Strategy and Development Direction - The company is preparing for the potential launch of SRP-9001, a gene therapy for Duchenne muscular dystrophy, with a PDUFA date set for May 29, 2023 [9][22] - The focus is on expanding the label for SRP-9001 to include non-ambulatory patients and addressing preexisting neutralizing antibodies [66][83] - The company aims to ensure accessibility and reimbursement for SRP-9001 through engagement with payers [32][49] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming FDA advisory panel for SRP-9001, emphasizing the importance of the evidence supporting the therapy [7][9] - The company is committed to serving the Duchenne community and believes that SRP-9001 could significantly improve patient outcomes [9][14] - Management acknowledged the complexities of access and reimbursement for rare disease therapies but remains optimistic about payer engagement [70][125] Other Important Information - The company has completed all FDA inspections, including GMP and GCP inspections, and is ready for the potential launch of SRP-9001 [23][117] - The company has approximately $1.9 billion in cash and investments as of March 31, 2023, positioning it well for future operations [57] Q&A Session Summary Question: What is your estimate of patient numbers for the initial indication in the U.S.? - Management indicated that the addressable patient population is estimated to be between 10,000 to 15,000 patients in the U.S., with a focus on ambulatory patients [83] Question: Can you talk about your manufacturing capacity and any major expansions needed? - Management confirmed that no major capital expenditures or expansions are required to launch SRP-9001, and they are confident in their ability to meet demand [83] Question: How are preliminary conversations with payers going regarding SRP-9001? - Management reported constructive dialogues with payers, emphasizing that they have been engaging with them for several years and are optimistic about access for patients [87][88] Question: Will there be a difference in ramp for patients naive to RNA therapies versus those on PMOs? - Management does not anticipate a difference in ramp-up for patients, stating that the therapy will be important for all eligible patients [92] Question: What is the company's plan for capital deployment post-approval? - Management plans to focus on R&D while also aiming for profitability, with pricing for SRP-9001 being determined based on its value to patients [127]