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% growth compared to Q4 2021 [21] - VYONDYS 53 generated $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% growth compared to the same quarter in 2021 [21] Market Data and Key Metrics Changes - Ex U.S. net product revenue was $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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number : 001-14895 Sarepta Therapeutics, Inc. (Exact name of registrant as specified in its charter) | Delaware | 93-0797222 | | --- | --- | | ( ...

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]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-14895 SAREPTA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 93-0797222 (State or other ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q2 2022 Earnings Conference Call August 2, 2022 4:30 PM ET Company Participants Mary Jenkins - Senior Manager of Investor Relations Doug Ingram - President & Chief Executive Officer Louise Rodino-Klapac - Executive Vice President, Chief Scientific Officer and Head of R&D Dallan Murray - Senior Vice President & Chief Customer Officer Ian Estepan - Executive Vice President & Chief Financial Officer Gilmore O'Neill - Chief Medical Officer & Executive Vice President Conf ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 For the transition period from to Commission file number 001-14895 SAREPTA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (I.R.S. Employer Identification No.) Regi ...

| --- | --- | --- | --- | |-----------------------------------------------------------------|-------|--------------------------------------------------------|-------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | SRP-9001: New Clinical Data and Integrated Analysis | | | | | | | | | | DOUG INGRAM President and Chief Executive Officer | | | | | LOUISE RODINO-KLAPAC, PH.D. | | | | | Executive Vice President, Head of R&D, Chief Scientific Officer | | | | | July 6, 2022 8:30 a.m. ET | | | | | | | ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q1 2022 Earnings Conference Call May 4, 2022 4:30 PM ET Company Participants Mary Jenkins - Senior Manager of Investor Relations Dallan Murray - Senior Vice President & Chief Customer Officer Doug Ingram - President & Chief Executive Officer Ian Estepan - Executive Vice President & Chief Financial Officer Louise Rodino-Klapac - Executive Vice President, Chief Scientific Officer and Head of R&D Conference Call Participants Colin Bristow - UBS Brian Abrahams - RBC Capi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-14895 SAREPTA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 93-0797222 (State or other juri ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q4 2021 Earnings Conference Call March 1, 2022 4:30 PM ET Company Participants Mary Jenkins - Senior Manager of Investor Relations Doug Ingram - President & Chief Executive Officer Ian Estepan - Executive Vice President & Chief Financial Officer Dallan Murray - Senior Vice President & Chief Commercial Officer Gilmore O'Neill - Executive Vice President of R&D & Chief Medical Officer Louise Rodino-Klapac - Executive Vice President & Chief Scientific Officer Conference ...