Sarepta Therapeutics : SRPT JPM deck 2023 FINAL

DOUG INGRAM

President and CEO

Sarepta Therapeutics, Inc. (NASDAQ:SRPT)

JPMorgan Healthcare Conference

San Francisco, California

JANUARY 9, 2023

FORWARD-LOOKING STATEMENTS

This presentation contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to future operations, financial performance and projections; our current guidance for 2023 for our three currently approved therapies of more than $925 million in net revenue; our opportunities in the rare disease space; potential solutions and market opportunities with our RNA technologies, gene therapy and gene editing; the potential benefits of our technologies and scientific approaches; the potential benefits of PMO and PPMO; the potentially transformative benefits of SRP-9001, including SRP-9001's potential to transform the trajectory of Duchenne, the potential restoration of DAPC, reduced CK and improved histopathology, and the potential of improved benefit received from SRP-9001 over time; our belief that the 9001-dytsrophin protein is reasonably likely to predict clinical benefit; our belief that our internal gene therapy capabilities complimented by partnerships will meet demand to launch SRP-9001, if approved, and drive competitive costs with continued improvements to drive upside; our belief that the transformative one-time therapy, SRP-9001, will cost the system less than the value it will provide to the Duchenne community; the potential of gene therapy's applicability across disease; the potential of our collaborations and partnerships; and expected milestones and plans, including our belief that we may receive an advisory Committee meeting for SRP-9001, launching SRP-9001 in the middle of 2023, if SRP-9001 is approved, having a readout of our confirmatory trial for SRP-9001 at the end of the year, expanding the available label of SRP-9001 after additional studies by 2024, publishing our perspective on the holistic approach to value innovative one-time therapies like SRP-9001, our expectation that we will have approximately 30 clinical trials ongoing by the end of 2023, continuing to build our pipeline, and our expectations related to our future financial performance, including if SRP-9001 is approved, our forecasted peek year SRP-9001 net product revenue will be nearly $4 billion, tracking to nearly $5 billion in total net product venue, by 2026, if we meet our strategic plan goals, including if SRP-9001 is approved, we will be cash positive and profitable by next year, and updating our guidance to include SRP-9001 net sales for 2023, if SRP-9001 is approved.

These forward‐looking statements involve risks and uncertainties, many of which are beyond our control and are based on our current beliefs, expectations and assumptions regarding our business. Actual results and financial condition could materially differ from those stated or implied by these forward‐looking statements as a result of such risks and uncertainties, and such risks and uncertainties could materially and adversely affect our business, results of operations and trading price. Potential known risk factors include, among others, the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to EXONDYS 51, VYONDY 53 and AMONDYS 45 in a timely manner or at all; our data for our different programs, including PPMO and gene therapy-based product candidates, may not be sufficient for obtaining regulatory approval; our product candidates, including those with strategic partners, may not result in viable treatments suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the impact of the COVID-19 pandemic; the expected benefits and opportunities related to our agreements with our strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreements, challenges and uncertainties inherent in product research and development and manufacturing limitations; if the actual number of patients living with Duchenne and LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; our dependence on our manufacturers to fulfill our needs for our clinical trials and commercial supply, including any failure on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand, may impair the availability of products to successfully support various programs, including research and development and the potential commercialization of our gene therapy product candidates; we may not be able to successfully scale up manufacturing of our product candidates in sufficient quality and quantity or within sufficient timelines; current reimbursement models may not accommodate the unique factors of our gene therapy product candidates; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, for various reasons including possible limitations of our financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K and most recent Quarterly Report on Form 10‐Q filed with the Securities and Exchange Commission (SEC) and in its other SEC filings.

For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's filings with the SEC. We caution investors not to place considerable reliance on the forward‐looking statements contained in this presentation. The forward‐looking statements in this presentation are made as of the date of this presentation only and, other than as required under applicable law, Sarepta does not undertake any obligation to publicly update its forward‐looking statements.

©SAREPTA THERAPEUTICS, INC. 20232. ALL RIGHTS RESERVED.

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A Bellwether Moment…

BENJAMIN

Living with Duchenne muscular dystrophy

THE OPPORTUNITY

SRP-9001

Poised to transform the trajectory of

Duchenne muscular dystrophy

©SAREPTA THERAPEUTICS, INC. 20232. ALL RIGHTS RESERVED.

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SCIENCE

Duchenne is progressive and causes irreversible muscle damage and loss of function

BIRTH

Lack of dystrophin causes muscle damage1,2,3

2-5 years

Delayed milestones, Gower's sign and toe-walking1,2,4

Gower's sign

Toe-walking

5-10years

Difficulty walking, balancing and climbing stairs, change in posture, use of walking aids required1,2,4

7-15years

Pain and limitations in upper limbs2,4,5

10-14years

LOA and wheelchair use at around 12 years,2,4,6,7,8 onset of scoliosis2,9,10

8-30years

Loss of upper limb function1,2,5

10-30years

Pulmonary decline1

15-30years

Cardiomyopathy and heart failure4,9

20-30years

Night-time ventilation progressing to

24h ventilation6,7,8,9,11

PROGRESSIVE & IRREVERSIBLE MUSCLE DAMAGE LEADING TO PREMATURE DEATH

0

5

10

15

20

25

30

YEARS

1. Birnkrant et al. Lancet Neurol. 2018 Mar;17(3):251-267;2. Verma et al. Clin Pediatr (Phila). 2010 Nov;49(11):1011-7;3. Aartsma-Rus et al. Hum Mutat. 2009 Mar;30(3):293-9;4. Duchenne UK, Stages of Duchenne https://www.duchenneuk.org/Pages/FAQs/Category/stages-of-duchenne (last accessed August 2018); 5. Janssen et al. J Neurol. 2014 Jul;261(7):1269-88;6. Rall and Grimm. Acta Myol. 2012 Oct;31(2):117-20;7. Koeks et al. J Neuromuscul Dis. 2017;4(4):293-306;8. Ryder et al. Orphanet J Rare Dis. 2017 Apr 26;12(1):79; 9. Birnkrant et al. Lancet Neurol. 2018

Apr;17(4):347-361;10. Archer et al. J Spine Surg. 2016 Sep;2(3):185-19;11. LoMauro et al. Ther Clin Risk Manag. 2015 28;11:1475-88.	©SAREPTA THERAPEUTICS, INC. 20232. ALL RIGHTS RESERVED.	5