Sarepta Therapeutics Announces First Quarter 2016 Financial Results and Recent Corporate Developments CAMBRIDGE, Mass. (BUSINESS WIRE)

Sarepta Therapeutics Announces First Quarter 2016 Financial Results and Recent Corporate Developments

CAMBRIDGE, Mass. (BUSINESS WIRE) May 5, 2016 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics,

today reported financial results for the three months ended March 31, 2016, and provided an update of recent corporate developments.

and Central Nervous System Advisory Committee met last week to review eteplirsen for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping, and we await our May 26 PDUFA date, said Edward Kaye, M.D., Sarepta s

interim chief executive officer and chief medical officer. Following the FDA s decision, we plan to provide a clinical and corporate update.

For the first quarter of 2016,

Sarepta reported a non-GAAP net loss of $52.5 million, or $1.15 per share, compared to a non-GAAP net loss of $47.4 million for the first quarter of 2015, or $1.15 per share. The incremental loss of $5.1 million was primarily the result of increased

On a GAAP basis, the net loss for the first quarter of 2016 was $59.8 million, or $1.31 per share (including $7.2 million of

stock-based compensation and restructuring expenses), compared to a net loss of $61.6 million, or $1.49 per share (including $14.2 million of stock-based compensation) for the first quarter of 2015.

No revenue was recognized for the three months ended in both March 31, 2016 and 2015.

Non-GAAP research and development expenses were $35.9 million for the first quarter of 2016, compared to $36.7 million for the first quarter of 2015, a

decrease of $0.8 million.

GAAP research and development expenses were $38.8 million for the first quarter of 2016 (including $3.0 million of stock-based compensation and restructuring expenses), compared to $39.2 million

for the first quarter of 2015 (including $2.4 million of stock-based compensation), a decrease of $0.3 million.

Non-GAAP general and administrative

expenses were $16.6 million for the first quarter of 2016, compared to $11.0 million for the first quarter of 2015, an increase of $5.6 million. GAAP general and administrative expenses were $20.9 million for the first quarter of 2016 (including

$4.3 million of stock-based compensation and restructuring expenses), compared to $22.7 million for the first quarter of 2015 (including $11.7 million of stock-based compensation), a decrease of $1.8 million.

The Company had $140.6 million in cash, cash equivalents, short-term investments and restricted cash as of March 31, 2016 compared to $204.0 million as of

December 31, 2015, a decrease of $63.4 million. The decrease was due to the use of cash to fund the Company s ongoing operations, commercial launch activities and related inventory build.

In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements: non-GAAP research and

development expenses, non-GAAP general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for certain items.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating performance internally. The Company also believes these non-GAAP measures provide the Company s investors with useful information regarding the

Company s historical operating results. These non-GAAP measures are not intended to replace the presentation of the Company s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP

general and administrative expenses, non-GAAP operating expense adjustments, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies. All relevant non-GAAP measures are

reconciled from their respective GAAP measures in the attached table Reconciliation of GAAP to Non-GAAP Net Loss.

Recent Corporate Developments

Duchenne Muscular Dystrophy Program

About Sarepta Therapeutics

Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the

development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of rare, infectious and other diseases.

For more information, please visit us at www.sarepta.com.

Forward-Looking Statements

In order to provide Sarepta s investors with an understanding of its current results and future prospects, this press release contains statements that

are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans,

expects, will, may, intends, prepares, looks, potential, possible and similar expressions are intended to identify forward-looking statements. These

forward-looking statements include statements relating to Sarepta s future operations,

financial performance, business plans, priorities and development of product candidates including: Sarepta awaiting its PDUFA date of May 26, 2016 and plans to provide a clinical and corporate

update following the FDA s decision. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta s control. Known risk factors include, among others: the FDA may not provide eteplirsen with marketing

approval by the applicable PDUFA date or at all; we may not be able to comply with all FDA requests, including with respect to our eteplirsen NDA submission and the addendums we have submitted to the FDA or with respect to our ongoing or planned

clinical trials, in a timely manner or at all; we may not be able to complete clinical trials required by the FDA for approval of our products or any submissions made in connection with our pipeline of product candidates; the results of our ongoing

research and development efforts and clinical trials for our product candidates including eteplirsen and technologies may not be positive or consistent with prior results or demonstrate a safe treatment benefit or support an NDA filing, positive

advisory committee recommendation or marketing approval by the FDA or other regulatory authority; we may not be able to execute on our business plans including meeting our expected or planned regulatory milestones and timelines, clinical development

plans and bringing our product candidates to market, including the planned commercialization of eteplirsen, for various reasons including possible limitations of Company financial and other resources, manufacturing limitations that may not be

anticipated or resolved for in a timely manner or at all, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks

identified under the heading Risk Factors in Sarepta s most recent Annual Report on Form 10-K for the year ended December 31, 2015 or Quarterly Report on Form 10-Q for the quarter ended March 31, 2016 filed with the Securities and

Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could

materially and adversely affect Sarepta s business, results of operations and the trading price of Sarepta s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the Company s

filings with the SEC. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on

events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the For Investors section of our web site at www.sarepta.com. We

encourage investors and potential investors to consult our website regularly for important information about us.

Condensed Consolidated Statements of Operations

(in thousands, except per share amounts)

Sarepta Therapeutics, Inc.

Reconciliation of GAAP to Non-GAAP Net Loss

(in thousands, except per share amounts)

Sarepta Therapeutics, Inc.

Balance Sheet Highlights

Source: Sarepta Therapeutics, Inc.

Media and Investors:

Sarepta Therapeutics, Inc.

Ian Estepan, 617-274-4052

Ryan Flinn, 415-946-1059

Mobile, 510-207-7616