Sarepta Therapeutics Announces First Quarter 2018 Financial Results and Recent Corporate Developments First quarter 2017 EXONDYS 51 (eteplirsen) total net revenues of $64.6 million Sarepta signs exclusive partnership and

Sarepta Therapeutics Announces First Quarter 2018 Financial Results and Recent Corporate Developments

First quarter 2017 EXONDYS 51 (eteplirsen) total net revenues of $64.6 million

Sarepta signs exclusive partnership and buy-out option with Myonexus Therapeutics; pipeline expands from

Company announces date of first R&D day, at which clinical data from gene therapy micro-dystrophin program will be

Company receives negative trend vote following its CHMP oral explanation; will request

re-examination and Scientific Advisory Group to be convened

CAMBRIDGE, Mass., May 3, 2018 (GLOBE

NEWSWIRE) Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, today reported financial results

for the three months ended March 31, 2018.

In the first quarter, we continued our successful launch of EXONDYS 51 and advanced our pipeline to

bring life-enhancing therapies to those suffering from rare disease around the world, said Doug Ingram, Sarepta s president and chief executive officer. We accelerated our gene therapy and RNA platform, and in that regard are

excited to announce that our first R&D day will take place on June 19 to showcase the breadth, depth and progress of our pipeline. Significantly, at this event we will report preliminary safety and gene expression data from at least two

patients from our micro-dystrophin gene therapy trial underway with Nationwide Children s Hospital.

Mr. Ingram continued, Aligned

with our stated goal of leveraging our expertise beyond DMD, we announced today a collaboration with Myonexus Therapeutics for the development of five potentially transformative gene therapies to treat a debilitating set of diseases, all under the

umbrella of Limb-girdle muscular

dystrophy. Through this collaboration, we have expanded our pipeline to 21 therapies in development. Our

confidence in the Myonexus collaboration comes from the similarities between the Myonexus and Sarepta approaches to gene therapy. Both are seeking to treat rare neuromuscular disease through the AAVrh.74 vector; and both rely upon the unparalleled

expertise of Dr. Louise Rodino-Klapac in developing and executing gene therapy constructs. This partnership with Myonexus enables us to expand our efforts beyond DMD while maintaining our unwavering commitment to those suffering from DMD.

Mr. Ingram concluded, Unfortunately, in addition to our successes in the first quarter, we also have had a delay in our effort to bring

eteplirsen to patients in Europe who could potentially benefit from it. I could not be prouder of our Sarepta team and the team of experts who spoke on behalf of eteplirsen at the CHMP oral explanation last week. The rigorous work that was done to

prepare for the hearing only strengthened our resolve that eteplirsen should urgently be made available to those waiting in Europe. Unfortunately, the CHMP s trend vote was negative. Based on discussions with CHMP representatives, it is our

understanding that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta has not yet met the regulatory threshold for conditional approval, in part due to the use of external controls as

comparators in the studies. Sarepta plans to file for re-examination and will request that a Scientific Advisory Group (SAG), which is made up of DMD and neuromuscular specialists, be convened to provide

expert guidance and insight into, among other things, the validity of the external controls used and the importance of slowing pulmonary decline in patients with DMD.

For the first quarter of 2018, on a

GAAP basis, Sarepta reported a net loss of $35.4 million, or $0.55 per basic and diluted share, compared to net income of $84.1 million reported for the same period of 2017, or $1.53 per basic share and $1.50 per diluted share. On a

non-GAAP basis, the net loss for the first quarter of 2018 was $17.9 million, or $0.28 per share, compared to a net loss of $31.4 million for the same period of 2017, or $0.57 per share.

For the three months ended

March 31, 2018, the Company recorded net product revenues of $64.6 million, compared to net revenues of $16.3 million for first quarter of 2017. The increase primarily reflects increasing demand for EXONDYS 51 in the

Cost and Operating Expenses

Cost of sales (excluding amortization of in-licensed rights)

For the three months ended March 31, 2018, cost of sales (excluding amortization of in-licensed rights) was $5.6 million, compared to

$0.2 million for the same period of 2017. The increase primarily reflects royalty payments to BioMarin Pharmaceuticals (BioMarin) as a result of the execution of the settlement and license agreements with BioMarin in July 2017 as well as

higher inventory costs related to increasing demand for EXONDYS 51 during 2018. Prior to the approval of EXONDYS 51, the Company expensed related manufacturing and material costs as research and development expenses.

Research and development

Research and development

expenses were $46.2 million for the first quarter of 2018, compared to $29.1 million for the same period of 2017, an increase of $17.1 million. The increase in research and development expenses primarily reflects the

Non-GAAP research and development expenses were $43.3 million for the first

quarter of 2018, compared to $26.7 million for the same period of 2017, an increase of $16.6 million.

Selling, general and administration

Selling general and administrative expenses were $43.3 million for the first quarter of 2018, compared to $26.2 million for the same period of

2017, an increase of $17.1 million. The increase in selling, general and administrative expenses primarily reflects the following:

Non-GAAP selling, general and administrative expenses were $33.7 million for the first quarter of 2018, compared

to $21.1 million for the same period of 2017, an increase of $12.6 million.

Amortization of in-licensed rights was $0.2 million during the first quarter of

2018, compared to less than $0.1 million for the same period of 2017. The increase was primarily due to the BioMarin transactions that occurred in July 2017.

Priority Review Voucher

In connection with the completion of the sale of the Priority Review Voucher (PRV) in March 2017, the Company recorded a

gain of $125.0 from sale of the PRV in the first quarter of 2017. There was no similar activity in the first quarter of 2018.

income and other, net

For the three months ended March 31, 2018 and 2017, the Company recorded $4.5 million interest expense and other,

net and $0.3 interest income and other, net, respectively. The period over period unfavorable change primarily reflects the interest expense accrued on the Company s debt facilities partially offset by interest income from higher balances of

cash, cash equivalents and investments.

Cash, Cash Equivalents, Restricted Cash and Investments

The Company had $1.0 billion in cash, cash equivalents, restricted cash and investments as of March 31, 2018 compared to $1.1 billion as of

December 31, 2017. The decrease is primarily driven by the use of cash to fund the Company s ongoing operations during the first quarter of 2018.

Use of Non-GAAP Measures

In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements. The non-GAAP loss is defined by the Company as GAAP net loss excluding interest expense/(income), income tax expense/(benefit), depreciation and amortization expense, stock-based compensation expense, restructuring

expense and other items. Non-GAAP research and development expenses are defined by the Company as GAAP research and development expenses excluding depreciation and amortization expense, stock-based

compensation expense, restructuring expense and other items. Non-GAAP selling, general and administrative expenses are defined by the Company as GAAP selling, general and administrative expenses excluding

depreciation and amortization expense, stock-based compensation expense, restructuring expense and other items.

1. Interest, tax, depreciation and

Interest income and expense amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates

driven by market conditions outside of the Company s operations. Tax amounts can vary substantially from period to period due to tax adjustments that are not directly related to underlying operating performance. Depreciation expense can vary

substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to the Company s operating performance. Amortization expense associated with in-licensed rights as well as patent costs are amortized over a period of several years after acquisition or patent application or renewal and generally cannot be changed or influenced by management.

2. Stock-based compensation expenses

Stock-based compensation

expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially

from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within management s control. Therefore, management believes that excluding these charges facilitates comparisons of the

Company s operational performance in different periods.

3. Restructuring expenses

The Company believes that adjusting for these items more closely represents the Company s ongoing operating performance and financial results.

The Company evaluates other items of expense and

income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relates to the Company s ongoing business operations, and (c) whether

the Company expects the items to continue on a regular basis. These other items include the aforementioned gain from the sale of the Company s PRV.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational

performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of

period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or 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 selling, general and administrative expenses, non-GAAP other income

adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from

similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from

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

First Quarter and Recent Corporate Developments

The Company will be hosting a conference call at 4:30 p.m. Eastern Time, to discuss these financial results and provide a corporate update. The conference call

may be accessed by dialing 844-534-7313 for domestic callers and

+1-574-990-1451 for international callers. The passcode for the call is 2798939. Please specify to the operator that you would

like to join the Sarepta First Quarter 2018 Earnings Call . The conference call will be webcast live under the investor relations section of Sarepta s website at www.sarepta.com and will be archived there following the call for 90

days. Please connect to Sarepta s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

EXONDYS 51 uses Sarepta s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the

dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51

skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Important Safety Information About

Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred

in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more

than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of

patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received 30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the

following events were reported in 10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper