Press Release

CAMBRIDGE, Mass., April 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the Company and the United States Army Medical Research Institute of Infectious Diseases (USAMRIID), the Department of Defense’s lead laboratory for medical biological defense research, have entered into a Cooperative Research and Development Agreement (CRADA). The purpose of the CRADA is to jointly identify antisense oligonucleotides using Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) platform with activity against SARS-CoV-2 for the potential treatment of COVID-19.  

Previously published clinical and preclinical studies of Sarepta’s RNA technology have found evidence of antiviral activity of Sarepta’s PMO technology in coronaviruses and other viruses.^{i,ii,iii, iv} Pursuant to the CRADA, Sarepta will design, synthesize, manufacture and provide to USAMRIID multiple peptide-conjugated PMO (PPMO) constructs based on genetic sequencing of SARS-CoV-2 for COVID-19. USAMRIID will evaluate the constructs on characterized wild-type SARS-CoV-2 viruses for their potential to inhibit viral infection. Based on the results, Sarepta and USAMRIID will consider collaborative funding proposals to advance the development of treatments for COVID-19.  

“We should all be proud and grateful that in collaboration with health agencies, many innovative companies across the biopharmaceutical ecosystem have mobilized to fight and solve this COVID-19 pandemic, investing significant resources to rapidly build diagnostics, find treatments, and develop effective vaccines,” said Doug Ingram, Sarepta’s President and Chief Executive Officer.  “While Sarepta’s mission to rapidly advance treatments for rare and often fatal genetic disease is focused, unwavering and undeterred, we cannot ignore the impact of this global pandemic on human health, and have answered the call to contribute our scientific expertise and provide our technology in the race to develop an effective treatment for COVID-19. Indeed, we have already built and manufactured therapeutic PPMO constructs and are providing them now to USAMRIID for testing and evaluation.”

About the U.S. Army Medical Research Institute of Infectious Diseases
For over 50 years, USAMRIID has provided leading edge medical capabilities to deter and defend against current and emerging biological threat agents. The Institute is the only laboratory in the Department of Defense equipped to safely study highly hazardous viruses requiring maximum containment at Biosafety Level 4. Research conducted at USAMRIID leads to medical solutions—vaccines, drugs, diagnostics, information, and training programs—that benefit both military personnel and civilians. Established in 1969, the Institute plays a key role as the lead military medical research laboratory for the Defense Threat Reduction Agency’s Joint Science and Technology Office for Chemical and Biological Defense. USAMRIID is a subordinate laboratory of the U.S. Army Medical Research and Development Command. For more information, visit www.usamriid.army.mil

[The information contained in this press release does not necessarily reflect the position or the policy of the Government and no official endorsement should be inferred.]

About Sarepta Therapeutics
At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statement
This press release contains forward-looking statements. 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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the purpose of the CRADA to jointly identify antisense oligonucleotides using Sarepta’s PMO platform with activity against SARS-CoV-2; the parties’ commitments under the CRADA; Sarepta and USAMRIID’s plan, subject to an evaluation of the constructs, to consider collaborative funding proposals to advance the development of treatments for COVID-19; the potential of the collaboration between Sarepta and USAMRIID to develop a treatment for COVID-19; and Sarepta’s mission to rapidly advance treatments for rare and often fatal genetic disease.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the CRADA may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; in particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s 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, 2019 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’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 Sarepta's 2019 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. 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 website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. 

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:
Ian Estepan, 617-274-4052
iestepan@sarepta.com

Media:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com

ⁱ Neuman BW, et al. J Virol. 2004 Jun;78(11):5891-9. Antisense morpholino-oligomers directed against the 5' end of the genome inhibit coronavirus proliferation and growth.

ⁱⁱ Burrer R, et al. J Virol. 2007 Jun;81(11):5637-48. Antiviral effects of antisense morpholino oligomers in murine coronavirus infection models.

ⁱⁱⁱ Neuman BW, et al. J Virol. 2005 Aug;79(15):9665-76. Inhibition, escape, and attenuated growth of severe acute respiratory syndrome coronavirus treated with antisense morpholino oligomers.

^iv Heald AE, et al. Antimicrob Agents Chemother. 2014 Nov;58(11):6639-47. Safety and Pharmacokinetic Profiles of Phosphorodiamidate Morpholino Oligomers with Activity Against Ebola Virus and Marburg Virus: Results of Two Single-Ascending-Dose Studies.

Source: Sarepta Therapeutics, Inc.