Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for
Microdystrophin Gene Therapy Program
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today
announced it has entered a research and option agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy
program. Dr. Jerry Mendell, M.D. and Dr. Louise Rodino-Klapac, Ph.D., are the lead principal investigators of the program.
“Given the complexities of Duchenne muscular dystrophy, we know that it is going to require multiple treatment approaches,” said
Edward Kaye, Sarepta’s chief executive officer. “With that goal in mind, we are excited to support clinical development for
Nationwide’s gene therapy program with the goal to help all boys with DMD.”
The initial trial, expected to go into Phase 1/2a trial in late 2017, will be conducted at Nationwide Children’s. Parent Project
Muscular Dystrophy (PPMD) has committed 2.2 million dollars to the trial, with support from additional Duchenne foundations and
families. Sarepta has committed to the trial through a separate research agreement with Nationwide Children’s, and has an exclusive
option to license the program. PPMD's grant provided incentive for Sarepta to help expand and accelerate this opportunity.
“We are thrilled Sarepta has entered into this research agreement with Nationwide. The additional resources will bolster
Nationwide’s ability to conduct an even more rigorous and robust trial,” said Pat Furlong, founding president and chief executive
officer of Parent Project Muscular Dystrophy.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique
RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The Company is primarily focused on rapidly advancing
the development of its potentially disease-modifying DMD drug candidates. For more information, please visit us at www.sarepta.com.
About The Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2016-17 list of “America’s Best
Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric healthcare
systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as
well as adult patients with congenital disease. As home to the Department of Pediatrics of The Ohio State University College of
Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The
Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing
pediatric research facilities in the U.S., supporting basic, clinical, translational and health services research at Nationwide
Children’s. The Research Institute encompasses three research facilities totaling 525,000 square feet dedicated to research. More
information is available at NationwideChildrens.org/Research.
Forward-Looking Statements
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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements.
These forward-looking statements include statements about the research and exclusive option agreement Sarepta has entered into with
Nationwide Children’s Hospital for their microdystrophin gene therapy program, that DMD is going to require multiple treatment
approaches, Sarepta supporting Nationwide’s gene therapy program, the goal to help all boys with DMD, Sarepta’s resources
bolstering Nationwide’s ability to conduct an even more rigorous and robust trial, and the expected clinical progress of the
program in 2017.
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 agreements 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
agreements 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 agreements, 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 and even if commercialization of any product under the agreements is achieved this may not
result in any significant revenues to the parties.
Any of the foregoing risks could 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 Sarepta's
2015 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2016 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 web site
at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for
important information about us.
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
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