-- Dosing with SRP-9001 commercial-process material to proceed following discussions with
-- Clinical dosing is expected to begin before the end of 2020 --
In light of the risks that the ongoing COVID-19 pandemic posed to the execution of a large, multi-center trial, Sarepta proposed a new clinical study in the
In the Type C meeting written response, the
“Our ability to rapidly address outstanding issues regarding our potency assay approach and commence Study 103 is an important milestone for SRP-9001 and for Duchenne muscular dystrophy patients who daily degenerate from this cruel disease,” said
About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)
SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing of SRP-9001 and plans to commercialize SRP-9001 in
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 and competitive 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.
Forward-Looking Statements
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 the expectation to commence clinical dosing with commercial-process material for SRP-9001 (study 103) before the end of 2020; the potential of Study 103 to accelerate Sarepta’s validation of commercial-process material; and the expectation to have a data read-out from our ongoing blinded, placebo-controlled trial (Study 102), in early 2021.
These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in pre-clinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; our data for SRP-9001 may not be sufficient for obtaining regulatory approval; 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, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company 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 with respect to patents that cover our product candidates, and the COVID-19 pandemic; and even if Sarepta’s programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended
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 the
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