-- FDA Grants Priority Review Status --
-- Regulatory Action Date is August 19, 2019 --
-- Golodirsen has been studied for the treatment of exon 53 amenable patients, approximately eight percent of patients with
Duchenne --
CAMBRIDGE, Mass., Feb. 14, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, announced the Food and Drug Administration, Division of Neurology (the Division), has
accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action
date of August 19, 2019. Golodirsen is a phosphordiamidate morpholino oligomer engineered to treat those individuals with Duchenne
muscular dystrophy (Duchenne) who have genetic mutations subject to skipping exon 53 of the dystrophin gene. Duchenne is a fatal
genetic neuromuscular disorder affecting an estimated one in approximately every 3,500 - 5,000 males born worldwide.
The company completed its NDA at the end of 2018 as part of a rolling submission and requested priority review,
which was granted. The company previously received orphan drug designation for golodirsen. The NDA includes data from the 4053-101
study assessing the safety, tolerability, pharmacokinetics and dystrophin expression of golodirsen in 25 boys with confirmed
deletions of the dystrophin gene amenable to exon 53 skipping. The study demonstrated statistically significant results in favor of
golodirsen on all biological endpoints, including properly exon-skipped RNA transcript using reverse transcription polymerase chain
reaction, increase in quantity of dystrophin expression from baseline using Western blot and increase in dystrophin intensity as
measured by immunohistochemistry.
“If approved, golodirsen will serve up to another 8 percent of the Duchenne community, bringing us closer to
helping as many Duchenne patients as possible,” said Doug Ingram, president and chief executive officer, Sarepta. “We look
forward to working with the FDA toward advancing this important therapy and rapidly bringing it to individuals with Duchenne who
are amenable to exon 53 skipping.”
Golodirsen is also being studied in Sarepta’s ongoing ESSENCE study (4045-301), a global, randomized
double-blind, placebo-controlled study assessing the safety and efficacy of golodirsen and casimersen, our exon 45 skipping agent.
The Division has previously confirmed that the ESSENCE study could possibly serve as a post-marketing confirmatory study.
About Golodirsen
Golodirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip
exon 53 of the DMD gene. Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,”
of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is
intended to allow for production of an internally truncated but functional dystrophin protein.
Dystrophin is a protein found in muscle cells that, while present in extremely small amounts (about 0.002
percent of total muscle protein), is crucial in strengthening and protecting muscle fibers. A devastating and incurable
muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key
structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas
of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or
cardiac failure.
About Sarepta Therapeutics
Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular
dystrophy (DMD) and more recently in gene therapies for 5 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth
(CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s
programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by
an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For
more information, please visit www.sarepta.com.
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 statements regarding the expected regulatory action date of August 19, 2019; the estimated
number of patients suffering from Duchenne and golodirsen’s potential to serve up to another 8 percent of the Duchenne
community; the possibility of ESSENCE serving as a post-marketing confirmatory study; the potential of exon skipping to
allow for production of an internally truncated but functional dystrophin protein; and Sarepta’s mission to profoundly improve and
extend the lives of patients with rare genetic-based diseases.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s
control. Known risk factors include, among others: Sarepta may not be able to complete clinical trials required by the FDA for
approval of golodirsen; priority review designation by the FDA for golodirsen may not lead to faster development or regulatory
review or approval process, and it does not increase the likelihood that golodirsen will receive marketing approval; golodirsen may
not result in a viable treatment suitable for commercialization due to a variety of reasons including the results of future
research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and
efficacy of product candidates; and even if golodirsen results in a commercialized product, Sarepta may not achieve any significant
revenues from the sale of such product; if the actual number of exon 53 amenable patients is smaller than estimated,
Sarepta’s revenue and ability to achieve profitability may be adversely affected; Sarepta may not be able to execute on its
business plans, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and
bringing its products to U.S. and ex-U.S. markets 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, 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, 2017 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 2017 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.
Contacts
Investors:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
