Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 & SRP-4053 for the Treatment of Du...
29 September 2016 - 12:00AM
Business Wire
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of
innovative RNA-targeted therapeutics, today announced the first
patient dosed in the phase III clinical trial of SRP-4045 and
SRP-4053 in patients with Duchenne muscular dystrophy amenable to
exon 45 or 53 skipping.
“We are excited to announce the first patient dosed in our
ESSENCE trial of SRP-4045 and SRP-4053, for DMD patients amenable
to Exon 45 and 53 skipping “ said Edward Kaye, Sarepta’s chief
executive officer. “It is our goal to treat as many patients
amenable to exon skipping as possible, and therefore is important
to advance our clinical pipeline candidates. We have made great
effort to develop a meaningful clinical trial, using our learnings
from our previous clinical programs and observations of the natural
history of Duchenne.”
The Phase III study, ESSENCE, is a double-blind,
placebo-controlled, multi-center study to evaluate the efficacy and
safety of SRP-4045 and SRP-4053. Eligible patients with
out-of-frame deletion mutations amenable to exon 45 or 53 skipping
will be randomized to receive once weekly intravenous (IV)
infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively
(combined-active group, 66 patients) or placebo (33 patients) for
up to 96 weeks (the placebo-controlled period of the trial). This
will be followed by an open label extension period in which all
patients will receive open-label active treatment for up to 96
weeks.
The study will enroll approximately 99 patients aged 7 to 13
years, inclusive, with a minimum target of 45 patients amenable to
exon 45 skipping and 45 patients amenable to exon 53 skipping.
Twice as many patients will receive active treatment as will
receive placebo. Approximately 66 patients will be randomized to
receive active treatment with either SRP-4045 or SRP-4053
(depending on deletion mutation), and 33 patients will be
randomized to receive placebo.
More information can be found at www.Sarepta.com or
www.clinicaltrials.gov, identifier: NCT02500381
About Duchenne Muscular Dystrophy (DMD)DMD is an X-linked
rare degenerative neuromuscular disorder causing severe progressive
muscle loss and premature death. One of the most common fatal
genetic disorders, DMD affects approximately one in every
3,500-5,000 males worldwide. 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. Eventually, increasing difficulty in breathing due to
respiratory muscle dysfunction requires ventilation support, and
cardiac dysfunction can lead to heart failure. The condition is
universally fatal, and death usually occurs before the age of
30.
About SRP-4045 and SRP-4053SRP-4045 and SRP-4053 are
designed to address the underlying cause of DMD by restoring the
messenger RNA (mRNA) reading frame, thus enabling the production of
a shorter form of the dystrophin protein. SRP-4045 and SRP-4053 use
Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO)
chemistry and exon-skipping technology to skip exons 45 and 53,
respectively, of the dystrophin gene. Promoting the synthesis of a
shorter dystrophin protein is intended to slow the decline of
ambulation and mobility seen in DMD patients. SRP-4045 and SRP-4053
have not been approved by the FDA or any regulatory authority for
the treatment of DMD.
About Sarepta TherapeuticsSarepta 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, including EXONDYS 51,
designed to skip exon 51 and approved under the accelerated
approval pathway. For more information, please visit us at
www.sarepta.com.
Forward Looking StatementsThis press release contains
forward-looking statements. These forward-looking statements
generally can be identified by the use of words such as “believes
or belief,” “anticipates,” “plans,” “expects,” “will,” “intends,”
“potential,” “possible,” “advance” and similar expressions. These
forward-looking statements include statements about the ESSENCE
study for product candidates SRP-4045 and SRP-4053 in DMD patients
amenable to exon 45 and exon 53 exon skipping, respectively,
advancing Sarepta’s pipeline with clinical studies such as ESSENCE,
Sarepta’s goal of treating as many patients amenable to
exon-skipping as possible, ESSENCE being a meaningful clinical
trial, and the ESSENCE study design.
Each forward-looking statement contained in this press release
is subject to risks and uncertainties that could cause actual
results to differ materially from those expressed or implied by
such statement. Applicable risks and uncertainties include, among
others: there may be delays in the ESSENCE study timelines, Sarepta
may not be able to successfully complete the ESSENCE study for
various reasons, including the possibility that the data does not
support safety or efficacy of SRP-4045 SRP-4053; SRP-4045 and
SRP-4053 could fail in development or may never receive regulatory
approvals required for commercialization as therapeutics for DMD
patients with mutations amenable to skipping of exon 45 and 53,
respectively, and those risks identified under the heading “Risk
Factors” in Sarepta’s Quarterly Report on Form 10-Q for the quarter
ended June 30, 2016 and Sarepta’s Annual Report on Form 10-K for
the year ended December 31, 2015 filed with the Securities and
Exchange Commission (SEC), and Sarepta’s other filings with the
SEC.
For a detailed description of risks and uncertainties Sarepta
faces, you are encouraged to review the Company’s filings with the
SEC. 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 InformationWe 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20160928006526/en/
Media and Investors:Sarepta Therapeutics, Inc.Ian Estepan,
617-274-4052iestepan@sarepta.comorW2O GroupBrian Reid,
212-257-6725breid@w2ogroup.com
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