Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicine to treat rare neuromuscular diseases,
announced today that the U.S. Food and Drug Administration (FDA)
has cleared its Investigational New Drug (IND) application for the
Company’s peptide phosphorodiamidate morpholino oligomer (PPMO)
exon 51 candidate, SRP-5051. Sarepta will immediately initiate its
Phase 1/2a clinical trial and begin screening patients with DMD
amenable to skipping exon 51.
Results of the Phase 1/2a trial will inform the
multi-center, double blind, placebo-controlled, multi-dose efficacy
study to evaluate dystrophin expression and clinical outcomes, and
is planned to initiate by mid-year 2018 or as soon as a therapeutic
dose has been identified.
Sarepta’s next-generation PPMO platform is
designed around a proprietary cell-penetrating peptide conjugated
to the phosphorodiamidate morpholino oligomer (PMO) backbone, with
the goal of increasing tissue penetration.
“Our PMO platform, on which EXONDYS 51 is based,
represents precision genetic medicine that is designed to elegantly
engineer the expression of dystrophin through modification of mRNA
in muscle cells. Our next-generation platform, PPMO, greatly
advances this science by using a proprietary peptide technology to
act as a transporter of the PMO into the muscle cell, which in
animal models has substantially increased mRNA modification and
dystrophin production,” said Douglas Ingram, Sarepta’s president
and chief executive officer. “SRP-5051, the first clinical
candidate from our PPMO platform, represents a decade’s long
investment and tireless work by Sarepta and its scientists.
Preclinical models point to the potential of this next-generation
class of chemistry to substantially increase efficacy while
reducing the frequency of dosing.”
Mr. Ingram continued, “DMD is a cruel and, at
least today, an invariably fatal disease. The clearance of this IND
to bring SRP-5051 to the clinic is not merely a success for
Sarepta. More importantly, it represents a potential approach for a
better life for children living with DMD. For that reason, we
intend to move urgently in further pursuit of our goal of
translating promising science into potentially life-saving and
life-enhancing medicines. Without delay, Sarepta will begin
enrolling patients in the study to identify a safe and therapeutic
dose of SRP-5051. Beyond that, we have crafted an ambitious
strategy to sequence and rapidly advance multiple PPMO-based
therapies designed to change the course of DMD. In parallel, if we
have positive signals in our first trial, we will explore the
potential of applying our PPMO technology to a broad range of other
neuromuscular diseases.”
About SRP-5051
SRP-5051 uses Sarepta’s PPMO chemistry and
exon-skipping technology to skip exon 51 of the dystrophin gene.
SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA,
resulting in exclusion of this exon during mRNA processing in
patients with genetic mutations that are amenable to exon 51
skipping. Exon skipping is intended to allow for production of an
internally truncated dystrophin protein. PPMO is Sarepta’s
next-generation chemistry platform designed around a proprietary
cell-penetrating peptide conjugated to the PMO backbone, with the
goal of increasing tissue penetration, increasing exon skipping and
significantly increasing dystrophin production. If successful, the
PPMO offers the potential for improved efficacy and less frequent
dosing for patients.
About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary
phosphorodiamidate morpholino oligomer (PMO) chemistry and
exon-skipping technology to skip exon 51 of the dystrophin gene.
EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA,
resulting in exclusion of this exon during mRNA processing in
patients with genetic mutations that are amenable to exon 51
skipping. Exon skipping is intended to allow for production of an
internally truncated dystrophin protein. Data from clinical studies
of EXONDYS 51 in a small number of DMD patients have demonstrated a
consistent safety and tolerability profile. The pivotal trials were
not designed to evaluate long-term safety and a clinical benefit of
EXONDYS 51 has not been established.
Important Safety Information About
EXONDYS 51
Adverse reactions in DMD patients (N=8) treated
with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion
with an incidence of at least 25% more than placebo (N=4) (Study 1,
24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%),
vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most
common adverse reactions were balance disorder and vomiting.
Because of the small numbers of patients, these represent crude
frequencies that may not reflect the frequencies observed in
practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is
not recommended.
In the 88 patients who received ≥30 mg/kg/week
of EXONDYS 51 for up to 208 weeks in clinical studies, the
following events were reported in ≥10% of patients and occurred
more frequently than on the same dose in Study 1: vomiting,
contusion, excoriation, arthralgia, rash, catheter site pain, and
upper respiratory tract infection.
There have been reports of transient erythema,
facial flushing, and elevated temperature occurring on the day of
EXONDYS 51 infusion.
For further information, please see the full
Prescribing Information.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicines to treat rare neuromuscular
diseases. The Company is primarily focused on rapidly advancing the
development of its potentially disease-modifying Duchenne muscular
dystrophy (DMD) drug candidates. 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 Sarepta’s plan to immediately initiate
a Phase 1/2a clinical trial in patients with DMD amenable to
skipping exon 51; the study design; the results of the Phase 1/2a
trial informing the multi-center, double blind, placebo-controlled,
multi-dose efficacy study to evaluate dystrophin expression and
clinical out-comes, and the plan to initiate such study by mid-year
2018 or as soon as a therapeutic dose has been identified; the goal
of Sarepta’s next-generation PPMO platform of increasing tissue
penetration; the PMO platform representing precision genetic
medicine that is designed to elegantly engineer the expression of
dystrophin through modification of mRNA in muscle cells and the
PPMO greatly advancing this science; the potential of PPMO to
substantially increase efficacy while reducing the frequency of
dosing; bringing SRP-5051 to the clinic representing a potential
approach for a better life for children living with DMD; Sarepta’s
intention to move urgently in further pursuit of its goal of
translating promising science into potentially life-saving and
life-enhancing medicines; Sarepta’s plan to begin enrolling
patients in the study to identify a safe and therapeutic dose of
SRP-5051; Sarepta’s ambitious strategy to sequence and rapidly
advance multiple PPMO-based therapies designed to change the course
of DMD and to explore the potential of applying the PPMO technology
to a broad range of other neuromuscular diseases; PPMO’s goal of
increasing tissue penetration, increasing exon skipping and
significantly increasing dystrophin production; and the potential
of PPMO for improved efficacy and less frequent dosing for
patients.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: there may be delays in
the study timelines and Sarepta may not be able to successfully
complete the study for various reasons, including any negative or
inconsistent safety and efficacy data; the study data and results
may not provide support for an NDA filing; Sarepta’s PPMO
technology, including SRP-5051, may not result in any viable
treatments 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 PPMO results in commercialized
products, Sarepta may not achieve any significant 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 December 31, 2016 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 2016 Annual Report on Form 10-K and
most recent Quarterly Report on Form 10-Q for the quarter ended
September 30, 2017 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.
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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