Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural...
December 27 2017 - 08:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicines to treat rare neuromuscular
diseases, today announced that the pulmonary function results from
eteplirsen-treated Duchenne muscular dystrophy (DMD) patients
(N=12) in Study 201/202 compared to natural history were published
in the December 20, 2017 online edition of The Journal of
Neuromuscular Diseases. A statistically significant and clinically
meaningful reduction in pulmonary decline as measured by forced
vital capacity percent predicted (FVC%p) was observed for
eteplirsen-treated patients as compared to natural history data
published in the scientific literature.
In eteplirsen-treated patients, the mean FVC%p
decreased from 97.7% to 85.3% over 216 weeks, a decrease of
2.8% per study year. In an age-adjusted mixed-model
repeated-measures (MMRM) analysis of FVC%p, an annual decrease of
2.3% was observed for eteplirsen-treated patients compared to an
annual decrease of 4.1% observed in a natural history cohort with a
similar age range from the United Dystrophinopathy Project
(UDP).
“Patients treated with eteplirsen in Study
201/202 experienced significantly less deterioration of respiratory
muscle function than natural history would predict,” said Douglas
Ingram, Sarepta’s president and chief executive officer. “The
results included both ambulant and non-ambulant patients who
received eteplirsen. Our mission is to develop and bring to the
community precision genetic therapies that can improve the lives of
those suffering from DMD, a cruel degenerative disease.”
About Eteplirsen
Eteplirsen uses Sarepta’s proprietary
phosphorodiamidate morpholino oligomer (PMO) chemistry and
exon-skipping technology to skip exon 51 of the dystrophin gene.
Eteplirsen 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 eteplirsen 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
eteplirsen has not been established.
Important Safety Information About
Eteplirsen
Adverse reactions in DMD patients (N=8) treated
with eteplirsen 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 (eteplirsen, 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 eteplirsen is
not recommended.
In the 88 patients who received ≥30 mg/kg/week
of eteplirsen 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
eteplirsen 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 the results of Study 201/202 and
Sarepta’s mission to develop and bring to the community precision
genetic therapies that can improve the lives of those suffering
from DMD.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Known risk factors include, among others: the results of Sarepta’s
studies for eteplirsen, or other product candidates, may not be
positive or consistent with prior results or demonstrate a safe
treatment benefit; Sarepta may not be able to meet expectations
with respect to the sales of its therapy for the treatment of DMD;
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; 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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