Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates...
May 18 2021 - 8:50AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced positive
12-week expression and safety results from the first 11
participants enrolled in Study SRP-9001-103, an open-label study
known as ENDEAVOR being conducted in partnership with Roche. In
results from the first clinical study using commercially
representative material, SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)
demonstrated robust expression of micro-dystrophin and no new
safety signals from prior studies, supporting its potentially
differentiated profile for the treatment of Duchenne muscular
dystrophy. SRP-9001 is an investigational gene transfer therapy
intended to deliver its micro-dystrophin-encoding gene to muscle
tissue for the targeted production of the micro-dystrophin protein.
“We are delighted by these seminal results from
the ENDEAVOR Study, our first trial results with SRP-9001 made by
our commercial-scale manufacturing process. These data show strong
transduction of the micro-dystrophin gene, resulting in robust
expression of the properly localized micro-dystrophin protein, and
did so with no new or unexpected safety signals,” said Doug Ingram,
president and chief executive officer, Sarepta. “In addition to
characterizing and differentiating SRP-9001, these results confirm
the extraordinary work done over the last two and a half years to
build an at-scale gene therapy manufacturing process and
corresponding analytics sufficient to meet the needs of the
Duchenne population with what we believe will be a potentially
life-changing therapy. Armed with these data, we will seek a
meeting with the FDA with the goal of rapidly starting our
registrational study.”
In the open-label study, 20 participants between
the ages of four and seven were treated with a single infusion of
SRP-9001 at a dose of 1.33x1014 vg/kg. In muscle biopsies from the
first 11 patients taken 12 weeks after treatment, the following
results were observed:
- All patients demonstrated robust
transduction, with mean micro-dystrophin expression of 55.4% of
normal, as measured by western blot.
- Muscle dystrophin levels
demonstrated a mean of 70.5% (baseline 12.8%) muscle fibers
expressing micro-dystrophin at 12 weeks with a mean intensity at
the sarcolemma of 116.9% (baseline 41.0%) compared to normal
biopsies, as measured by immunofluorescence. Comparisons between
baseline and post-treatment measures were statistically significant
(p=0.001 for positive fibers, and p=0.002 for intensity).
- Mean vector genome copies per
nucleus reached 3.87.
The safety profile of
SRP-9001 observed in the first 11 participants in ENDEAVOR is
consistent with the safety seen in earlier studies using clinical
manufacturing process material. In line with previously reported
clinical data, no clinically relevant complement activation was
observed in these 11 patients. Two patients experienced serious
adverse events (transaminase elevation in one patient and nausea
and vomiting in a second patient) that fully resolved.
About SRP-9001-103 (ENDEAVOR)
Study SRP-9001-103 (Study 103) is an open-label clinical trial of
SRP-9001 that has enrolled 20 participants with Duchenne muscular
dystrophy between the ages of 4-7. Study 103 uses commercially
representative SRP-9001 and the primary endpoint is the change from
baseline in the quantity of micro-dystrophin protein expression
measured by western blot at 12 weeks. Secondary outcome measures
include change from baseline in micro-dystrophin expression fiber
intensity as measured by immunofluorescence (IF) and
micro-dystrophin expression measured by IF percent dystrophin
positive fibers at 12 weeks. Exploratory endpoints include the
change in vector genome copies per nucleus, North Star Ambulatory
Assessment (NSAA) and certain timed functional tests. Including the
initial 12-week period, patients will be followed for a total of
five years.
About SRP-9001
(rAAVrh74.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 for SRP-9001 and plans to
commercialize SRP-9001 in the United States upon receiving FDA
approval. In December 2019, Roche partnered with Sarepta to combine
Roche’s global reach, commercial presence and regulatory expertise
with Sarepta’s gene therapy candidate for Duchenne to accelerate
access to SRP-9001 for patients outside the United States. Sarepta
has exclusive rights to the micro-dystrophin gene therapy program
initially developed at the Abigail Wexner Research Institute at
Nationwide Children’s Hospital.
About Duchenne Muscular
DystrophyDuchenne muscular dystrophy (DMD) is a rare,
fatal neuromuscular genetic disease that occurs in approximately
one in every 3,500-5,000 males worldwide. DMD is caused by a change
or mutation in the gene that encodes instructions for dystrophin.
Symptoms of DMD usually appear in infants and toddlers. Affected
children may experience developmental delays such as difficulty in
walking, climbing stairs or standing from a sitting position. As
the disease progresses, muscle weakness in the lower limbs spreads
to the arms and other areas. Most patients require full-time use of
a wheelchair in their early teens, and then progressively lose the
ability to independently perform activities of daily living such as
using the restroom, bathing and feeding. 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 patients
usually succumb to the disease in their twenties.
About Sarepta
TherapeuticsSarepta is on an urgent mission: engineer
precision genetic medicine for rare diseases that devastate lives
and cut futures short. We hold leadership positions in Duchenne
muscular dystrophy (DMD) and limb-girdle muscular dystrophies
(LGMDs), and we currently have more than 40 programs in various
stages of development. Our vast pipeline is driven by our
multi-platform Precision Genetic Medicine Engine in gene therapy,
RNA and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
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.
Forward-Looking StatementsThis 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 potentially
differentiated profile of SRP-9001 for the treatment of Duchenne
muscular dystrophy; the potential for SRP-9001 to deliver
micro-dystrophin-encoding gene to muscle tissue for the targeted
production of the micro-dystrophin protein; the potential of our
gene therapy manufacturing process and corresponding analytics to
meet the needs of the Duchenne population with what we believe will
be a potentially life-changing therapy and our plan to meet with
the FDA with the goal of rapidly starting our registrational
study.
These forward-looking statements involve risks
and uncertainties that may cause actual results to differ
materially from those expressed or implied in the forward-looking
statements. Many of these risks and uncertainties are
beyond our control. Known risk factors include, among others:
success in preclinical and clinical trials, especially if based on
a small patient sample, does not ensure that later clinical trials
will be successful; the data presented in this release may not be
consistent with the final data set and analysis or result in an
assessment that SRP-9001 provides a safe or effective treatment
benefit; different methodologies or assumptions than we utilize to
assess particular safety or efficacy parameters may yield different
statistical results, and, even if we believe the data collected
from clinical trials are positive, the data may not be sufficient
to support approval by the FDA or foreign regulatory authorities;
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, many of which
are outside of our control, including possible limitations on
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; the impact of the COVID-19
pandemic; and those risks identified under the heading “Risk
Factors” in our most recent Annual Report on Form 10-K for the year
ended December 31, 2020, and most recent Quarterly Report on Form
10-Q filed with the Securities and Exchange Commission (SEC) as
well as other SEC filings we make, 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 we face, we encourage you to
review our SEC filings. We caution investors not to place
considerable reliance on the forward-looking statements contained
in this press release. We undertake no obligation to update
forward-looking statements based on events or circumstances after
the date of this press release.
Source: Sarepta Therapeutics, Inc.
Investor Contact: Ian Estepan,
617-274-4052iestepan@sarepta.com
Media Contact: Tracy
Sorrentino, 617-301-8566tsorrentino@sarepta.com
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Mar 2024 to Apr 2024
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Apr 2023 to Apr 2024