Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced top-line
results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing,
randomized, double-blind, placebo-controlled clinical trial to
evaluate the safety, efficacy and tolerability of a single dose of
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) in 41 patients with
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.
At 12 weeks post-treatment compared to baseline,
the study met its primary biological endpoint of micro-dystrophin
protein expression (P<0.0001). Participants who received
SRP-9001 (n=20) had mean micro-dystrophin expression of 28.1%, as
measured by western blot. Accompanying secondary biological
endpoints including vector genome copies per nucleus, percent
positive fibers, intensity, and reduction in creatine kinase
(exploratory) were also met.
In the primary functional endpoint,
SRP-9001-treated participants showed an increase in NSAA total
score compared to placebo at 48 weeks; however, the difference was
not statistically significant (P=0.37). At every time point
measured, the cohort of SRP-9001 treated participants outperformed
the placebo group, and, at 48 weeks, participants in the treatment
group demonstrated a statistically significant increase of 1.7
points in NSAA total score compared to baseline (P=0.009), while
participants in the placebo group saw an increase of 0.9 points on
the NSAA total score compared to baseline, which was not
statistically significant (n=21, P=0.1411).
Study randomization was stratified by age group
and, in the pre-specified analysis of participants aged 4-5 (n=16)
at the time of treatment, the treatment group demonstrated a
statistically significant 4.3-point improvement on NSAA total score
at 48 weeks post treatment compared to a 1.9-point improvement in
the age-matched placebo group (P=0.0172). The functional status at
baseline for participants in the 4-5 age group was balanced across
the placebo and treatment cohorts. A statistically significant
imbalance (P=0.0046) in baseline NSAA total score was present in
the cohort of 6-7-year-old participants (n=25), resulting in milder
participants in the placebo arm (n=13) than in the treated arm
(n=12). The significantly different baseline characteristics
between treatment and control groups in the 6-7 age group may have
contributed to the inability to observe a treatment effect in the
6-7 age group at the week 48 timepoint in Part 1.
The results from Study 102 reinforce the
favorable safety and tolerability profile of SRP-9001 with no new
safety signals identified. In line with previously reported
clinical data, no clinical complement activation was observed. 85%
of participants in the treatment group experienced at least one
treatment-related adverse event compared to 43% in the placebo
group. Among participants with treatment-related adverse events,
82% were mild or moderate in severity, and 4 participants
experienced serious treatment-related adverse events including 3
participants in the treatment group (2 cases of rhabdomyolysis, 2
transaminase elevations) and 1 participant in the placebo
(rhabdomyolysis).
Study 102 is ongoing and remains blinded to
participants, investigators, site staff and sponsor staff with
direct site interaction. All 41 participants have completed their
Part 1, 48-week assessment and have entered the Part 2 crossover
phase. Participants continue to be monitored for safety and will
undergo another biopsy at week 12 in Part 2 to assess expression
and biological markers, in addition to longer-term assessments of
functional outcomes.
“Study 102 reinforces our confidence in the
potentially transformative benefits of SRP-9001, including among
other things, the fact that in the Study’s pre-specified analysis,
the participants in the 4-5 age group robustly achieved a
statistically significant and clinically meaningful improvement in
NSAA over placebo, as predicted by our prior Study 101. For the
entire population, while we saw separation at every time point
between the active and placebo cohorts, Study 102 did not achieve
statistical significance on the primary functional endpoint. In
this regard, we are very disappointed that the randomization
process resulted in a significant imbalance in baseline NSAA scores
between the active and placebo cohorts of the participants ages
6-7, making the 6-7 age groups non-comparable and likely
substantially contributing to the inability to achieve statistical
significance,” said Doug Ingram, president and chief executive
officer, Sarepta. “Study 102 remains blinded and we will analyze
the functional results for all patients, including cross-over
participants, once they have achieved the 48-week timepoint in Part
2. We have already enrolled and dosed 11 participants in Study 103,
using our commercial process material, and we will have biomarker
and safety results from that cohort in the second quarter. And very
importantly, Study 102 has provided us with a wealth of information
and insight which we will use to refine and complete the protocol
for our upcoming trial using commercial process material. We intend
to continue to move forward with diligence and urgency to generate
the evidence necessary to bring SRP-9001 to waiting Duchenne
patients around the world.”
Sarepta will host an investor webcast and
conference call on Thursday, Jan. 7, 2021 at 4:30 pm Eastern Time,
to discuss these results. The presentation will be webcast live
under the investor relations section of Sarepta's website at
https://investorrelations.sarepta.com/events-presentations and
slides will be archived there following the call for one year.
Please connect to Sarepta's website several minutes prior to the
start of the broadcast to ensure adequate time for any software
download that may be necessary. The conference call may be accessed
by dialing (844) 534-7313 for domestic callers and (574) 990-1451
for international callers. The passcode for the call is 2538387.
Please specify to the operator that you would like to join the
"Micro-dystrophin SRP-9001 Study 102 Top-line Results Call."
*The NSAA is a 17-item rating scale that is used
to measure functional motor abilities in ambulant children with
Duchenne. It is used to monitor the progression of the disease and
treatment effects which makes it suitable as an endpoint in
clinical trials for Duchenne.
About SRP-9001-102 Study
SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized,
placebo-controlled clinical trial of SRP-9001 in 41 participants
with Duchenne muscular dystrophy between the ages of 4-7. Study 102
uses clinical process SRP-9001 and has two primary endpoints:
micro-dystrophin expression at 12 weeks and change in NSAA total
score at 48 weeks compared to placebo. Secondary endpoints include
certain timed functional tests; micro-dystrophin expression
measured by immunofluorescence (IF) fiber intensity; and
micro-dystrophin expression measured by IF percent dystrophin
positive fibers. In Part 1, results from the treatment and placebo
groups are compared through 48 weeks following treatment. In Part
2, the study remains blinded while all participants in the placebo
group cross over to active treatment and all participants are
followed for another 48 weeks while safety and efficacy continue to
be evaluated.
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, the Company announced a licensing
agreement granting Roche the exclusive right to launch and
commercialize SRP-9001 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, neck 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
TherapeuticsAt Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Sarepta Forward-Looking
StatementThis 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 transformative
benefits of SRP-9001; our plan to analyze the functional results
for all the patients in Study 102 once they have achieved the
48-week timepoint; the expectation to have biomarker and safety
results from Study 103 in the second quarter of 2021; our plan to
use the information and insight from Study 102 to refine and
complete the protocol for our upcoming trial using commercial
process material; and our intention to continue to move forward
with diligence and urgency to generate the evidence necessary to
bring SRP-9001 to waiting Duchenne patients around the world.
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, 2019, 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.
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.
Sarepta Therapeutics, Inc.
Investors:Ian Estepan,
617-274-4052iestepan@sarepta.com Media:Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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