Solid Biosciences Provides Data Update from SGT-001 Development Program
December 18 2019 - 6:30AM
Solid Biosciences Inc. (Nasdaq: SLDB) today announced biomarker
data from two patients dosed in the second cohort of IGNITE DMD,
its Phase I/II study of SGT-001. SGT-001 is the company’s gene
transfer candidate under investigation for Duchenne muscular
dystrophy (Duchenne). The data from these patients show SGT-001
microdystrophin expression and associated neuronal nitric oxide
synthase (nNOS) function, providing evidence that SGT-001 has the
potential to result in therapeutic benefit for patients with
Duchenne. The company also announced that the previously reported
serious adverse event experienced by the third patient in the 2E14
vg/kg dose group has fully resolved, and the patient has resumed
his normal activities. The company has received the clinical hold
letter from the U.S. Food and Drug Administration (FDA) and will
continue working internally, and with the FDA and other external
experts, to address the clinical hold and determine the path
forward.
“We now have evidence that SGT-001 can lead to microdystrophin
expression at levels that we believe are meaningful and warrant
further clinical development,” said Ilan Ganot, Chief Executive
Officer, President and Co-Founder of Solid Biosciences. “I’m also
pleased to say that the patient who experienced the event announced
in November is doing well. We are steadfast in our commitment to
bringing a transformative and safe therapy to the Duchenne
community and are working diligently to resolve the clinical hold
and resume dosing with SGT-001.”
Six patients have been dosed with SGT-001 as part of IGNITE DMD;
three at the 5E13 vg/kg dose and three at the 2E14 vg/kg dose.
Three-month biopsies were recently analyzed from the fourth and
fifth patients, both administered SGT-001 at 2E14 vg/kg. Using
immunofluorescence assays, 10%-20% of microdystrophin positive
muscle fibers were determined to express SGT-001 microdystrophin in
the fourth patient and 50%-70% microdystrophin positive fibers in
the fifth patient. Immunofluorescence also showed clear
stabilization and co-localization of nNOS and beta-sarcoglycan with
SGT-001 microdystrophin in both patients. Inclusion of this nNOS
coding region of the dystrophin protein may result in
microdystrophin protein that has unique activity, potentially
providing important functional benefits such as diminished muscle
fatigue and protection against ischemic muscle damage. Using
western blot, the expression levels for the fourth patient were
detectable and estimated to be near the assay’s level of
quantification which is 5% of non-dystrophic control samples, with
one assay replicate at 5.5%. Expression for the fifth patient was
17.5% of normal control samples. The levels of serum creatine
kinase, a highly variable biochemical marker of muscle damage,
declined from baseline in both patients. Collectively, these data
provide evidence supporting the biological activity of SGT-001.
“SGT-001 represents the most advanced dystrophin biology in
development for Duchenne,” said Jeffrey Chamberlain, Professor of
Neurology and McCaw Endowed Chair in Muscular Dystrophy at the
University of Washington School of Medicine. “SGT-001’s novel
construct was specifically selected to drive expression of the
drug’s unique microdystrophin in cardiac and skeletal muscle,
critical not only to mobility, but also to cardiac and pulmonary
function. Continued evaluation of SGT-001 is essential to determine
the ultimate clinical benefits that SGT-001 may provide for
patients.”
Conference Call InformationThe company will
host a conference call and webcast at 8:30 a.m. ET today to discuss
the program update. Participants are invited to listen by dialing
+1 866-763-0341 (domestic) or +1 703-871-3818 (international) five
minutes prior to the start of the call and providing the passcode
5371089. A listen-only webcast of the conference call can also be
accessed through the "Investors" tab on the Solid Biosciences
website, www.solidbio.com, and a replay of the call will be
available for approximately six weeks after the call.
About SGT-001Solid’s lead candidate, SGT-001,
is a novel adeno-associated viral (AAV) vector-mediated gene
transfer under investigation for its ability to address the
underlying genetic cause of Duchenne, mutations in the dystrophin
gene that result in the absence or near absence of dystrophin
protein. SGT-001 is a systemically administered candidate that
delivers a synthetic dystrophin gene, called microdystrophin, to
the body. This microdystrophin encodes for a functional protein
surrogate that is expressed in muscles and stabilizes essential
associated proteins, including neuronal nitric oxide synthase
(nNOS). Data from Solid’s preclinical program suggests that SGT-001
has the potential to slow or stop the progression of Duchenne,
regardless of genetic mutation or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by
Dr. Jeffrey Chamberlain of the University of Washington and Dr.
Dongsheng Duan of the University of Missouri. SGT-001 has been
granted Rare Pediatric Disease Designation, or RPDD, in the United
States and Orphan Drug Designations in both the United States and
European Union.
About Solid BiosciencesSolid Biosciences is a
life science company focused solely on finding meaningful therapies
for Duchenne muscular dystrophy (Duchenne). Founded by those
touched by the disease, Solid is a center of excellence for
Duchenne, bringing together experts in science, technology and care
to drive forward a portfolio of candidates that have life-changing
potential. Currently, Solid is progressing programs across four
scientific platforms: Corrective Therapies, Disease-Modifying
Therapies, Disease Understanding and Assistive Devices. For more
information, please visit www.solidbio.com.
Forward-Looking StatementsThis press release
contains “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements regarding the Company’s IGNITE DMD clinical trial, the
safety or potential efficacy of SGT-001 and other statements
containing the words “anticipate,” “believe,” “continue,” “could,”
“estimate,” “expect,” “intend,” “may,” “plan,” “potential,”
“predict,” “project,” “should,” “target,” “would,” “working” and
similar expressions. Any forward-looking statements are based on
management’s current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in,
or implied by, such forward-looking statements. These risks and
uncertainties include, but are not limited to, risks associated
with Solid’s ability to satisfactorily respond to requests from the
FDA for further information and data regarding IGNITE DMD;
successfully resolve the clinical hold with regard to IGNITE DMD;
obtain and maintain necessary approvals from the FDA and other
regulatory authorities and investigational review boards at
clinical trial sites; enroll patients in its clinical trials;
continue to advance SGT-001 in clinical trials; replicate in
clinical trials positive results found in preclinical studies and
earlier stages of clinical development; advance the development of
its product candidates under the timelines it anticipates in
current and future clinical trials; successfully scale its
manufacturing process; obtain, maintain or protect intellectual
property rights related to its product candidates; compete
successfully with other companies that are seeking to develop
DMD/Duchenne treatments and gene therapies; manage expenses; and
raise the substantial additional capital needed to achieve its
business objectives. For a discussion of other risks and
uncertainties, and other important factors, any of which could
cause the Company’s actual results to differ from those contained
in the forward-looking statements, see the “Risk Factors” section,
as well as discussions of potential risks, uncertainties and other
important factors, in the Company’s most recent filings with the
Securities and Exchange Commission. In addition, the
forward-looking statements included in this press release represent
the Company’s views as of the date hereof and should not be relied
upon as representing the Company’s views as of any date subsequent
to the date hereof. The Company anticipates that subsequent events
and developments will cause the Company's views to change. However,
while the Company may elect to update these forward-looking
statements at some point in the future, the Company specifically
disclaims any obligation to do so.
Investor Contact: Carlo Tanzi, Ph.D. Kendall
Investor Relations 617-337-4680 investors@solidbio.com
Media Contact: Courtney Heath ScientPR
416-301-7966 media@solidbio.com
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