CAMBRIDGE, Mass., March 14, 2018 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB) today announced it has
received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for SGT-001
microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.
IGNITE DMD is designed to assess the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents
with DMD. The first patient dosed in the clinical trial was a non-ambulatory adolescent who received 5E13 vg/kg of SGT-001 on
February 14, 2018. Several days after administration the patient was hospitalized due to laboratory findings that included a
decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation. The patient
showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests.
The patient responded well to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or
returned to normal, and he is continuing outpatient assessments per protocol.
Solid reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse
Reaction (SUSAR). The FDA informed the company that the clinical hold was due to the event. Solid has halted enrollment and dosing
in IGNITE DMD and is awaiting the formal Clinical Hold letter from the FDA to understand the requirements for resuming the clinical
trial. Solid will work closely with the Agency to resolve the Clinical Hold.
About SGT-001
Solid’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 DMD, 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 DMD, 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 Biosciences
Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD).
Founded by those touched by the disease, Solid is a center of excellence for DMD, 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 Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of
1995, including statements regarding Solid’s intentions regarding communications with the FDA and its ongoing IGNITE DMD clinical
trial. 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 and the partial clinical hold with respect to the high dose of SGT-001; 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 later clinical trials
positive results found in preclinical studies and earlier stage clinical trials of SGT-001 and its other product candidates;
advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain,
maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that
are seeking to develop DMD 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 our 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 our 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.
Contact:
Solid Biosciences
Kate Niazi-Sai
617-337-4680
media@solidbio.com
investors@solidbio.com