Item 8.01 Other Events.
On October 1, 2020, Solid Biosciences Inc. (the "Company") announced that the
U.S. Food and Drug Administration (the "FDA") lifted the clinical hold placed on
the Company's IGNITE DMD Phase I/II clinical trial. As previously announced in
July 2020, the FDA had requested further manufacturing information, updated
safety and efficacy data for all patients dosed, and provided direction on total
viral load to be administered per patient. Based on the Company's response to
these requests, the FDA acknowledged that the Company satisfactorily addressed
all clinical hold questions.
In connection with the lifting of the clinical hold, the Company is reducing the
maximum weight of the next two patients dosed in IGNITE DMD to 18 kg per
patient, with safety outcomes from these two patients driving potential weight
increase of patients dosed subsequently. This reduction, in conjunction with the
delivery of fewer viral particles as a result of the Company's manufacturing
process improvements, will reduce patients' total viral load while continuing
dosing at the 2E14 vg/kg dose. Additionally, to mitigate the risk of serious
drug-related adverse events, the Company is amending the IGNITE DMD clinical
protocol to include the prophylactic use of both anti-complement inhibitor
eculizumab and C1 esterase inhibitor, and increasing the prednisone dose in the
first month post dosing.
The Company is currently working to complete all activities necessary to resume
dosing, which it expects to occur in the first quarter of 2021.
This Current Report on Form 8-K contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995, including
statements regarding the timing and ability of the Company to resume dosing and
move the IGNITE DMD clinical trial forward, 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
the Company's ability to resume and/or continue IGNITE DMD on the timeline
expected or at all; obtain and maintain necessary approvals from the FDA and
other regulatory authorities; obtain and maintain the necessary approvals from
investigational review boards at IGNITE DMD clinical trial sites and the IGNITE
DMD data safety monitoring board; enroll patients in IGNITE DMD; 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 optimize and
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,
on the timeline necessary, to resume dosing in the IGNITE DMD trial, continue
development of SGT-001, achieve its other business objectives and continue as a
going concern. 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 Current Report on Form 8-K 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.
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