Full Press Release Details
Solid Biosciences Reports Positive Interim Efficacy and Safety Data from the Ongoing IGNITE DMD
Clinical Trial and Resumption of Patient Dosing in the 2E14 vg/kg Cohort
- Interim data from six patients provide evidence of a potential benefit of SGT-001 in functional
endpoints of North Star Ambulatory Assessments (NSAA), 6-minute walk test (6MWT), pulmonary function tests (PFTs), and clinically validated patient reported outcome measures (PROMs) -
- Patient 7 safely dosed with SGT-001 experienced transient and manageable adverse events, none of
which were serious; six patients previously dosed showed no new drug-related safety findings 17-37 months post dosing; Screening and enrolling of patients into IGNITE DMD continue -
- Presentations to follow at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific
- Company to host conference call and webcast today at 4:30 PM ET to discuss clinical data and
CAMBRIDGE, Mass., March 15, 2021 Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company
focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today reported encouraging interim functional (NSAA, 6MWT and PFTs) and biomarker data, and patient reported outcome measures (PROMs) from six patients after
treatment in the ongoing IGNITE DMD Phase I/II clinical trial of its lead gene therapy candidate, SGT-001. The Company also announced that patient 7 in IGNITE DMD was safely dosed, with transient and
manageable adverse events, none of which were serious. Patient 7 was the first patient dosed in IGNITE DMD under a previously reported clinical protocol amendment and using SGT-001 manufactured with its
second-generation process. Additionally, the six patients previously dosed showed no new drug-related safety findings, 17-37 months post dosing. The totality of data collected, and the re-initiation of dosing support the continued enrollment of patients into the IGNITE DMD study.
These data will also be
presented in an oral session and at a company-sponsored symposium at the 2021 MDA Virtual Clinical & Scientific Conference on Thursday, March 18.
The totality of the functional and biomarker data, as well as the patient reported outcome measures reported today suggest that SGT-001 may provide benefit to patients with Duchenne, a serious disease for which there is no cure, said Barry Byrne, M.D., Ph.D., Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center
at the University of Florida, and Principal Investigator of the IGNITE DMD clinical study. I am particularly encouraged by these early data when compared with the natural history of this disease. I look forward to the continued enrollment in
IGNITE DMD and evaluating the data as the study progresses.
We are encouraged with the successful resumption of dosing in the IGNITE DMD
trial under our amended clinical protocol and using SGT-001 manufactured with a second-generation process. The safe dosing of the seventh patient gives us increased confidence in our dosing strategy as we move
forward with clinical development in the IGNITE DMD clinical trial. We are grateful to this patient and his family, and to all those who choose to participate in clinical trials said Ilan Ganot, Chief Executive Officer, President and Co-Founder. We look forward to continuing to dose patients and reporting clinical outcomes from additional patients in the second half of 2021.
The data reported were collected from the first six patients dosed in IGNITE DMD twelve to twenty-four months after treatment and include data from three
patients dosed at the low dose (5E13 vg/kg) and three patients dosed at the high dose (2E14 vg/kg). Data from the delayed treatment cohort, analyzed as an untreated control cohort, were evaluated alongside representative natural history data. The
six patients ranged in age from five to 14-years-old at baseline. These data have been previously shared with FDA, as well with members of the IGNITE DMD Data Safety
Monitoring Board and clinical consultants.
Patient Reported Outcome Measures (PROMs) Data
Patient reported outcome measures taken after one year of treatment revealed a trend towards dose-ordered improvements in motor function subscales and
fatigability assessments, providing real-world evidence to support the clinical and biomarker findings of varying degrees of benefit to patients in the low and high dose cohorts.
As previously reported, three of
the first six patients dosed prior to the protocol amendments introduced in 2020 developed four serious adverse events (SAEs). All prior SAEs have fully resolved, and no new drug-related safety findings have been identified with post-dosing follow
up of 17-37 months. Additionally, as reported today, with resumption of dosing in IGNITE DMD, patient 7 was dosed safely with mild to moderate adverse events all of which have fully resolved. The resumption of
dosing was under an amended clinical protocol and using SGT-001 manufactured with an improved process, both of which are designed to enhance patient safety.
Data Presentations at MDA
Dr. Byrne will present
the IGNITE DMD efficacy and safety data during a virtual oral session at the MDA conference on Thursday, March 18, 2021 at 4:00 PM ET. Also, on March 18, the PROM data will be presented during a Company-sponsored lunch symposium at 12:00
p.m. ET. Registration for the MDA conference is required to attend the oral presentation and the lunch symposium. Registration information is available at: https://mdavirtualconference.org/en/registration.
MDA Presentation Details
Title: IGNITE-DMD: Phase I/II Ascending Dose Study of Single IV Infusion of SGT-001 Microdystrophin Gene Therapy for DMD: One Year Efficacy and Safety Results
Presentation Date: Thursday, March 18, 2021, 4:00 PM ET
Also, on March 18, Solid
Biosciences will sponsor a symposium, Real World Outcome Measures in Duchenne Muscular Dystrophy: Current and Novel Assessments of Meaningful Patient Benefit at 12 PM ET. The symposium will feature:
Conference Call Today at 4:30 PM ET
Management will host a webcast and conference call to review the IGNITE DMD data and will take questions on this data and Solid s financial results
on March 15, 2021, today at 4:30 PM ET.
A live webcast of the call will be available on the Company s website at www.solidbio.com under the
News & Events tab in the Investor Relations section, or by clicking here. Participants may also access the call, by dialing 866-763-0341 for
domestic callers or 703-871-3818 for international callers, referencing conference ID# 1669808.
The archived webcast will be available for in the News and Events section of the Company s website.
Solid s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to
address the underlying genetic cause of Duchenne. Duchenne is caused by 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 clinical 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, and Fast Track Designation in the United States and Orphan Drug
Designations in both the United States and European Union.
About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne.
Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy
candidate, SGT-001. 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 the ability of the Company to continue dosing patients in the IGNITE
DMD trial, the implication of interim clinical data, the safety or potential treatment benefits of SGT-001 in patients with DMD, the Company s expectations for reporting future data from the IGNITE DMD
trial, the Company s regulatory plans and timelines and other statements containing the words anticipate, believe, continue, could, estimate, expect, intend,
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 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 independent data safety monitoring board; enroll patients
in IGNITE DMD on the timeline expected; the Company s dosing strategy; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; whether the interim data presented in this release
will be predicative of the final results of the trial or will demonstrate a safe or effective treatment benefit of SGT-001; whether the methodologies, assumptions and applications we utilize to assess
particular safety or efficacy parameters will yield meaningful statistical results; 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 Duchenne treatments and gene therapies; manage expenses; and
raise the substantial additional capital needed, on the timeline necessary, to 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 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.