Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD showed SGT-001 microdystrophin protein expression and associated neuronal nitric oxide synthase (nNOS) function.

As previously reported, the IGNITE DMD clinical program was placed on hold. Solid has received the Clinical Hold letter from the FDA and is working to address the hold and the path forward. PPMD appreciates this update from Solid and their ongoing pursuit of gene therapy.

Solid’s Letter to the Duchenne Community:

Dear Duchenne Community,

This morning we issued a press release providing a business update, including new patient data from IGNITE DMD, Solid’s Phase I/II clinical trial of SGT-001. Our company is working with urgency and determination to address the IGNITE DMD clinical hold so we may continue to evaluate the ability of SGT-001 to help patients with Duchenne muscular dystrophy.

As you may recall, in December 2019, Solid announced biomarker data from two patients dosed in the 2E14 vg/kg dose cohort of IGNITE DMD. The data showed expression of SGT-001 microdystrophin and neuronal nitric oxide synthase (nNOS) function, providing further evidence of the therapeutic potential of SGT-001.

Today, we announced biomarker data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, including three-month biopsy data. Using immunofluorescence assays, 50%-70% of the muscle fibers were determined to express SGT-001 microdystrophin. Immunofluorescence also showed stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin. Inclusion of the dystrophin nNOS coding region in SGT-001 may result in unique activity, potentially providing important functional benefits such as diminished muscle fatigue and protection against muscle damage. Using western blot, expression was 8% of normal control samples. The levels of serum creatine kinase, a highly variable biochemical marker of muscle damage, declined from baseline.

We are encouraged by these data and continue to make progress on internal investigations and analyses to support the goal of resolving the IGNITE DMD clinical hold and advancing the SGT-001 program.

As always, we appreciate the continued support of the Duchenne community and we are looking forward to sharing our future progress.

Sincerely,

Your Solid Biosciences team

Click here to read the full letter.

Read Solid’s Press Release:

Solid Biosciences Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update

– Biopsy results from the third patient dosed at 2E14 vg/kg in the SGT-001 IGNITE DMD clinical trial provide further support for continued development –

– Solid continues to make progress to address the IGNITE DMD clinical hold and advance the next steps for the SGT-001 program –

CAMBRIDGE, Mass., March 12, 2020 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB) today reported financial results for the fourth quarter and full year ending December 31, 2019 and provided a business update. “We are working to advance our lead program, SGT-001, a gene therapy candidate for Duchenne muscular dystrophy. We are pleased that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD showed SGT-001 microdystrophin protein expression and associated neuronal nitric oxide synthase (nNOS) function, providing further evidence of the therapeutic potential of SGT-001. Our priority is to address the IGNITE DMD clinical hold so we can continue to evaluate the ability of SGT-001 to help patients with Duchenne,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences.

Recent Developments

• Today, Solid announced biomarker data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, the company’s Phase I/II clinical trial of SGT-001, including three-month biopsy data. Using immunofluorescence assays, 50%-70% of the muscle fibers were determined to express SGT-001 microdystrophin. Immunofluorescence also showed stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin. Inclusion of the dystrophin nNOS coding region in SGT-001 may result in unique activity, potentially providing important functional benefits such as diminished muscle fatigue and protection against ischemic muscle damage. Using western blot, expression was 8% of normal control samples. The levels of serum creatine kinase, a highly variable biochemical marker of muscle damage, declined from baseline.
• In January 2020, Solid announced changes to its organizational structure to create a leaner company focused on advancing SGT-001. The corporate changes implemented reduce the company’s planned corporate expenses and extend the expected cash runway.
• In December 2019, Solid announced biomarker data from two patients dosed in the 2E14 vg/kg dose cohort of IGNITE DMD. The data showed expression of SGT-001 microdystrophin and nNOS function that provides evidence SGT-001 could provide therapeutic benefit for patients with Duchenne.
• In November 2019, Solid reported that the U.S. Food and Drug Administration (FDA) placed IGNITE DMD on clinical hold following a serious adverse event in the sixth patient dosed. In December 2019, the company announced that the adverse event had fully resolved, and that the patient had resumed his normal activities. For all patients dosed in IGNITE DMD, any clinical or laboratory abnormalities observed following SGT-001 administration have fully resolved.

Click here to read the full press release.