Insights:Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

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.


Your Solid Biosciences team