Business
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 – –
About this update from Solid Biosciences Inc.
[{"type":"text","content":"– 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 –\n – 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...