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Solid Biosciences Reports 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),
About this update from Solid Biosciences Inc.
[{"type":"text","content":"- 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 enrollment of patients into IGNITE DMD continue - - Presentations to follow at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference - - Company to host conference call and webcast today at 4:30 PM ET to discuss clinical data and financial results - CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- 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...