- Solid Biosciences Inc (NASDAQ:SLDB) has reported two-year interim efficacy and safety data from the first three patients treated with SGT-001 in the high dose (2E14 vg/kg) cohort as part of IGNITE DMD.
- Results suggest durable benefit 24-months post-administration of SGT-001 compared to natural history, consistent with results reported at the 12-month and 18-month periods.
- Data from Patients 4-6 suggest improved motor function at two years post-infusion, against expected natural history declines.
- In addition, the data suggested improved pulmonary function and sustained or improved patient-reported outcome measures compared to both baseline and natural history.
- Related: Solid Biosciences Stock Gains After Duchenne Dystrophy Gene Therapy Data.
- Solid Biosciences also reported data from skeletal muscle biopsies collected three months after infusion of SGT-001 from the most recently dosed Patients 7-9.
- All six patients dosed with SGT-001 in the high dose cohort have demonstrated microdystrophin expression and proper membrane localization.
- No new drug-related safety findings have been identified in Patients 1-9 in post-dosing periods of 90 days to approximately four years.
- The Company plans to conclude dosing patients as part of IGNITE DMD this year and begin End of Phase II discussions with the FDA.
- Solid held $207.8 million in cash, cash equivalents, and available-for-sale securities, sufficient to fund its operations and capital expenditures into Q3 of 2023.
- Price Action: SLDB shares are down 6.25% at $0.99 during the market session on the last check Monday.
