Solid Biosciences Posts Two-Year Efficacy, Safety Data From Duchenne Gene Therapy Trial - Solid Biosciences (NASDAQ:SLDB)

Solid Biosciences Inc 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.

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