Solid Biosciences Reports 1.5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001 - Seite 2

Functional Data

• NSAA scores at 1.5 years showed minimal change compared with baseline and suggest benefit after treatment when compared to trajectories typically observed in natural history data. Natural history analyses suggest that patients similarly aged to those enrolled in IGNITE DMD would normally be expected to exhibit a 4.5-point decline in NSAA over 1.5 years. In contrast, Patients 4-6 exhibited a mean decrease of 1.7 points (Range: -3 to 0 points) from baseline and a mean difference of +2.8 points compared with natural history data over the same time period.
• 6MWT distances were maintained 1.5 years post dosing, while natural history analyses suggest that similarly aged patients would normally be expected to exhibit a 63.5-meter decline over the same period. The mean increase in the 6MWT for Patients 4-6 at 1.5 years was 15.3 meters (Range: -17 to +56 meters) compared with baseline, and the mean difference compared with natural history data was +78.8 meters over the same period.
• The percent predicted FVC for Patients 4-6 continued to show stability or improvement 1.5 years following SGT-001 administration, while natural history analyses suggest that similarly aged patients would normally be expected to exhibit a decline of 7.5% over the same period. The mean improvement in percent predicted FVC from baseline to 1.5 years for Patients 4-6 was 4.1% (Range: +0.6% to +9.2%), and the mean difference compared with natural history data was +11.6% over the same time period.
  

Patient-Reported Outcome Measures
Patient-reported outcome measures showed meaningful sustained improvements at 1.5 years compared with baseline as assessed using the PODCI global (range of change from baseline of +7 to +18 points), sports (Range: +14 to +23 points), transfer (Range: -6 to +3 points) and upper extremity scales (Range: +2 to +9 points). Data from natural history studies demonstrate a decline in the global (7.6 points), sports (4.7 points) and transfer (14.9 points) scales over the same period of time.  

Safety Findings
No new drug-related safety findings have been reported in Patients 4-6, who have post-dosing periods of more than 1.5 years to 2.5 years, or any of Patients 1-8, who have post-dosing periods of more than 5 months to 3.5 years.

About SGT-001
Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.