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Item 8.01. Other Events.
On September 23, 2021, Solid Biosciences Inc. (the “Company”) issued a press release reporting 1.5-year functional data and patient-reported outcome measures (Pediatric Outcomes Data Collection Instrument) (“PODCI”) for patients four through six in the Company’s ongoing IGNITE DMD Phase I/II clinical trial of SGT-001, all of whom received 2E14 vg/kg of SGT-001 manufactured using the Company’s first-generation manufacturing process. The data will be presented at the World Muscle Society 2021 Virtual Congress on September 23, 2021.
North Star Ambulatory Assessment (“NSAA”) scores at 1.5 years post-dosing 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 four through six 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.
Six minute walk test (“6MWT”) distances were maintained 1.5 years post-dosing, while natural history analyses suggest that similarly aged patients to those enrolled in IGNITE DMD would normally be expected to exhibit a 63.5-meter decline over the same period. The mean increase in the 6MWT for patients four through six 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 time period.
The percent predicted forced vital capacity (“FVC”) for patients four through six continued to show stability or improvement 1.5 years following SGT-001 administration, while natural history analyses suggest that similarly aged patients to those enrolled in IGNITE DMD would normally be expected to exhibit a decline of 7.5% over the same time period. The mean improvement in percent predicted FVC from baseline to 1.5 years for patients four through six 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-body strength scales (Range: +2 to +9 points). Data from natural history studies suggest that patients similarly aged to those enrolled in IGNITE DMD would normally be expected to 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.
No new drug-related safety findings have been reported in patients four through six, who have post-dosing periods of more than 1.5 years to 2.5 years, or any of patients one through eight, who have post-dosing periods of more than five months to 3.5 years.
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
|SOLID BIOSCIENCES INC.|
|Date: September 23, 2021||By:|
Name: Ilan Ganot
Title: Chief Executive Officer