Item 8.01. Other Events.
As previously reported in a Current Report on Form 8-K (the "Original 8-K")
filed on
FVC Functional Data
The 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 8.5% (Range: +0.6% to +22.5%), and the mean difference compared with natural history data was +16.0% over the same time period.
The following table summarizes the 1 year and 1.5 year FVC functional data of IGNITE DMD for patients treated with the high dose 2E14 vg/kg of SGT-001:
Summary of FVC Interim Functional Data of IGNITE DMD Absolute Change From Baseline to 1 Year and 1.5 Years FVC% FVC% 1 yr 1.5 yr Change Change 1.5 from from Baseline 1 yr yrs Baseline Baseline Patient Patient 4 Age at Baseline 10.7 years 93.7 96.8 94.3 +3.1 % +0.6 % Patient 5 6.8 years 73.4 110.1 95.9 +36.7 % +22.5 % Patient 6 7.7 years 104.2 111.5 106.7 +7.3 % 2.5 % Mean Change Patients 4-6 +15.7 % +8.5 %
--------------------------------------------------------------------------------
© Edgar Online, source