Applied Genetic's Vision Loss Gene Therapy Shows Encouraging Safety, Efficacy At Three Months

Applied Genetic Technologies Corp AGTC reported three-month interim data from its ongoing Phase 2 Skyline trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP), an inherited condition causing progressive vision loss in boys and young men.
AGTC-501 is a recombinant AAV viral vector-based gene therapy targeting mutations in the RPGR gene.
There were robust improvements in visual sensitivity, the trial's primary efficacy endpoint, in multiple patients three months after dosing, with a 62.5% response rate in dose group B and a 25% response rate in dose group A.
This is well above the statistically significant 50% response rate the Vista Phase 2/3 trial for XLRP is powered to detect.
The number of loci that improved by at least 7 dB was between 9 and 17.
Mean visual sensitivity of the entire macula increased, and the area of the macula with visual sensitivity also increased in the patients who were responders.
Although these results exceeded the current standard set by the FDA that at least 5 loci increase by at least 7 dB, the improved loci were not pre-specified, as also required in the FDA standard.
There was a significant difference in visual sensitivity in the treated eyes compared with the untreated eyes of all Skyline responders at 3 months.
AGTC-501 was generally well-tolerated, with no clinically relevant safety concerns attributed to the study agent.
Price Action: AGTC shares closed 5.05% lower at $0.71 during after-hours trading on Monday.