- Applied Genetic Technologies Corp (NASDAQ: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.
