Applied Genetic Technologies Corporation announced the presentation of additional positive findings from the ongoing X-linked retinitis pigmentosa (XLRP) Phase 1/2 study of AGTC-501, including 18-month safety and efficacy data, at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting, May 1-4, 2022 in Denver, Colorado. The data presented at ARVO update previously reported data from this non-randomized, open-label Phase 1/2 study that the Company believes showed both safety and biological activity of AGTC-501 12 months after treatment. The 18-month data appear to show safety and efficacy signals similar to the study's 12-month findings, including improvements to the ellipsoid zone (EZ).

In patients with XLRP, the EZ, a defined region within the photoreceptor layer of the retina, degenerates over time and is eventually lost. Eighteen months after treatment, of the eyes in the study with visible foveal EZ at baseline that underwent subretinal administration of AGTC-501, two thirds showed recovery of foveal EZ and nearly half had improved EZ appearance, which correlated with improvement in macular sensitivity. At 18 months post treatment, AGTC-501 appeared to be well-tolerated across a wide dose range.

The majority of adverse events were mild to moderate in severity, including those related to the subretinal injection procedure and importantly, immunological assessments did not indicate safety concerns.