Gene therapy demonstrates safety and efficacy in XLRP

AGTC-501, administered in 2 different doses, was generally safe and well-tolerated in patients with X-linked retinitis pigmentosa (XLRP), according to 24-month interim results of the Phase 2 SKYLINE trial presented at AAO 2024.

The treatment showed some signs of efficacy, with improvements in visual sensitivity, particularly in the high-dose group.

In the trial, 14 participants were randomized into low- and high-dose groups. At the 24-month mark, 11 participants had completed the study. No serious unexpected adverse reactions, cases of endophthalmitis, or early terminations due to adverse events were observed.

In the high-dose group, no ocular serious adverse events were reported; 2 occurred in the low-dose group, though neither was linked to AGTC-501. A small number of non-serious ocular adverse events were recorded in both groups.

In terms of efficacy, the mean sensitivity on microperimetry improved by 3.03 dB in the high-dose group compared to the low-dose group after 12 months.

Reference
Sisk RA, et al. Subretinal AGTC-501 Gene Therapy for XLRP: Twenty-Four-Month Interim Results of the Phase 2 SKYLINE Trial. Poster presented at: American Academy of Ophthalmology Annual Meeting; October 2024; Chicago.