Beacon registers positive 12-month data from phase 2 SKYLINE trial of AGTC-501 in patients with X-linked retinitis pigmentosa

Beacon Therapeutics Holdings Limited, a leading ophthalmic gene therapy company, presented interim results from the phase 2 SKYLINE trial for X-linked retinitis pigmentosa (XLRP) at the 47th Annual Macula Society Meeting in Palm Springs, California.

At twelve months, males with XLRP treated with a high dose (6.8 E+11 vg/eye) of AGTC-501 showed a 63% response rate, demonstrating improved retinal sensitivity. Low-dose (7.5 E+10 vg/eye) AGTC-501 and untreated eyes in the high-dose group had a 0% response rate. Notably, high-dose recipients experienced significant visual function improvement, including mean retinal sensitivity. AGTC-501 was well tolerated, with no significant safety concerns, and any adverse events were mild to moderate.

XLRP, often caused by RPGR gene mutations, lacks approved treatments. AGTC-501, expressing full-length RPGR protein, aims to restore both rod and cone photoreceptor function.

These findings highlight AGTC-501's potential for XLRP, addressing an unmet need in this debilitating orphan disease. Established in 2023, Beacon Therapeutics focuses on ophthalmic gene therapy to combat retinal diseases leading to blindness.