Beacon Therapeutics announced positive 24-month interim safety and efficacy results from its Phase 2 SKYLINE trial of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP) at the American Academy of Ophthalmology's Annual Meeting in Chicago, Illinois. The data demonstrated sustained efficacy and a favorable safety profile, supporting the ongoing clinical development of AGTC-501.
The 24-month data from the SKYLINE trial indicated that AGTC-501 was generally safe and well-tolerated, with no clinically significant safety events associated with the treatment. In the high-dose cohort (6.8 E+11 vg/eye), 57% (4/7) of study eyes showed a response, defined as an improvement in retinal sensitivity of at least 7 decibels (dB) in at least 5 loci, as assessed by microperimetry. Patients in the high-dose cohort also showed a robust improvement in mean retinal sensitivity. The low-dose cohort (7.5 E+10 vg/eye) showed a response rate of 25% (1/4).
Continued Clinical Development
Beacon Therapeutics is currently enrolling patients in its pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials to further evaluate AGTC-501 in XLRP. These trials, along with the SKYLINE data, are expected to support regulatory submissions in the US and EU.
XLRP and AGTC-501
XLRP is a severe, aggressive, inherited retinal disease that leads to blindness by middle age, with limited treatment options. It primarily affects young males, with an estimated prevalence of 3.4 to 4.4 per 100,000 individuals in the US, Europe, and Australia. The disease is caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
AGTC-501 is an adeno-associated virus (AAV) vector-based gene therapy designed to express the full-length RPGR protein. By addressing the entirety of photoreceptor damage, including both rod and cone loss, AGTC-501 aims to improve the lives of patients with XLRP.
Management Commentary
Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics, stated, "Our Phase 2 SKYLINE 24-month data reinforces AGTC-501's favorable safety profile and robust improvements in mean retinal sensitivity. We will continue to assess the long-term safety and durability of AGTC-501 but are encouraged by the results we've seen in the SKYLINE trial to date."
