Beacon Therapeutics Reports Positive 24-Month Phase 2 SKYLINE Trial Data for AGTC-501 in X-Linked Retinitis Pigmentosa

Beacon Therapeutics Holdings Limited, a prominent gene therapy company focused on ophthalmology, has disclosed the interim results from their 24-month Phase 2 SKYLINE trial. This trial investigates the safety and efficacy of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP), with the findings presented at the American Academy of Ophthalmology’s Annual Meeting in Chicago, Illinois.

The trial's data indicated that 57% of the study participants (4 out of 7) who received a high dose (6.8 E+11 vg/eye) of AGTC-501 exhibited a positive response. This response was defined as an improvement in retinal sensitivity, evaluated by microperimetry, showing an increase of at least 7 decibels (dB) in at least five loci. Additionally, these patients demonstrated a significant improvement in average retinal sensitivity. On the other hand, a lower dose of AGTC-501 (7.5 E+10 vg/eye) yielded a 25% response rate (1 out of 4 patients). The trial reported no major safety issues linked to AGTC-501, with most adverse events being non-serious and mild to moderate in severity.

XLRP is a severe, inherited retinal disease that commonly leads to blindness by middle age, and currently, there are no available treatments. The disease affects approximately 3.4 to 4.4 per 100,000 young males with RPGR mutations across the US, Europe, and Australia. AGTC-501 aims to express the full-length RPGR protein, potentially addressing the extensive damage to photoreceptors caused by XLRP, which includes both rod and cone cell loss. This comprehensive approach positions AGTC-501 as a promising treatment for improving the lives of XLRP patients.

Lance Baldo, MD, CEO of Beacon Therapeutics, commented on the findings, highlighting AGTC-501’s promising safety profile and significant improvements in mean retinal sensitivity. He emphasized the importance of continuing to evaluate the long-term safety and effectiveness of AGTC-501, expressing optimism based on the current results from the SKYLINE trial.

Beacon Therapeutics is also advancing its clinical development with other trials, including the pivotal Phase 2/3 VISTA and the open-label Phase 2 DAWN trials, aiming to develop a reliable treatment for XLRP.

The interim results of the Phase 2 SKYLINE trial were presented by Dr. Robert Sisk, a distinguished ophthalmologist and retinal surgeon from the University of Cincinnati and Cincinnati Eye Institute. Dr. Sisk also serves as the Director of Pediatric Retinal Surgery and Ophthalmic Genetics at Cincinnati Children’s Hospital.

Beacon Therapeutics was established in 2023 with the mission to save and restore vision in patients suffering from various retinal diseases that lead to blindness. The company boasts a strong scientific foundation and is currently developing AGTC-501 as a treatment for XLRP. Additionally, Beacon Therapeutics is working on preclinical programs targeting dry age-related macular degeneration (AMD) and inherited cone-rod dystrophy (CRD). The company utilizes a target generation technology platform to identify and screen new therapeutic proteins in the field of ophthalmology.

Supported by investments from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences, and other investors, Beacon Therapeutics is committed to pioneering advancements in ophthalmic gene therapy and improving patient outcomes.