Beacon Therapeutics Reports 36-Month Interim Results from Phase I/II HORIZON Trial of AGTC-501 in XLRP Patients

Beacon Therapeutics Holdings Limited, a prominent company in the field of ophthalmic gene therapy, recently shared pivotal 36-month interim results from its Phase I/II HORIZON trial of AGTC-501 at the 24th EURETINA Congress in Barcelona, Spain. The company's mission focuses on saving and restoring vision for patients suffering from debilitating retinal diseases.

The HORIZON trial evaluated the safety and efficacy of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP), a genetic disorder characterized by progressive vision loss. The trial enrolled 29 patients, all of whom have tolerated the therapy well with no significant safety concerns reported. Importantly, the data revealed a sustained difference in visual function between the treated and untreated eyes even after 36 months. This favorable benefit-risk profile underscores the potential of AGTC-501 for ongoing clinical development in treating XLRP caused by mutations in the RPGR gene.

Dr. Lance Baldo, CEO of Beacon, emphasized the importance of these longer-term data, noting that they offer clinical validation for the safety of AGTC-501 in treating XLRP. He also highlighted the company's upcoming milestones, including the release of 24-month data from the Phase 2 SKYLINE trial and continued patient enrollment in the open-label Phase 2 DAWN trial and Phase 2/3 VISTA trial.

HORIZON is structured as a Phase 1/2, open-label, dose-escalation study involving subretinal administration of AGTC-501. The trial has successfully completed enrollment, and all participants are currently in long-term follow-up. The 36-month interim results were presented by Dr. Paul Yang, Chief of the Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute, underscoring the clinical expertise backing the trial.

Founded in 2023, Beacon Therapeutics aims to combat a variety of retinal diseases that lead to blindness through advanced gene therapy techniques. The company is built on a solid scientific foundation, boasting a late-stage development candidate for XLRP and two additional preclinical programs targeting dry age-related macular degeneration (AMD) and cone-rod dystrophy (CRD). Leveraging a target generation technology platform, Beacon is equipped to identify and screen secreted proteins pertinent to ophthalmology.

AGTC-501, the company's lead product, is designed to treat XLRP, an inherited disorder that results in progressive vision loss, predominantly affecting boys and young men. The condition is mainly caused by mutations in the RPGR gene, which are addressed by AGTC-501 through the expression of the full-length RPGR protein. This approach aims to mitigate photoreceptor damage, including both rod and cone loss, associated with XLRP.

Beacon Therapeutics benefits from the financial backing of notable investors such as Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, and Advent Life Sciences, among others. The company's rapid progress and robust support illustrate its potential to transform the treatment landscape for blinding retinal diseases.