Beacon Therapeutics Begins VISTA Trial for AGTC-501

Beacon Therapeutics Holdings Limited, a key player in ophthalmic gene therapy, has initiated the treatment phase in its pivotal VISTA clinical trial with the investigational gene therapy AGTC-501 (laruparetigene zovaparvovec). The VISTA trial is designed to evaluate the efficacy, safety, and tolerability of AGTC-501 in treating X-Linked Retinitis Pigmentosa (XLRP), a severe inherited retinal disease that often leads to blindness by middle age.

The trial is a global, randomized, controlled, and masked multi-center study. It will assess two dose levels of AGTC-501 compared to an untreated control group. The study aims to enroll patients primarily in the United States, with additional sites planned in multiple countries. AGTC-501 expresses the full length RPGR protein, potentially addressing the entire spectrum of photoreceptor damage caused by XLRP.

Beacon Therapeutics plans to use data from the VISTA trial, along with information from previous Phase 1/2 HORIZON and Phase 2 SKYLINE studies, to support regulatory submissions in both the United States and Europe. The gene therapy has received several key designations to expedite its development and review process, including Innovative Medicine Designation (ILAP) in the UK, Priority Medicine (PRIME) in the EU, and Fast Track in the US.

Darin Curtiss, Global Vice President of Clinical Development at Beacon Therapeutics, highlighted the significance of this milestone, emphasizing the potential of AGTC-501 to improve or maintain vision in patients with XLRP. Dr. David Birch from the Retina Foundation of the Southwest echoed this sentiment, expressing optimism about the trial’s potential to bring hope to thousands of young men affected by XLRP.

XLRP is considered an orphan disease, affecting around 17,000 patients in the US and EU5. It is predominantly caused by mutations in the RPGR gene. The disease is aggressive and inherited, with no current treatment options available. By expressing the full length RPGR protein, AGTC-501 aims to restore function to both rod and cone photoreceptors, positioning it as a promising treatment for XLRP.

Beacon Therapeutics, established in 2023, is dedicated to saving and restoring vision for patients with various retinal diseases. The company’s leading development candidate, AGTC-501, targets XLRP, a monogenic recessive disorder causing progressive vision loss in males. Besides AGTC-501, Beacon is also advancing preclinical programs for conditions like dry age-related macular degeneration (AMD) and cone-rod dystrophy (CRD).

The company’s scientific foundation is strengthened by its access to a target generation technology platform, which aids in identifying and screening potential therapies in the ophthalmology field. Beacon Therapeutics is backed by funding from Syncona and other investors, including Oxford Science Enterprises.

The successful advancement of AGTC-501 through the VISTA trial represents a significant step towards providing a viable treatment for XLRP, offering new hope to those affected by this debilitating condition.