Beacon Therapeutics Reports Positive 3-Month Phase 2 DAWN Trial Data for laru-zova in XLRP Patients

To date, laru-zova has been well-tolerated by all participants in the Phase 2 DAWN study. Data show promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function. Data build confidence in laru-zova as a potential treatment for patients with XLRP. Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is currently enrolling.

Beacon Therapeutics Holdings Limited, a prominent ophthalmic gene therapy company focused on combating retinal diseases leading to blindness, revealed the three-month interim safety and efficacy results of its Phase 2 DAWN trial. These findings were presented at the FLORetina-ICOOR Meeting 2024 in Florence, Italy.

Key highlights from the presentation indicate that laru-zova has been well-tolerated by participants, with no treatment emergent adverse events (TEAEs) directly related to the study agent. Additionally, no ocular inflammatory adverse events were reported. The data also demonstrated promising early improvements in low luminance visual acuity (LLVA), which is a crucial measure of visual function.

The DAWN study is a non-randomized, open-label investigation of laru-zova in participants with XLRP who had previously undergone full-length AAV-vector based gene therapy targeting the RPGR protein. The objective of DAWN is to evaluate two different doses of laru-zova for their efficacy, safety, and tolerability in the untreated eye of participants who had already received gene therapy for XLRP.

XLRP, or X-linked retinitis pigmentosa, is a severe, inherited retinal disease that typically leads to blindness by middle age. It predominantly affects young males, with an estimated prevalence of 1 in 25,000 males in the US, Europe, and Australia suffering from XLRP due to RPGR mutations. Laru-zova expresses the full-length RPGR protein, potentially addressing the complete range of photoreceptor damage caused by XLRP, including both rod and cone loss. As such, it represents a potentially best-in-class treatment for progressive vision loss in patients with XLRP.

Dr. Lance Baldo, CEO of Beacon Therapeutics, expressed optimism regarding the early results from the Phase 2 DAWN study. He noted that the strong safety profile observed so far is bolstered by promising early improvements in low luminance visual acuity, a significant functional measure of vision in patients with XLRP. These findings not only support the ongoing pivotal VISTA study but also reinforce the company's commitment to bringing hope to patients and families affected by this debilitating disease.

Beacon Therapeutics is actively enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova, as part of its efforts to develop this potential treatment for XLRP. The company's lead development candidate, laru-zova (AGTC-501), is currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that leads to progressive vision loss, primarily affecting boys and young men. XLRP is mainly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. By expressing the full-length RPGR protein, laru-zova aims to address the full spectrum of photoreceptor damage caused by XLRP, encompassing both rod and cone loss.

Beacon Therapeutics was founded in 2023 with the mission to save and restore the vision of patients affected by various prevalent and rare retinal diseases that result in blindness. The company has a robust scientific foundation, combining a late-stage development candidate for X-linked retinitis pigmentosa and two preclinical programs targeting dry age-related macular degeneration (AMD) and inherited cone-rod dystrophy (CRD). Additionally, Beacon Therapeutics has access to a target generation technology platform designed to identify, screen, and search for secreted proteins within the ophthalmology field. The company is supported by funding from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, and Advent Life Sciences, among other investors.