Beacon Therapeutics Reports Positive Interim Phase 2 Data in X-Linked Retinitis Pigmentosa

LONDON and CAMBRIDGE, Mass. – May 6, 2025 (GLOBE NEWSWIRE) — Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’), a clinical-stage biotechnology company dedicated to developing therapies to preserve and restore vision in patients with blinding retinal diseases, today announced encouraging 6-month interim safety and efficacy findings from its Phase 2 DAWN trial of laru-zova (laruparetigene zovaparvovec) in individuals with X-linked retinitis pigmentosa (XLRP). These results were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting, held in Salt Lake City from May 4-8, 2025. The interim analysis indicated that laru-zova was generally well-tolerated among all DAWN participants evaluated at or beyond 6 months, with initial data suggesting promising improvements in various key measures of visual function.

“The accumulated safety and efficacy data on laru-zova from three distinct clinical studies over the past five years are compelling,” stated Lance Baldo, M.D., Chief Executive Officer of Beacon Therapeutics. “We are pleased to share this 6-month update from the DAWN Phase 2 study, which continues to demonstrate laru-zova’s potential to enhance vision in patients with XLRP, evidenced by improvements across multiple assessments of visual function. We remain focused on advancing this exciting investigational treatment option for individuals affected by XLRP.”

XLRP is a genetic retinal disease frequently caused by mutations in the RPGR gene, affecting approximately 1 in 25,000 males in the U.S., Europe, and Australia. This condition often leads to blindness by middle age, and currently, there are no approved treatments available. Laru-zova is a potential best-in-class gene therapy designed to restore the natural function of both rod and cone photoreceptor cells in XLRP. It achieves this by delivering a functional copy of the RPGRORF15 gene using a well-established viral vector with a proprietary capsid engineered for efficient delivery to photoreceptors, along with a codon-optimized gene designed to produce the full-length RPGR protein.

Key data highlights include:

• Ocular treatment-emergent adverse events (TEAEs) were predominantly non-serious and mild to moderate in severity, with the majority being related to the surgical procedure and the use of steroids as required by the study protocol. These events have since resolved. Notably, there were no reports of suspected unexpected serious adverse reactions, retinal detachments, or endophthalmitis.
• The data demonstrated early improvements in low luminance visual acuity (LLVA), a crucial measure of visual function, with a greater number of participants showing two and three line improvements in their treated eyes compared to their previously treated fellow eyes at or beyond the 6-month evaluation.
• Data also revealed early and sustained improvements in mean retinal sensitivity in the treated eyes, as measured by microperimetry, indicating enhanced visual function in participants evaluated at or beyond month 6.

The DAWN study is an open-label trial investigating laru-zova in participants with XLRP who have previously received treatment with a full-length AAV vector-based gene therapy targeting the RPGR protein. The study aims to evaluate the efficacy, safety, and tolerability of two different dose levels of laru-zova in the untreated eye of participants who had previously undergone gene therapy for XLRP.

Beacon Therapeutics is actively continuing to enroll patients in its pivotal Phase 2/3 VISTA trial, which is also evaluating laru-zova for the treatment of XLRP.