Beacon Therapeutics Reports Sustained 12-Month Visual Gains With Laru-Zova in XLRP

This story first appeared on Ophthalmology Times, a sister publication to CGTLive.

Beacon Therapeutics has reported positive 12-month safety and efficacy findings from the phase 2 DAWN trial evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP). The data were presented during an oral session at the 2026 Association for Research in Vision and Ophthalmology Annual Meeting, held May 3-7 in Denver, Colorado. According to the company, the latest results demonstrated sustained improvements in visual function through 1 year, building on the 9-month interim findings previously shared at EURETINA 2025 and further supporting the therapy’s advancement ahead of topline results from the pivotal phase 2/3 VISTA trial expected later this year.

The ongoing, fully enrolled phase 2 DAWN study (NCT06275620) is assessing laru-zova in the fellow eye of male patients with XLRP who had previously received an adeno-associated virus (AAV)-based gene therapy delivering the full-length RPGR protein. Participants were assigned to either a high- or low-dose cohort to evaluate the therapy’s safety, tolerability, and efficacy. DAWN also represents the first laru-zova study to prospectively evaluate low luminance visual acuity (LLVA), an outcome that serves as a primary endpoint in the ongoing VISTA trial.

At 12 months, investigators observed sustained improvements across the trial’s primary efficacy measures. In the high-dose cohort, 50% of treated participants experienced at least a 2-line improvement from baseline in LLVA, equivalent to a gain of 10 or more ETDRS letters, while 25% achieved gains of at least 3 lines, or 15 or more ETDRS letters. Among those receiving the low dose, 67% achieved at least a 2-line improvement from baseline. Investigators also reported sustained increases in mean macular sensitivity on microperimetry testing across both dose groups, findings the company said were consistent with the previously reported 9-month data set.

Laru-zova continued to demonstrate a generally favorable safety profile through 12 months of follow-up. Most ocular treatment-emergent adverse events were classified as mild or moderate, with many attributed to the surgical procedure or corticosteroid regimen rather than the investigational gene therapy itself.

“These new data continue to showcase the safety and tolerability of laru-zova, along with sustained improvements across key measures of visual function in participants' study eyes,” Daniel Chung, DO, MA, chief medical officer of Beacon Therapeutics, said in a statement. “As we continue to build one of the most significant bodies of evidence for a gene therapy in ocular disease, these findings further support the ongoing clinical development of laru-zova and reinforce its potential as a meaningful treatment option for people living with XLRP.”

The DAWN findings arrive as Beacon continues enrollment follow-up in the pivotal VISTA trial (NCT04850118), a global, randomized, masked, controlled phase 2/3 study evaluating laru-zova across 2 treatment groups compared with an untreated control arm. The study is using improvement in LLVA and change from baseline in mean microperimetry sensitivity as co-primary endpoints, mirroring the efficacy measures that demonstrated sustained benefit in DAWN. VISTA enrolled male patients aged 12 to 50 years across sites in North America, the United Kingdom, and Australia, ultimately surpassing initial enrollment targets when recruitment concluded in June 2025. Topline data are anticipated in the second half of 2026.

Regulatory momentum for laru-zova has also continued to grow. The investigational therapy has received Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA, as well as Priority Medicines designation from the European Medicines Agency, Innovative Licensing and Access Pathway designation from the UK Medicines and Healthcare products Regulatory Agency, and Orphan Drug designation from both US and European regulators. Collectively, these designations underscore both the unmet clinical need in XLRP and the strength of the emerging clinical data supporting the therapy.

In addition to the DAWN presentation, Beacon showcased 3 other laru-zova-related posters at ARVO 2026. These included presentations detailing the design and baseline characteristics of the VISTA study, a post-hoc microperimetry analysis from the phase 2 SKYLINE trial, and 5-year safety findings from the completed phase 1/2 HORIZON study. Together, the presentations represented one of the largest collections of inherited retinal disease gene therapy data presented at the meeting.

Earlier findings from the phase 2 SKYLINE trial were also highlighted at the 2025 American Society of Retinal Specialists Annual Meeting in Long Beach, California. At that meeting, Robert A. Sisk, MD, FACS, FASRS, of the Cincinnati Eye Institute, discussed data showing significantly greater microperimetry improvements in the high-dose cohort compared with the low-dose group. The findings also emphasized the importance of preserving the ellipsoid zone, suggesting that patients with more intact retinal anatomy may derive greater benefit from gene therapy treatment.

REFERENCE
1. Beacon Therapeutics Announces Positive 12-Month Safety and Efficacy Update from Phase 2 DAWN Trial of laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at ARVO 2026 Annual Meeting. News release. Beacon Therapeutics. May 7, 2026. Accessed May 8, 2026. https://www.globenewswire.com/news-release/2026/05/07/3290452/30580/en/beacon-therapeutics-announces-positive-12-month-safety-and-efficacy-update-from-phase-2-dawn-trial-of-laru-zova-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-at-arvo-2026-ann.html