Laru-zova sustains visual function improvements at 12 months in XLRP gene therapy trial

Beacon Therapeutics has announced positive 12-month safety and efficacy results from the phase 2 DAWN trial of laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP), presenting the data in an oral session at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, held in Denver, Colorado, from May 3-7, 2026. The findings confirm sustained improvements in visual function through 1 year, extend the pattern established at the 9-month interim readout, and advance the clinical case for laru-zova ahead of pivotal VISTA trial topline data expected in the second half of 2026.

DAWN (NCT06275620) is an ongoing, fully enrolled, phase 2, open-label trial evaluating laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The trial assesses 2 dose levels—high and low—for efficacy, safety, and tolerability, and is the first study in the laru-zova program to collect and evaluate low luminance visual acuity (LLVA) as an end point, the same measure being used as a primary endpoint in the pivotal VISTA trial.

12-month results from the DAWN trial

The 12-month data showed sustained improvements across both primary efficacy measures. In the LLVA analysis, 50% of participants who received the high dose achieved at least a 2-line (10 or more ETDRS letters) improvement from baseline in study eyes, with 25% of high-dose participants achieving at least a 3-line (15 or more ETDRS letters) improvement. In the low-dose group, 67% of participants achieved at least a 2-line improvement from baseline. Microperimetry data showed sustained improvement in mean macular sensitivity compared to baseline across both dose groups. The company described these findings as concordant with the 9-month results previously reported at EURETINA 2025.²

On the safety side, laru-zova continued to be generally well-tolerated. Ocular treatment-emergent adverse events (AEs) were mostly mild or moderate in severity, with the majority attributed to the surgical procedure and corticosteroid regimen rather than the investigational 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."

Looking ahead to the VISTA trial

The DAWN 12-month results arrive with the pivotal VISTA trial (NCT04850118) fully enrolled and on track. VISTA is a global, randomized, controlled, masked, multicenter phase 2/3 study evaluating laru-zova in 2 study groups compared with an untreated control, with LLVA improvement and change from baseline in mean microperimetry sensitivity as the primary end points—the same measures showing sustained benefit in DAWN. The trial enrolled male patients aged 12 to 50 years across sites in North America, the United Kingdom, and Australia, exceeding initial enrollment projections when it completed enrollment in June 2025. Topline data are expected in the second half of 2026.

Laru-zova carries both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, Priority Medicines (PRIME) designation from the European Medicines Agency, Innovative Licensing and Access Pathway designation from the UK's MHRA, and Orphan Drug Designation from both the FDA and EMA. This regulatory profile reflects both the unmet need of the disease as well as the strength of the clinical evidence accumulated to date.

Three additional laru-zova posters were also presented at ARVO 2026, including the design and baseline characteristics of VISTA, a post-hoc microperimetry analysis from the phase 2 SKYLINE trial, and 5-year safety results from the completed phase 1/2 HORIZON trial—collectively representing one of the most comprehensive presentations of gene therapy data in inherited retinal disease at this year's meeting.

At the 2025 American Society of Retinal Specialists (ASRS) annual meeting, held in Long Beach, California, Ophthalmology Times spoke with Robert A. Sisk, MD, FACS, FASRS, from the Cincinnati Eye Institute, about the phase 2 SKYLINE trial. The key findings at the time revealed a significant improvement in microperimetry response in the high-dose group compared to the low-dose group. The research highlighted the importance of the ellipsoid zone in gene therapy, with patients having near-normal retinal anatomy more likely to benefit from the treatment.

X-linked retinitis pigmentosa: Disease background and unmet need

X-linked retinitis pigmentosa is an inherited retinal disease that predominantly affects males, caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. These mutations result in progressive photoreceptor loss beginning in childhood, with worsening night and peripheral vision, visual field constriction, and eventual blindness. The condition affects approximately 1 in 25,000 males in the US, Europe, and Australia and accounts for roughly 10% of the estimated 100,000 retinitis pigmentosa patients in the United States. There are currently no approved treatments.

Laru-zova is designed to restore the natural function of both rods and cones by delivering a functional copy of the RPGR ORF15 gene to produce the full-length protein—an approach intended to address the underlying molecular deficit driving photoreceptor degeneration in XLRP.

REFERENCES
1. Beacon Therapeutics Holdings Limited. 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. GlobeNewswire. May 7, 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
2. Beacon Therapeutics Announces Positive Interim 9+ Month Results from DAWN Trial and 36-Month Phase 2 SKYLINE Trial Data for Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at EURETINA 2025 - Beacon Therapeutics. Beacon Therapeutics. Published September 4, 2025. Accessed September 8, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-announces-positive-interim-9-month-results-fromdawn-trial-and-36-month-phase-2-skyline-trial-data-for-laru-zova-in-patients/