LONDON and CAMBRIDGE, Mass
September 21, 2026 — Beacon Therapeutics announced positive topline results from the pivotal VISTA trial of laruparetigene zovaparvovec (laru-zova) in male patients with X-linked retinitis pigmentosa (XLRP). The Phase 2/3 study met its FDA-endorsed primary endpoint, demonstrating statistically significant low luminance visual acuity (LLVA) responder rates at Month 12 compared with an untreated control group. In the high-dose group, 31.0% of participants achieved at least a 15-letter improvement in LLVA (p=0.0019), compared with 24.1% in the low-dose group (p=0.0106) and no responders in the untreated control group.
VISTA Demonstrates Significant Improvement in Visual Function
The VISTA study evaluated 85 male participants aged 12 to 48 years with XLRP caused by RPGR gene mutations, across high- and low-dose laru-zova groups and an untreated control group. Additional efficacy measures supported the primary endpoint, including improvements in mean macular sensitivity measured by microperimetry. At Month 12, the least-squares mean difference versus untreated control was 1.201 decibels in the high-dose group and 1.312 dB in the low-dose group. A ≥10-letter LLVA improvement was achieved by 48.3% of high-dose participants and 58.6% of low-dose participants, compared with 3.7% in the untreated control group.
Laru-zova Shows Favorable Safety Profil
Laru-zova demonstrated a favorable safety and tolerability profile consistent with previous clinical studies, according to Beacon. Most treatment-emergent adverse events were mild to moderate ocular events and were largely attributed to the surgical procedure. Laru-zova-related adverse events were reported in 25% of participants in the high-dose group and 38% in the low-dose group. Two ocular serious adverse events occurred in the low-dose group, both attributed to the surgical procedure. The VISTA findings expand Beacon’s clinical dataset to five years of experience across 110 treated participants in the laru-zova development program.
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Beacon Prepares Global Regulatory Submissions for Laru-zova
Beacon plans to begin pre-submission discussions with global regulatory authorities and initiate a rolling U.S. Biologics License Application (BLA) submission later in 2026. Additional VISTA results are scheduled for presentation at Retina Subspecialty Day during the American Academy of Ophthalmology Annual Meeting in New Orleans in October 2026. Laru-zova is designed to deliver a functional copy of the full RPGRORF15 gene using a subretinal gene therapy approach intended to restore retinal function in XLRP. The candidate has received FDA RMAT and Fast Track designations, EMA PRIME designation, UK ILAP designation and FDA and EMA Orphan Drug Designations, but remains investigational and has not been approved by the FDA.
Source : Beacon Therapeutics press release
