- Late-breaking oral presentation at Retina Subspecialty Day outlines data from Beacon’s pivotal VISTA trial, which met its FDA-endorsed primary endpoint with statistical significance
- 31.0% of participants in the high dose group (p=0.0019) and 24.1% of participants in the low dose group (p=0.0106) achieved an improvement of ≥15 letters in LLVA at Month 12 as compared to no responders in the untreated control group
- Early and sustained improvement in change from baseline in mean macular sensitivity as measured by microperimetry (MP) across the whole grid was observed in participants treated with laru-zova as compared with the untreated control group
- Laru-zova demonstrated a favorable safety and tolerability profile, consistent with prior trials
LONDON and CAMBRIDGE, Mass., Oct. 12, 2026 (GLOBE NEWSWIRE) -- Beacon Therapeutics (Beacon or the Company), a leading clinical-stage biotechnology company with a mission to save and restore vision in people with rare and prevalent ocular diseases, presented data from its registration-enabling VISTA trial evaluating its investigational program laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP) in a late-breaking oral presentation during Retina Subspecialty Day at the American Academy of Ophthalmology (AAO) Annual Meeting held October 9-12, 2026, in New Orleans, Louisiana.
Robert Sisk, MD, FACS, FASRS, Professor of Ophthalmology, Cincinnati Eye Institute presented the results in a late-breaking oral presentation on Saturday, October 10 at 4:13pm CDT entitled ‘Subretinal Gene Therapy Laru-zova for X-linked Retinitis Pigmentosa (XLRP): Pivotal VISTA Trial First-time Results’.
The presentation followed the Company’s prior announcement of the topline results from the pivotal VISTA trial on September 21. The trial met its FDA-endorsed primary endpoint, demonstrating statistically significant low luminance visual acuity (LLVA) responder rates at Month 12 compared to the untreated control group. In addition, laru-zova demonstrated a favorable safety and tolerability profile in the VISTA trial, consistent with prior trials.
“VISTA is the first pivotal trial in XLRP to meet its primary endpoint, marking an important milestone for the field and for people living with this devastating disease. The detailed data presented at AAO further strengthen our confidence in laru-zova, demonstrating a consistent treatment effect across multiple measures of visual function and a favorable safety profile,” said Daniel Chung, D.O., M.A., Chief Medical Officer of Beacon Therapeutics. “Together, these results provide compelling support for laru-zova’s potential as a first treatment option for people with XLRP and we look forward to working closely with regulators to advance laru-zova towards patients as quickly as possible.”
VISTA AAO Presentation Summary
Trial Design
VISTA (NCT04850118) is a randomized, controlled trial designed to evaluate the efficacy, safety, and tolerability of laru-zova in male patients with XLRP caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The pivotal trial evaluated 85 male participants aged 12 to 48 over a 12-month period and included two laru-zova dosing groups: a high dose group of 6.8 E+11 vg/eye and a low dose group of 3.7 E+11 vg/eye. Participants were randomized 1:1:1 to a high dose (n=29), low dose (n=29) or untreated control (n=27) group, and baseline characteristics and demographics were well-matched between treated and control groups.
Efficacy
A statistically significant proportion of participants achieved a ≥15-letter improvement in LLVA as compared to the untreated control group at Month 12, with 31.0% of participants in the high dose group (p=0.0019), 24.1% of participants in the low dose group (p=0.0106), and no participants in the untreated control group demonstrating this level of improvement.
Other secondary endpoints demonstrated supportive positive trends reinforcing consistency of effect across measures of visual function, including the proportion of participants achieving a ≥10 letter improvement in LLVA at Month 12 as well as improvements in change from baseline in mean macular sensitivity as measured by MP across the whole grid at Month 12 in treated participants as compared to the untreated control group.
At Month 12, 48.3% of participants in the high dose group (p=0.0002), 58.6% in the low dose group (p<0.0001), and 3.7% in the untreated control group achieved a ≥10-letter improvement in LLVA. Additionally, participants treated with laru-zova demonstrated early and sustained improvements in mean macular sensitivity as measured by MP across the whole grid compared with the untreated control group. The least-squares mean difference versus untreated control was 1.201 decibels (dB) in the high dose group (p=0.0614) and 1.312 dB in the low dose group (p=0.0405).
While not a defined endpoint of the VISTA trial, a ≥20-letter improvement in LLVA at Month 12 was also achieved by 13.8% of participants in the high dose group, 6.9% of participants in the low dose group, and no participants in the untreated control group.
Safety and Tolerability
Laru-zova demonstrated a favorable safety and tolerability profile, with predominantly mild to moderate ocular Treatment Emergent Adverse Events (TEAEs) observed that were balanced across treatment groups and largely attributed to the surgical procedure. There were no unexpected Adverse Events (AEs) related to laru-zova based upon previous clinical trial experience. Laru-zova-related TEAEs were reported in 25% of participants in the high dose group and 38% of participants in the low dose group. Two ocular serious adverse events were observed in the low dose group, both attributed to the surgical procedure, and both considered resolved by the Principal Investigators before the Month 12 visit.
The AAO data presentation can be viewed on our website here.
Beacon plans to engage with global regulatory authorities with the initiation of a rolling Biologics License Application submission planned for later this year. These results expand the substantial body of evidence supporting the safety and efficacy profile of laru-zova, which now includes five years of clinical experience across 110 treated participants.
About laru-zova
Laruparetigene zovaparvovec (laru-zova) is a potential best-in-class gene therapy currently being evaluated for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Laru-zova has the potential to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the full RPGRORF15 gene designed to produce the full-length protein. Laru-zova has Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA), Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Innovative Licensing and Access Pathway (ILAP) from the UK's Medicines and Healthcare products Regulatory Agency (MHRA), as well as Orphan Drug Designation (ODD) from the FDA and EMA.
Laru-zova is investigational and has not been approved by the FDA for use.
About VISTA
VISTA (NCT04850118) is a Phase 2/3 pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. The study will evaluate the proportion of participants with improvement from baseline in LLVA, mean sensitivity as observed by microperimetry, and additional measures of functional vision.
About X-linked Retinitis Pigmentosa
X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease that predominantly affects males, typically caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The mutations, which affect approximately 1 in 25,000 males in the U.S., Europe and Australia, result in progressive photoreceptor loss over time and visual dysfunction beginning in childhood, eventually leading to blindness and impacting quality of life with no approved treatments. RPGR mutations underlie at least 70% of XLRP cases, and patients typically progress to legal blindness by a median age of 45. It is typically caused by mutations to the retinitis pigmentosa GTPase regulator (RPGR) gene and affects about 10% of the 100,000 retinitis pigmentosa patients in the United States. Laru-zova is a potential best-in-class gene therapy designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the full RPGRORF15 gene designed to produce the full-length protein.
About Beacon Therapeutics
Beacon Therapeutics is a clinical-stage biotechnology company dedicated to saving and restoring sight for people living with rare and prevalent ocular diseases. The Company is harnessing the transformative power of gene therapy to deliver the most meaningful outcomes for severe ocular diseases. Beacon’s pipeline currently targets devastating blinding retinal diseases such as X-linked retinitis pigmentosa (XLRP) and geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
Beacon Therapeutics’ investors include Advent Life Sciences, Forbion, Life Sciences at Goldman Sachs Alternatives, Oxford Science Enterprises, the Gund Vision Fund, Syncona Limited, and TCGX, among others. Learn more about Beacon Therapeutics at beacontx.com and follow on LinkedIn for more updates.
Contact:
info@beacontx.com
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beacon@icrhealthcare.com
