- Data show promising and sustained improvements across key measures of visual function, including low luminance visual acuity and mean macular sensitivity as measured by microperimetry.
- Laru-zova was generally well-tolerated through month 12, with data supporting ongoing clinical development for the treatment of patients with XLRP caused by mutations in the RPGR gene.
- Beacon remains on track to report topline data from the pivotal VISTA trial of laru-zova in the second half of 2026.
LONDON and CAMBRIDGE, Mass., May 07, 2026 (GLOBE NEWSWIRE) -- Beacon Therapeutics Holdings Limited (Beacon Therapeutics 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, today announced a 12-month safety and efficacy update from its Phase 2 DAWN trial evaluating the Company's lead program, laruparetigene zovaparvovec (laru-zova), in patients with XLRP in a presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting held May 3-7, 2026 in Denver, Colorado.
These data demonstrated that laru-zova was generally well-tolerated by DAWN trial participants through month 12 and showed sustained improvements across several key measures of visual function, including low luminance visual acuity (LLVA) and mean macular sensitivity as measured by microperimetry, concordant with the 9-month results previously reported by the Company.
"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," said Daniel Chung, D.O., M.A., Chief Medical Officer of Beacon Therapeutics. "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. We remain on track to report topline data from our pivotal VISTA trial in the second half of this year, and we look forward to advancing this important program in close collaboration with patients and regulators."
XLRP is an inherited retinal disease that leads to progressive vision loss and legal blindness, with no available treatment options. 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 RPGRORF15 gene designed to produce the full-length protein.
Key DAWN data highlights include:
- Laru-zova continued to be generally well-tolerated, and ocular treatment-emergent adverse events (TEAEs) were mostly mild or moderate in severity, with the majority attributed to the surgical procedure and corticosteroid regimen.
- Data continued to show sustained improvements in LLVA compared to baseline, representing enhanced visual function in participants evaluated through month 12.
- 50% of participants who received a high dose in study eyes achieved at least 2-line (10+ ETDRS letters) improvement from baseline, with 25% of participants who received a high dose achieving at least 3-line (15+ ETDRS letters) improvement from baseline.
- 67% of participants who received a low dose in study eyes achieved at least 2-line (10+ ETDRS letters) improvement from baseline.
- Data also showed sustained improvement in microperimetry mean sensitivity as compared to baseline.
Beacon is also evaluating the proportion of participants with improvement in LLVA and change from baseline in mean macular sensitivity as measured by microperimetry across the whole grid, among other measures of visual function, in its ongoing pivotal VISTA trial (NCT04850118) of laru-zova for the treatment of XLRP. Beacon completed enrollment of this trial in June 2025 and expects to report topline data in the second half of 2026.
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 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 FDA for use.
About the DAWN and VISTA Trials
DAWN (NCT06275620) is an ongoing, fully enrolled, Phase 2, open-label trial of 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 objective of DAWN is to assess two different dose levels of laru-zova for efficacy, safety and tolerability in the target population. DAWN is also evaluating the changes in visual function and functional vision, and is the first trial in the laru-zova clinical development program that is collecting and evaluating low luminance visual acuity (LLVA) data.
VISTA (NCT04850118) is an ongoing, fully enrolled 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.
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, Retinal Degeneration 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
