- Topline data expected in H2 2026
LONDON and CAMBRIDGE, Mass., July 08, 2025 (GLOBE NEWSWIRE) -- Beacon Therapeutics Holdings Limited (“Beacon Therapeutics” or ”the Company”), a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, today announces that it has successfully completed enrollment in its registrational Phase 2/3 VISTA trial evaluating laru-zova for the treatment of X-linked retinitis pigmentosa (XLRP).
The study enrolled eligible male patients aged 12 to 50 across sites in North America, the United Kingdom, and Australia, exceeding the initial enrollment projection – a reflection of the strong interest from both patients and investigators in laru-zova. Twelve month topline data from this trial are expected in the second half of 2026.
The VISTA trial (NCT04850118) is a global, randomized, controlled, masked, multi-center Phase 2/3 study designed to evaluate the efficacy, safety, and tolerability of laru-zova in male patients with XLRP caused by mutations in the RPGR gene. The trial is assessing two dose levels of laru-zova compared to an untreated control group, evaluating the proportion of participants with improvement in low-luminance visual acuity (LLVA) and mean sensitivity as observed by microperimetry, among other measures of visual function.
Beacon plans to use the data from the VISTA trial, alongside long-term data from the ongoing Phase 2 DAWN trial, the Phase 1/2 HORIZON and Phase 2 SKYLINE studies to support regulatory submissions in the United States and Europe.
“Completion of enrollment in our VISTA trial marks a significant milestone for Beacon and, more importantly, for patients in the XLRP community,” said Lance Baldo, M.D., Chief Executive Officer of Beacon Therapeutics. "We thank our clinical trial sites, investigators and most importantly the study participants and their families who made this possible.”
“The VISTA study has been carefully designed to provide the clinical evidence needed to demonstrate laru-zova's potential to improve functional vision in patients with XLRP,” added Dr. Daniel Chung, D.O., M.A., Chief Medical Officer of Beacon Therapeutics. “We are applying our deep understanding of both ocular disease and gene therapy by using a highly efficient AAV capsid and a stabilized gene cassette that expresses the full-length RPGR protein, to support better vision outcomes. We are cautiously optimistic about the potential for laru-zova to treat a condition that has long been considered untreatable.”
Beacon continues to progress its Phase 2 DAWN trial, an open-label study 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. In May, the Company announced six-month interim results from DAWN, demonstrating greater 2- and 3-line improvements in study eyes compared to previously treated fellow eyes, and a safety profile consistent with previous studies.
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About laru-zova
Laru-zova (laruparetigene zovaparvovec) is a potential best-in-class gene therapy currently being investigated 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 VISTA and DAWN Studies
VISTA (NCT04850118) is a Phase 2/3, randomized, controlled, masked, multi-center 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.
DAWN (NCT06275620) is a Phase 2, open-label study 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.
About XLRP
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 with a mission to save and restore the vision of patients with blinding retinal diseases. The Company has an established scientific foundation that includes a late-stage clinical development candidate to treat XLRP and two preclinical programs targeting dry age-related macular degeneration (AMD) and an inherited cone-rod dystrophy (CRD).
Beacon Therapeutics’ investors include Syncona Limited, Forbion, Oxford Science Enterprises, TCGX and Advent Life Sciences, among others. Learn more about Beacon Therapeutics at beacontx.com.
