Beacon Therapeutics has completed the enrolment in its randomised, registrational Phase II/III VISTA trial of laru-zova (laruparetigene zovaparvovec) as a potential treatment for X-linked retinitis pigmentosa (XLRP).
XLRP is a hereditary retinal disease that primarily affects males.
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The controlled, global, masked, multi-centre trial has enrolled male subjects aged 12 to 50 across sites in Australia, North America, and the UK, surpassing the initial enrolment projections.
It is designed to assess the safety, tolerability, and efficacy of the therapy in males with XLRP occurred due to mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
It is evaluating two dose levels of the therapy against an untreated control group, focusing on the proportion of subjects showing improvements in low-luminance visual acuity and mean sensitivity as measured by microperimetry, among other visual function metrics.
Beacon noted that the twelve-month top-line data from the VISTA trial are anticipated in the second half of 2026.
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By GlobalDataThe company plans to utilise the VISTA trial data, in conjunction with long-term data from the ongoing Phase II DAWN trial and the Phase I/II HORIZON and Phase II SKYLINE trials, to back regulatory submissions in the US and Europe.
The open-label Phase II DAWN trial of laru-zova in the fellow eye of male subjects with XLRP who have been treated with an adeno-associated virus (AAV) vector-based gene therapy previously is also progressing.
Beacon reported six-month interim outcomes from the DAWN trial in May, showing improvements in study eyes in comparison with previously treated fellow eyes.
Beacon Therapeutics chief medical officer Dr Daniel Chung said: “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.
“We are applying our deep understanding of both ocular disease and gene therapy by using a highly efficient AAV capsid and a stabilised gene cassette that expresses the full-length RPGR protein, to support better vision outcomes.”
