Beacon Therapeutics Holdings Limited announced that it has successfully completed enrollment in its pivotal phase 2/3 VISTA trial evaluating its gene therapy laruparetigene zovaparvovec (laru-zova) for the treatment of X-linked retinitis pigmentosa (XLRP). According to a company press release, 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. Twelve-month topline data from this trial are expected in the second half of 2026.

VISTA 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. Laru-zova is designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene using a well-established vector with a proprietary capsid designed for high transduction of photoreceptors, and a codon-optimized gene to produce the full-length protein.

The trial is assessing 2 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 said it 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. In May, the company announced 6-month interim results from DAWN, demonstrating greater 2-line and 3-line improvements in study eyes compared to previously treated fellow eyes, and a safety profile consistent with previous studies. RP