Beacon Therapeutics, a clinical-stage biotechnology company focused on ocular diseases, on Monday reported positive topline results for its registration-enabling VISTA trial evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP).

VISTA is a randomised, 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 subjects 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. The VISTA trial met its FDA-endorsed primary endpoint with statistical significance. Additionally, laru-zova demonstrated a favourable safety and tolerability profile, consistent with prior studies.

Based on these positive topline results, Beacon plans to engage with global regulatory authorities with initiation of a rolling Biologics License Application (BLA) submission planned for later this year.