Beacon Therapeutics reported positive pivotal phase II/III results for its ocular gene therapy laruparetigene zovaparvovec (laru-zova) in X-linked retinitis pigmentosa (XLRP), marking another step toward a biologics license application. The company said the Vista trial met its FDA-endorsed primary endpoint using low luminance visual acuity (LLVA) and showed improvements in a significant portion of both high- and low-dose groups versus untreated controls. The outcome supports the regulatory story for a disorder where therapeutic options remain limited, and it positions Beacon against the broader category of retinal gene therapies competing for limited ophthalmic development and commercialization capacity. The trial results also align with investors’ interest in whether the effect size can translate into label-relevant functional gains, especially for endpoints designed to reflect real-world night vision performance.
Get the Daily Brief