Beacon's XLRP gene therapy set for filing after phase 3 win

Beacon Therapeutics is preparing to file for approval of its gene therapy for eye disorder X-linked retinitis pigmentosa (XLRP) after reporting positive results in a pivotal trial.

In the phase 2/3 VISTA trial, laruparetigene zovaparvovec (laru-zova; formerly AGTC-501) achieved its main objective with statistical significance in the low luminance visual acuity (LLVA) responder rate – the primary endpoint – at 12 months compared to an untreated control group.

Data showed that a significant proportion of participants treated with laru-zova achieved an improvement of 15-letters or more on an eye chart, compared to no responders in the untreated control group. That threshold was reached by 31% of XLRP patients treated with a high dose of the gene therapy, and 24.1% of those receiving a low dose.

Beacon said the gene therapy also demonstrated a favourable safety and tolerability profile that was consistent with prior studies.

Based on the results, the UK- and US-based company said it plans to start filing a rolling Biologics License Application (BLA) submission with the FDA later this year. If approved, laru-zova would be the first therapy for XLRP, and the first ocular gene therapy since the approval of Roche/Genentech's Luxturna in 2017 for inherited retinal disease caused by mutations in the RPE65 gene.

Laru-zova is vying to become the first-ever treatment for XLRP, an inherited condition affecting one in 40,000 people globally that causes boys to develop night blindness by the age of 10, and generally leads to profound loss of sight by the time they reach their early forties.

"Today’s data [is] both statistically significant and clinically meaningful," said chief executive Lance Baldo, who added that the results represent "an important milestone for ocular gene therapy and [demonstrate] the potential for a one-time treatment to change the course of an inherited retinal disease."

The gene therapy was acquired from US biotech AGTC in 2022 by Syncona, which set up and provided financing to Beacon in its early days after acquiring laru-zova as part of its takeover of Applied Genetic Technologies Corporation (AGTC). Beacon’s pipeline also features programmes for other eye diseases, including dry age-related macular degeneration (AMD) and cone-rod dystrophy (CRD).

"We are incredibly proud of the role Syncona has played in supporting Beacon on this journey," said the investment group's CEO, Chris Hollowood. "Seeing laru-zova generate positive pivotal data is a powerful validation of the potential we saw in this asset and of the work of everyone who has helped bring it to this point."