XLRP Gene Therapy Trial Underway

There has been “strong interest” in a Phase 2/3 trial evaluating laru-zova for the treatment of X-linked retinitis pigmentosa (XLRP), according to Beacon Therapeutics.

The clinical-stage biotechnical company has announced that it has successfully completed enrolment of eligible male patients aged 12 to 50 across sites in North America, the United Kingdom, and Australia, exceeding the initial enrolment projection.

The VISTA trial (NCT04850118) is a global, randomised, controlled, masked, multi-centre Phase 2/3 study designed to evaluate the efficacy, safety, and tolerability of laru-zova in male patients with XLRP caused by variations in the RPGR gene.

The trial is assessing two dose levels of laruzova compared to an untreated control group, evaluating the proportion of participants with improvement in low-luminance visual acuity and mean sensitivity as observed by microperimetry, among other measures of visual function.

“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,” said Dr Daniel Chung, Chief Medical Officer of Beacon Therapeutics.

“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. We are cautiously optimistic about the potential for laru-zova to treat a condition that has long been considered untreatable.”

X-linked retinitis pigmentosa is an inherited retinal disease that predominantly affects males. The disease, which affects approximately one in 25,000 males in the US, Europe, and Australia, results in progressive photoreceptor loss over time, and visual dysfunction beginning in childhood, eventually leading to blindness.