Beacon Therapeutics will seek approval for its gene therapy, laruparetigene zovaparvovec (laru-zova), in patients with X-linked retinitis pigmentosa (XLRP) after the Phase III VISTA trial met its endpoints.
The VISTA trial (NCT04850118) reported a statistically significant proportion of patients achieved a ≥15-letter improvement in Low Luminance Visual Acuity (LLVA) as compared to the untreated control group.
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This was achieved by 31% of patients in the high dose group, 24.1% of patients in the low dose group, and no participants in the untreated control group.
There were also improvements in mean macular sensitivity, with a least-squares mean difference versus the untreated control group of 1.201 decibels (dB) in the high dose group and 1.312 dB in the low dose group.
Additionally, 48.3% of participants in the high dose group, 58.6% in the low dose group and 3.7% in the untreated control group achieved a ≥10-letter improvement in LLVA after 12 months.
Dr Robert Sisk of the Cincinnati Eye Institute, professor of ophthalmology at the University of Cincinnati, and trial investigator, said: “Beacon selected endpoints that would best capture improvements that matter to patients, particularly their ability to see in low-light conditions, which is one of the most challenging aspects of living with XLRP. Achieving high statistical significance in 15-letter or greater improvement in LLVA demonstrates a clinically meaningful and consistent treatment effect in VISTA, reinforcing confidence in laru-zova’s potential to become a first-in-disease therapy.”
Laru-zova demonstrated a favourable safety and tolerability profile, with predominantly mild to moderate ocular treatment-emergent adverse events (TEAEs). Laru-zova-related TEAEs were reported in 25% of participants in the high dose group and 38% in the low dose group. Two ocular serious adverse events (SAEs) were observed in the low dose group, both of which were attributed to the surgical procedure.
Beacon said that VISTA is the first and only pivotal trial in XLRP to achieve its primary endpoint. It also builds on positive data from the UK biotech’s prior HORIZON, SKYLINE, and DAWN (NCT06275620) trials. Laru-zova is also being investigated in the LANDSCAPE trial (NCT07174726).
Based on the VISTA data, Beacon plans to engage with global regulatory authorities with the initiation of a rolling Biologics License Application (BLA) submission for laru-zova.
Laru-zova is a gene therapy designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene designed to produce the full-length protein. There are currently no approved pharmaceutical treatments or gene therapies for specifically for XLRP.
A GlobalData report predicts cases of RP will increase across the 16 major pharmaceutical markets (16MM: the US, France, Germany, Italy, Spain, the UK, Japan, Australia, Brazil, Canada, China, India, Mexico, Russia, South Africa, and South Korea), from 1.04 million in 2024 to 1.08 million by 2029.
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