Beacon Therapeutics completes enrollment in phase 2/3 VISTA trial

(Image Credit: AdobeStock/Ralf Geithe)

Beacon Therapeutics has completed enrollment in its registrational phase 2/3 VISTA trial evaluating laruparetigene zovaparvovec (laru-zova) for the treatment of X-linked retinitis pigmentosa (XLRP).¹

VISTA (NCT04850118) is a randomized, controlled, masked, multicenter study evaluating and comparing 2 dose levels of laru-zova with an untreated control group.² A single subretinal injection of laru-zova dose 1 or dose 2 will be administered to patients in 2 treatment groups, while patients in the untreated control group will be followed and evaluated, after which they will be evaluated to determine if they are eligible to receive treatment with dose 2 of laru-zova. Patients will be evaluated by the improvement in low-luminance visual acuity and mean sensitivity as observed by microperimetry, among other measures of visual function.

The trial enrolled approximately 75 male patients aged between 12 and 50 years with XLRP caused by RPGR mutations across sites in North America, the United Kingdom, and Australia. Those enrolled will be randomly assigned in a ratio of 1:1:1.

The company noted that it plans to use the data from VISTA, alongside long-term data from the ongoing phase 2 DAWN, phase 1/2 HORIZON, and phase 2 SKYLINE studies, to support regulatory submissions in the United States and Europe.

Lance Baldo, MD, CEO of Beacon Therapeutics, and Daniel Chung, DO, MA, chief medical officer of Beacon Therapeutics, commented on the trial in a press release from the company.

“Completion of enrollment in our VISTA trial marks a significant milestone for Beacon and, more importantly, for patients in the XLRP community,” said Baldo.

“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,” added Chung. “We are applying our deep understanding of both ocular disease and gene therapy by using a highly efficient AAV [adeno-associated virus] capsid and a stabilized gene cassette that expresses the full-length RPGR protein to support better vision outcomes.”

Beacon noted that its phase 2 DAWN trial continues to progress, and the company released 6-month interim safety and efficacy results in May 2025.³ According to the company, laru-zova was generally well tolerated by all DAWN participants evaluated at 6 months or beyond, and initial data showed promising improvements in visual function across several key measures.

Twelve-month topline data from the VISTA trial are expected in the second half of 2026.

References

1. Beacon Therapeutics completes enrollment in registrational phase 2/3 VISTA trial of laru-zova for patients with XLRP. News release. Beacon Therapeutics Holdings Limited. July 8, 2025. Accessed July 8, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-completes-enrollment-in-registrational-phase-2-3-vista-trial-of-laru-zova-for-patients-with-xlrp/

2. A clinical trial evaluating the safety and efficacy of a single subretinal injection of AGTC-501 in participants with XLRP. ClinicalTrials.gov. Updated May 18, 2025. Accessed July 8, 2025. https://clinicaltrials.gov/study/NCT04850118

3. Crago SM. Beacon Therapeutics shares 6-month interim safety and efficacy results from phase 2 DAWN study for XLRP. Ophthalmology Times. May 8, 2025. Accessed July 8, 2025. https://www.ophthalmologytimes.com/view/beacon-therapeutics-shares-6-month-interim-safety-and-efficacy-results-from-phase-2-dawn-study-for-xlrp