Enrollment complete for Beacon Therapeutics' phase 2/3 VISTA trial

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

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

The trial enrolled approximately 75 male patients aged 12 to 50 years with X-linked retinitis pigmentosa caused by RPGR mutations, across sites in North America, the United Kingdom, and Australia. Those enrolled will be randomized 1:1:1.

The company noted that it plans to use the data from the VISTA trial, alongside long-term data from the ongoing Phase 2 DAWN trial and the 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, CMO 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 capsid and a stabilized gene cassette that expresses the full-length RPGR protein to support better vision outcomes.”

The company noted it continues to progress its phase 2 DAWN trial, of which 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. Published 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. NCT04850118. https://clinicaltrials.gov/study/NCT04850118

3. Crago S. Beacon Therapeutics shares 6-month interim safety and efficacy results from phase 2 DAWN study for XLRP. Published 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