Beacon Therapeutics's Laru-Zova Hits Primary Endpoint in Pivotal XLRP Trial

Beacon Therapeutics announced positive topline results from the pivotal phase 2/3 VISTA trial of laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP). The results meet the company’s FDA-endorsed primary endpoint with statistical significance, Beacon announced September 21, 2026.¹ According to the company, VISTA is the first and only pivotal trial in XLRP to achieve its primary endpoint, and laru-zova demonstrated a favorable safety and tolerability profile consistent with prior studies.¹

"These results represent a landmark moment for the hundreds of thousands of patients worldwide living with XLRP who currently have no treatment options and no way to slow the loss of their sight," said Lance Baldo, MD, CEO of Beacon Therapeutics, in a company press release.¹ "Today's data are both statistically significant and clinically meaningful, representing an important milestone for ocular gene therapy and demonstrating the potential for a one-time treatment to change the course of an inherited retinal disease."

What did the VISTA trial show?

VISTA (NCT04850118) is a randomized, controlled trial that evaluated 85 male patients ages 12 to 48 with XLRP caused by mutations in the RPGR gene, comparing high-dose (6.8E+11 vg/eye) and low-dose (3.7E+11 vg/eye) laru-zova against an untreated control group over 12 months.¹^,² At month 12, a statistically significant proportion of patients achieved a 15-letter or greater improvement in low luminance visual acuity (LLVA) compared with untreated controls: 31.0% in the high-dose group (p=0.0019) and 24.1% in the low-dose group (p=0.0106), versus no responders in the control group.¹ A larger proportion of patients achieved a 10-letter or greater improvement, reported in 48.3% of the high-dose group, 58.6% of the low-dose group, and 3.7% of controls.¹ Secondary measures of macular sensitivity by microperimetry also showed supportive positive trends favoring treatment.¹

What are the safety findings?

Laru-zova showed a favorable safety and tolerability profile, with predominantly mild to moderate ocular adverse events balanced across treatment groups and largely attributed to the surgical procedure. Treatment-related adverse events occurred in 25% of the high-dose group and 38% of the low-dose group.¹ Two ocular serious adverse events occurred in the low-dose group, both attributed to the surgical procedure.¹ These results build on 5 years of clinical experience with laru-zova across 110 treated participants, including the completed phase 1/2 HORIZON trial, in which 29 patients received a single subretinal dose with adverse events generally mild and related to the injection procedure.¹^,³

What did outside experts say about the results?

"Beacon selected endpoints that would best capture improvements that matter to patients, particularly their ability to see in low-light conditions," said Robert Sisk, MD, FACS, FASRS, at the Cincinnati Eye Institute and professor of ophthalmology at the University of Cincinnati, in the release.¹ In addition, Jason Menzo, CEO of the Foundation Fighting Blindness, called the results "the news we have been waiting years to hear" for a relentlessly progressive disease with no approved treatments.¹

Why is XLRP significant, and what's next for laru-zova?

XLRP is caused by mutations in the RPGR gene and affects approximately 1 in 25,000 males in the United States, Europe, and Australia. It leads to progressive photoreceptor loss beginning in childhood, eventually causing blindness, with no approved treatments.¹ Based on the VISTA results, Beacon plans to begin pre-submission discussions with global regulatory authorities and initiate a rolling biologics license application (BLA) submission later this year.¹

Laru-zova holds regenerative medicine advanced therapy (RMAT) and fast track designations from FDA, priority medicines (PRIME) designation from the European Medicines Agency (EMA), innovative licensing and access pathway designation from the United Kingdom's Medicines and Healthcare products Regulatory Agency, and orphan drug designation (ODD) from FDA and EMA.¹

What are the limitations?

These are topline results, and detailed statistical data have not yet been published in a peer-reviewed journal. Additional data are expected at the American Academy of Ophthalmology's Retina Subspecialty Day in October 2026. VISTA is an open-label trial with an untreated rather than sham-treated control group, and its primary endpoint improvements, while statistically significant, are based on a relatively small patient population of 85 participants.

References

1. Beacon Therapeutics. Beacon Therapeutics reports positive topline data from the pivotal VISTA trial of laru-zova for the treatment of X-linked retinitis pigmentosa (XLRP). Press release. Published September 21, 2026. Accessed September 21, 2026. https://www.beacontx.com/news-and-events/beacon-therapeutics-reports-positive-topline-data-from-the-pivotal-vista-trial-of-laru-zova-for-the-treatment-of-x-linked-retinitis-pigmentosa-xlrp/
2. A clinical trial evaluating the safety and efficacy of a single subretinal injection of AGTC-501 in participants with XLRP. ClinicalTrials.gov; NCT04850118. Updated April 7, 2026. Accessed September 21, 2026. https://clinicaltrials.gov/study/NCT04850118
3. Yang P, Birch D, Lauer A, et al. Subretinal gene therapy drug AGTC-501 for XLRP phase 1/2 multicenter study (HORIZON): 24-month safety and efficacy results. Am J Ophthalmol. 2025;271:268-285. doi:10.1016/j.ajo.2024.11.021