Phase 2 DAWN for XLRP data presented at ARVO 2025

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Beacon Therapeutics has released 6-month interim safety and efficacy results from the Phase 2 DAWN (NCT06275620) trial for the pharmaceutical candidate laru-zova (laruparetigene zovaparvovec). This candidate is being evaluated for treatment of patients with X-linked retinitis pigmentosa (XLRP). According to the data, 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.¹

Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics spoke to these interim results in a quote to Ophthalmology Times and Modern Retina, he said, “The 6-month interim results from the Phase 2 DAWN trial further highlight laru-zova's potential to improve vision in patients with XLRP with the findings to-date demonstrating advancements in multiple visual function measures. We are committed to advancing laru-zova to offer new hope for those affected by XLRP who are currently without treatment options, and we eagerly await further data from the trial.”

DAWN is a phase 2, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess 2 different dose levels of laru-zova for efficacy, safety and tolerability in the target population.¹

The interim study results were presented at the 2025 ARVO meeting held in Salt Lake City, Utah. Highlights from this presentation include:

• Ocular treatment-emergent adverse events (TEAEs) were generally non-serious and mild or moderate in severity, with a majority related to surgical procedures and steroids required by the protocol that have since resolved.
• There were no suspected unexpected serious adverse reactions, retinal detachments or endophthalmitis reported.
• Data demonstrated early improvements in low luminance visual acuity (LLVA), an important measure of visual function, with a greater number of 2 and 3 line improvements in the study eyes compared to previously treated fellow eyes in participants evaluated at month 6 or beyond.
• Data also showed early and sustained improvements in mean sensitivity in study eyes, as observed by microperimetry, indicating enhanced visual function in participants evaluated at month 6 or beyond.¹

Beacon Therapeutics continues to enroll patients for its pivotal Phase 2/3 VISTA trial of laru-zova for patients with XLRP. VISTA (NCT04850118) is a Phase 2/3, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. The study will evaluate the proportion of participants with a 15 or more letter increase from baseline in LLVA and additional measures of functional vision.¹

About laru-zova

Laru-zova (laruparetigene zovaparvovec) is a potential best-in-class gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Laru-zova has the potential 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.¹

Reference:

1. Beacon Therapeutics Announces Positive Phase 2 Interim 6-Month Data from DAWN Trial of Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at ARVO 2025. Beacon Therapeutics. May 6, 2025. Accessed May 7, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-announces-positive-phase-2-interim-6-month-data-from-dawn-trial-of-laru-zova-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-at-arvo-2025/