News

Article

Beacon Therapeutics shares 6-month interim safety and efficacy results from Phase 2 DAWN study for XLRP

Author(s):

Key Takeaways

  • Laru-zova showed promising safety and efficacy in the Phase 2 DAWN trial for XLRP, with mild or moderate adverse events.
  • Improvements in low luminance visual acuity and mean sensitivity were observed, indicating enhanced visual function.
SHOW MORE

Beacon Therapeutics reveals promising interim results for laru-zova, a gene therapy showing potential to improve vision in XLRP patients.

Image credit: AdobeStock/vivekFx

(Image credit: AdobeStock/vivekFx)

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/

Newsletter

Don’t miss out—get Ophthalmology Times updates on the latest clinical advancements and expert interviews, straight to your inbox.

Related Videos
(Image credit: Ophthalmology Times) ASCRS 2025: Mark Lobanoff, MD, on making the move to office-based surgery
Barsha Lal, PhD, discusses the way low dose atropine affects accommodative amplitude and dynamics at the 2025 ARVO meeting
At the Association for Research in Vision and Ophthalmology (ARVO) meeting, Katherine Talcott, MD, a retina specialist at Cleveland Clinic, shared her findings on EYP-1901 (EyePoint Pharmaceuticals) in the phase 2 DAVIO study.
Dr. Jogin Desai, founder of Eyestem Research, discusses his research at the Association for Research in Vision and Ophthalmology.
(Image credit: Ophthalmology Times) ASCRS 2025: Michael Rivers, MD, shares his takeaways as a panelist at the inaugural SightLine event
(Image credit: Ophthalmology Times) ASCRS 2025: Karl Stonecipher, MD, on LASIK outcomes using an aspheric excimer laser for high myopia
John Tan talks about an emergency triage framework for retinal artery occlusion at the 2025 Association for Research in Vision and Ophthalmology (ARVO) meeting.
Dr Robert Maloney at the 2025 Controversies in Modern Eye Care meeting
Wendy Lee, MD, MS, at Controversies in Modern Eye Care 2025.
© 2025 MJH Life Sciences

All rights reserved.