Beacon Therapeutics Holdings Limited announced 6-month interim safety and efficacy results from the phase 2 DAWN trial of the company’s lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP) at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting.
Laru-zova is a potential best-in-class gene therapy designed to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene using a well-established vector with a proprietary capsid designed for high transduction of photoreceptors, and a codon-optimized gene to produce the full-length protein.
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, the company said in a press release.
Key data highlights 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.
DAWN is an open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting the RPGR protein. The study aims to assess 2 dose levels of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.
The company said it continues to enroll patients for its pivotal phase 2/3 VISTA trial of laru-zova for patients with XLRP.
“Over the past 5 years we have built a compelling body of safety and efficacy data on laru-zova across 3 different clinical studies,” said Lance Baldo, MD, chief executive officer of Beacon Therapeutics, in the press release. “We are pleased to be sharing the 6-month data update from the DAWN phase 2 study that continues to demonstrate laru-zova's potential to enhance vision in patients with XLRP, including improvements in multiple measures of visual function. We look forward to continuing the advancement of this exciting novel treatment option for patients suffering from XLRP.”