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Beacon Therapeutics Holdings Limited, a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, announced 6 months interim safety and efficacy results from the phase 2 DAWN trial of the company’s lead programme, 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 being held May 4-8, 2025 in Salt Lake City. 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.
“Over the past five years we have built a compelling body of safety and efficacy data on laru-zova across three different clinical studies,” said Lance Baldo, CEO of Beacon Therapeutics. “We are pleased to be sharing the 6 months 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.”
XLRP is an inherited retinal disease often caused by mutations to the RPGR gene, affecting 1 in 25,000 males in the US, Europe and Australia. The disease often leads to blindness by middle age, with no available treatment options. 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.
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 two and three 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 two dose levels of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.
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. Laru-zova has Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the US Food and Drug Administration (FDA), Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Innovative Licensing and Access Pathway (ILAP) from the UK's Medicines and Healthcare products Regulatory Agency, as well as Orphan Drug Designation from the US FDA and EMA.
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