Beacon Therapeutics has announced positive 3-month interim data from its Phase 2 DAWN trial of laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP). The data, presented at the FLORetina ICOOR 2024 meeting, show early promise in improving low luminance visual acuity (LLVA), a key measure of visual function in patients with this debilitating disease.
The DAWN trial is a non-randomized, open-label study investigating two doses of laru-zova in participants with XLRP who had previously received full-length AAV-vector-based gene therapy targeting the RPGR protein. The trial is designed to assess the efficacy, safety, and tolerability of laru-zova in the untreated eye of these patients.
Key Findings from the DAWN Trial
The interim data highlighted that laru-zova was well-tolerated by all participants, with no treatment-emergent adverse events (TEAEs) related to the study agent, including no ocular inflammatory events. Furthermore, early data demonstrated improvements in LLVA, a functional visual outcome that is particularly relevant for patients with XLRP.
"The strong safety profile of laru-zova, coupled with the early signs of improvement in low luminance visual acuity, underscores the potential of this therapy to significantly impact patients with XLRP," said Lance Baldo, MD, CEO of Beacon Therapeutics, in a statement. "These data not only support the ongoing VISTA trial but also reflect our commitment to bringing hope to the patients and families affected by this devastating disease."
About X-Linked Retinitis Pigmentosa (XLRP)
XLRP is a severe, progressive inherited retinal disease that leads to blindness, typically by middle age, and currently has no approved treatments. It primarily affects young males, with an estimated prevalence of 1 in 25,000 males in the US, Europe, and Australia. XLRP is most commonly caused by mutations in the RPGR gene.
Laru-zova (AGTC-501): A Potential Treatment for XLRP
Laru-zova is a gene therapy designed to express the full-length RPGR protein, which may address both rod and cone photoreceptor loss caused by the disease. The therapy aims to halt or slow disease progression, potentially representing a best-in-class treatment for XLRP. Beacon Therapeutics is currently enrolling patients in its pivotal Phase 2/3 VISTA trial, a multi-center study designed to further evaluate the efficacy and safety of laru-zova in XLRP.
Professor Paulo Eduardo Stanga, MD, founder and chief medical officer of The Retina Clinic London, presented the data at FLORetina-ICOOR 2024. The presentations included "Subretinal Gene Therapy Laru-Zova (AGTC-501) for XLRP: Phase 2 DAWN Trial Preliminary Results" and "RPGR Gene Therapy and Beacon's Clinical Trials: Beacon Therapeutics' Subretinal Gene Therapy Laru-Zova (AGTC-501) for XLRP."
