Beacon Reports Phase 2 XLRP Gene Therapy Results

Beacon Therapeutics has announced topline results from two Phase 2 clinical trials, SKYLINE and DAWN, evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP). The data were presented at the 2025 EURETINA Congress, held September 4–7 in Paris.

Background on XLRP and Laru-Zova

X-linked retinitis pigmentosa (XLRP) is a rare, inherited retinal disease that affects approximately 1 in 25,000 males in the U.S., Europe, and Australia. It is commonly caused by mutations in the RPGR (retinitis pigmentosa GTPase regulator) gene and often results in progressive vision loss and eventual blindness. Currently, no approved treatments exist for XLRP.

Laru-zova is an investigational gene therapy designed to deliver a functional copy of the RPGR ORF15 gene, which produces the full-length RPGR protein. This approach is intended to restore natural function of both rods and cones in patients with XLRP.

DAWN Trial Overview and Results

The DAWN study (NCT06275620) is a fully enrolled, Phase 2, open-label trial evaluating two different dose levels of laru-zova in the fellow eye of male participants with XLRP who were previously treated with an AAV-based gene therapy. DAWN is the first trial in the laru-zova clinical program to collect and evaluate low luminance visual acuity (LLVA) data.

Key Findings from DAWN:

• Early improvements in LLVA and sustained gains in mean sensitivity (assessed via microperimetry) were observed in study eyes at month 9 or beyond, indicating enhanced visual function.

• Laru-zova was well-tolerated by all participants evaluated at month 9 or longer.

The trial also evaluates broader endpoints related to visual function and functional vision, including safety and tolerability.

SKYLINE Trial Overview and Results

The SKYLINE study (NCT06333249) is a randomized, controlled, fully enrolled Phase 2 trial evaluating the safety, efficacy, and tolerability of laru-zova in 14 male patients with XLRP. The primary endpoint is the proportion of response by microperimetry between the study and fellow eye at month 12.

Key Findings from SKYLINE:

• Participants who received the high dose of laru-zova demonstrated durable improvements in retinal sensitivity through month 36, as measured by microperimetry.

• The high-dose group showed a greater response rate compared to the low-dose group or untreated fellow eye.

• Laru-zova remained well-tolerated in both dose groups through the 36-month evaluation period.

Regulatory Status and Company Commentary

Laru-zova has received multiple regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT) and Fast Track status from the U.S. Food and Drug Administration (FDA), PRIME designation from the European Medicines Agency (EMA), Innovative Licensing and Access Pathway (ILAP) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and Orphan Drug Designation (ODD) from both the FDA and EMA.

Dr. Daniel Chung, Chief Medical Officer of Beacon Therapeutics, stated:

“We are pleased to be sharing key data from our DAWN and SKYLINE trials, building on one of the most significant bodies of evidence for a gene therapy in ocular disease. These new data updates reinforce our belief in the potential for laru-zova through clinical development while engaging with regulators and the patient community.”