Beacon Therapeutics reports positive phase 2 results for gene therapy in X-linked retinitis pigmentosa

Beacon Therapeutics recently revealed topline data results for 2 of the company’s phase 2 trials—SKYLINE and DAWN, both investigating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP)^.1

The data was presented during the 2025 European Society of Retina Specialists (EURETINA) annual meeting held September 4-7 in Paris.

In both trials, Beacon’s lead program, laru-zova, was found to be well-tolerated by SKYLINE participants through month 36 and DAWN patients at month 9 or longer.¹ Sustained improvements across several key measures of visual function—including low luminance visual acuity and microperimetry—were seen.

With no currently approved or available treatment options, XLRP is an inherited retinal disease, often leading to blindness. Affecting 1 in 25,000 males in the United States, Europe, and Australia, XLRP is typically caused by mutations to the retinitis pigmentosa GTPase regulator (RPGR) gene.¹

Laru-zova is a gene therapy currently being investigated for the treatment of patients with XLRP, with the potential to restore natural function of both rods and cones in XLRP by delivering a functional copy of the RPGR ORF15 gene, designed to produce the full-length protein.¹

The objective of the DAWN study (NCT06275620) is to assess 2 different dose levels of laru-zova for efficacy, safety, and tolerability in the target population.¹ It is an ongoing, fully enrolled, phase 2, open-label study of laru-zova in the fellow eye of male participants with XLRP previously treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. Additionally, DAWN is evaluating the changes in visual function and functional vision. It is the first trial in the laru-zova clinical development program collecting and evaluating low luminance visual acuity (LLVA) data.¹

Key DAWN data highlights:

• Data continued to show early improvements in LLVA and early and sustained improvements in mean sensitivity in study eyes, as observed by microperimetry, representing enhanced visual function in participants evaluated at month 9 or beyond.¹
• Laru-zova continued to be well-tolerated by all participants evaluated at month 9 or beyond.¹

The primary endpoint of SKYLINE (NCT06333249) is the proportion of response by microperimetry between the study and fellow eye at month 12. It is an ongoing, fully enrolled, phase 2, randomized, controlled study evaluating the safety, efficacy, and tolerability of laru-zova in 14 male patients with XLRP.¹

Key SKYLINE data highlights:

• Participants who received the high dose of laru-zova showed durable improvements in retinal sensitivity through month 36, as observed by microperimetry.¹
• There was a greater response rate in the high-dose study eyes compared to the low-dose group or untreated fellow eye.¹
• Laru-zova continued to be well-tolerated by participants in both low- and high-dose groups through month 36.¹

“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,” Daniel Chung, DO, MA, Chief Medical Officer of Beacon Therapeutics, said in a press release.¹

“These new data updates reinforce our belief in the potential for laru-zova through clinical development while engaging with regulators and the patient community.”

Laru-zova currently has regenerative medicine advancement 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 (MHRA), as well as orphan drug designation (ODD) from both the FDA and the EMA.¹

Reference:

1. Beacon Therapeutics Announces Positive Interim 9+ Month Results from DAWN Trial and 36-Month Phase 2 SKYLINE Trial Data for Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at EURETINA 2025 - Beacon Therapeutics. Beacon Therapeutics. Published September 4, 2025. Accessed September 8, 2025. https://www.beacontx.com/news-and-events/beacon-therapeutics-announces-positive-interim-9-month-results-fromdawn-trial-and-36-month-phase-2-skyline-trial-data-for-laru-zova-in-patients/