Clinical Trials

The LANDSCAPE clinical trial is an open-label study evaluating the safety of laru-zova administered bilaterally. The study is enrolling males between the ages of 12 and 50 with a confirmed diagnosis of X-Linked Retinitis Pigmentosa (XLRP). The LANDSCAPE study will also evaluate changes in visual function after administering bilaterally via subretinal injection.

The pivotal VISTA clinical trial is an ongoing, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of laru-zova compared to an untreated control group. VISTA completed enrollment with 85 male participants with confirmed diagnosis of XLRP, between the ages of 12-50 years (inclusive). Participants in both treatment groups received one of two dose levels of laru-zova in one eye.

Beacon will use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON, Phase 2 SKYLINE and Phase 2 DAWN studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP.

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

SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled trial evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. The trial’s primary endpoint is the proportion of response by microperimetry between the study and fellow eye at month 12.

HORIZON is a completed 5-year Phase 1/2, open-label, dose escalation trial of laru-zova in patients with XLRP due to mutations in the RPGR gene. The trial included 29 male participants, who received a single sub-retinal dose of laru-zova in one eye to obtain initial data about safety and potential efficacy of laru-zova gene therapy.