Beacon Therapeutics Holdings Ltd announced positive 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with X-Linked Retinitis Pigmentosa (XLRP). The 3-month data shows that laru-zova has been well-tolerated by all participants, and no study agent-related treatment emergent adverse events were reported, including no ocular inflammatory adverse events. The data also shows promising early improvements in low luminance visual acuity, a critical measure of visual function, the company said in a press release. 

The benefit-risk profile supports ongoing clinical development for the treatment of patients with XLRP caused by RPGR mutations, the company said.

DAWN is a non-randomized, 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 purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.

Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, "We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity — a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease."

Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP, the company said.