Beacon says gene therapy succeeds in key trial of rare eye disease XLRP

Syncona's shares surged around 6% on Monday following news from its sister company, Beacon Therapeutics. Beacon announced that a late-stage gene therapy trial for a rare inherited eye condition met its primary objective. The VISTA trial, involving 85 male patients with X-linked retinitis pigmentosa (XLRP) due to RPGR gene mutations, demonstrated promising results.

Over a 12-month period, 31% of those receiving the high dose of the therapy and 24.1% of those on the low dose showed improvements of at least 15 letters in visual acuity, compared to none in the control group. Secondary measures indicated that 48.3% of high-dose patients and 58.6% of low-dose patients experienced improvements of at least 10 letters, while control group patients saw only a 3.7% improvement.

Mean macular sensitivity also showed significant improvement in both dose groups, with p-values indicating statistical significance. Most adverse events were mild to moderate and evenly distributed between groups, mainly attributable to the surgical procedure. Treatment-related events occurred in 25% of high-dose patients and 38% of low-dose patients, with two serious ocular issues reported in the low-dose group, both linked to surgery.

Beacon intends to begin discussions with global regulators and commence a rolling Biologics License Application process later this year. However, laru-zova, the gene therapy in question, remains unapproved by the U.S. Food and Drug Administration.