Beacon gene therapy hits main goal in XLRP trial

Beacon Therapeutics reported on Monday that its experimental gene therapy, laru-zova, met the main goal of a late-stage trial in patients with X-linked retinitis pigmentosa (XLRP), an inherited eye disease that causes progressive vision loss.

XLRP predominantly affects boys and young men. It typically begins with night blindness and is followed by a narrowing of the peripheral field of vision. According to National Institutes of Health data, the condition affects about 4 in 100,000 males across the United States and Europe, and there are currently no approved treatments.

The 12-month trial enrolled 85 male participants aged 12 to 48. A significant proportion of those who received the therapy improved their ability to read at least 15 additional letters in low-light conditions, while no participants in the untreated control group achieved that level of improvement.

"We are now the only company with a study that is read out positively on its primary endpoint within a pivotal phase three study, and so that's giving us a chronologic advantage," said CEO Lance Baldo.

The privately held company said it will hold discussions with global regulatory authorities regarding a marketing application submission based on the trial results.