Beacon Therapeutics reports positive pivotal trial results for X-linked retinitis pigmentosa gene therapy
Beacon Therapeutics said its gene therapy for X-linked retinitis pigmentosa (XLRP) met the primary endpoint in a phase 2/3 Vista study. The trial enrolled 85 male patients and compared two dose levels of laruparetigene zovaparvovec with a control group.
At twelve months, 24.1% of the low-dose group and 31% of the high-dose group achieved the predefined visual acuity improvement, while none of the control patients met the responder criteria. The endpoint required reading at least 15 additional letters on an eye chart under low-light conditions.
CEO Lance Baldo, M.D., said the data demonstrate a potential treatment effect for a disease that affects hundreds of thousands worldwide and that the company will submit the results to regulators in the coming months.
This writeup was produced by pharmadog from original reporting by Fierce Biotech.
Original headline: “Beacon lights path to FDA approval with pivotal trial win for rare vision loss gene therapy”
read at Fierce Biotech ↗
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