Beacon Therapeutics reported positive topline results from the pivotal Phase 2/3 VISTA trial of laru-zova (laruparetigene zovaparvovec) for X-linked retinitis pigmentosa (XLRP), an inherited disease that can cause progressive vision loss and blindness.
The trial met its FDA-endorsed primary endpoint:
after 12 months, 31.0% of patients receiving the high dose and 24.1% receiving the low dose improved by at least 15 letters on a low-light visual-acuity test, compared with no responders in the untreated control group.
Beacon said the therapy showed a favorable safety and tolerability profile, with reported adverse events predominantly mild to moderate.
The company plans to begin pre-submission discussions with regulators and start a rolling U.S. Biologics License Application later this year. The therapy remains investigational and is not yet FDA-approved.
If approved, laru-zova could become the first treatment for XLRP, which currently has no approved therapies.
Sources:
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