Beacon claimed the findings represent the first time a treatment for X-linked retinitis pigmentosa, a rare and potentially blinding condition, met its main goal in a pivotal trial.
Syncona's shares surged by approximately 6% on Monday following Beacon Therapeutics' announcement that its gene therapy for a rare inherited eye disease, X-linked retinitis pigmentosa (XLRP), met its primary endpoint in a late-stage trial. The VISTA trial, which tested laruparetigene zovaparvovec (laru-zova), involved 85 male patients aged 12 to 48 with XLRP caused by RPGR gene mutations.
At 12 months, the trial showed that 31% of patients receiving the high dose and 24.1% of those receiving the low dose improved by at least 15 letters in low-luminance visual acuity, compared to none in the untreated control group. Beacon plans to initiate discussions with global regulators and commence a rolling Biologics License Application later this year.
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