UniQure (QURE) said Thursday the Independent Data Monitoring Committee for a phase 1/2 trial of AMT-162 to treat amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene recommended going ahead with enrollment in the second group after reviewing safety data from the first group in the study.
The committee's review didn't identify any "significant safety concerns," the company said, adding it plans to start enrollment of the second dose group in Q1 2025.
The trial is being conducted in the US and has three dose-escalating groups with up to four patients each getting a short course of immunosuppression before and after an intrathecal infusion of AMT-162, the company said.
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