uniQure says IDMC recommends proceeding with dose escalation in AMT-162 trial

uniQure (QURE) announced that the Independent Data Monitoring Committee for EPISOD1, uniQure’s Phase I/II clinical trial of AMT-162, an investigational gene therapy for amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 gene, has met and reviewed 28-day safety data from the first study cohort. The IDMC’s review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort. AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons causing degeneration that, over time, leads to muscle weakness, loss of function and eventually death. AMT-162 may offer a novel one-time, intrathecally administered approach to slowing or halting the progression of SOD1-ALS.