NEW YORK – UniQure on Thursday said it will begin treating a second cohort of patients with a genetic form of amyotrophic lateral sclerosis (ALS) with a higher dose of its gene therapy in the Phase I/II EPISOD1 trial.
UniQure is developing the gene therapy, AMT-162, as a treatment for ALS caused by mutations in the SOD1 gene. SOD1-ALS is the most common genetic cause of ALS, though only about 2 percent of ALS cases are related to mutations in the gene.
The Amsterdam-based company's decision to advance the clinical trial follows an independent data monitoring committee's review of 28-day safety data from the first cohort of patients treated with AMT-162 within the study. The committee did not identify any significant safety concerns and recommended UniQure proceed with dose escalation and enrollment of a second cohort.
UniQure expects to begin enrolling patients into the second cohort in the first quarter of 2025.
AMT-162 is designed to slow progression of SOD1-ALS using an adeno-associated virus rh10 vector that expresses a microRNA that knocks down expression of the mutated SOD1 protein.
Within the open-label EPISOD1 trial, patients with SOD1-ALS will receive a single dose of AMT-162, administered intrathecally, at one of three doses. Investigators will evaluate the safety and tolerability of the gene therapy and assess exploratory biomarkers including neurofilament light chain and SOD1 protein.