ALS (SOD1)

AMT-162 was designed to be a novel, one-time, intrathecally administered gene therapy for ALS caused by mutations in SOD1, a rapidly progressing, rare motor neuron disease that leads to loss of everyday functions and is uniformly fatal.

In October 2024, we announced that the first patient had been dosed in the Phase I/II clinical trial of AMT-162.

In 2025, the Company voluntarily paused enrollment in the Phase I/II EPISOD1 multi-center, open-label U.S. trial of AMT-162 for the treatment of SOD1-ALS, following an Independent Data Monitoring Committee review of available preliminary safety and efficacy data, including a SAE determined to be a dose-limiting toxicity observed in one patient in the second cohort. Following review of the preliminary efficacy and safety data generated from EPISOD1, the decision was made to discontinue development of AMT-162. Safety data will continue to be collected from the five patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements.