05/05/2026

UniQure provides an update on the status of the AMT-162 investigational gene therapy program for SOD1-ALS.

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 severe adverse event (SAE) determined to be a dose-limiting toxicity observed in one patient in the second cohort.

Following a comprehensive review of the preliminary efficacy and safety data generated from EPISOD1, the difficult decision was made to discontinue development of AMT-162.   Long term follow-up with safety data collection will continue for the five patients dosed in EPISOD1, consistent with applicable safety and regulatory requirements.

This decision was not made lightly, and UniQure values the ALS community’s engagement that has been meaningful to everyone at uniQure involved in this program.

Source: UniQure press release and personal communication