What does the drug do?

ION363 (also known as Jacifusen) is an investigational antisense medicine that is designed to reduce the production of the Fused in Sarcoma (FUS) protein which can lead to rapid, progressive loss of motor neurons in some patients. Patients with a mutation in the FUS gene develop a rare form of ALS, referred to as FUS-ALS.

Phase 3

Current status: Open for recruitment

The Phase 3 trial to evaluate the safety and efficiency of ION363 is split into two parts. The first part of the trial will consist of patients randomised to receive a multi-dose regime of ION363 or placebo for 61 weeks. The second part is an open-label period where all patients in the trial will receive ION363 for 85 weeks. The primary outcome of the trial is change from day 1 to day 225 in rate of decline measured by the ALS functional rating scale (ALSFRS-R). Find out more on the MND Clinical Studies Group website or on clinicaltrials.gov.

How can I take part in the trial?

Recruiting Sites

The trial is recruiting in the UK at the following sites:

Please contact the individual trial site, by clicking each site above, for more information on how to take part in the trial. 

Trial Information

Phase: 1-3

Placebo controlled: Yes - this means that people joining the trial could be randomly assigned to receive the placebo (dummy drug). 

Open-Label Extension: Yes - this means that all participants on the trial, regardless of whether they received the treatment or placebo, will have an opportunity to access the treatment once the randomised part of the trial is complete. 

Key eligibility criteria: This trial is only recruiting people with MND who have a FUS gene mutation. More criteria here.

Latest News


May 2022 - The protocol of the trial was changed to increase the length of the double-blind phase (from 29 weeks to 61 weeks). This is to increase the likelihood of detecting a clinically meaningful signal.


Dec 2021 - King's College London was the first trial centre in Europe to recruit participants for ION363

Last updated: 11/03/2024