What is the drug thought to do in the body?
AP-101 is designed to target the faulty SOD1 protein which is made in people with MND who have a change in the SOD1 gene. These changes are thought to occur in around 2% of MND cases and lead to clumps of faulty SOD1 protein within neurons which can cause damage and cell death. In laboratory studies, AP-101 has been shown to slow the progression of the disease and prolong survival.
Trial Outcome
Top-line results of the phase 2a trial found that AP-101 was safe, well tolerated and showed promising signs of slowing disease progression in people with Sporadic and SOD1-MND after twelve months. Results found that those treated with the drug experienced changes in clinical outcome measures related to survival and non-invasive ventilation, compared with those on placebo. The results also showed that those on AP-101 had a stabilisation of clinical symptoms and levels of some disease biomarkers. You can read about these results in a press release.
Previous Clinical Trials
Phase 1 trial
The phase 1 study of AP-101 was an open label study, meaning that there was no placebo used and everyone who took part in the trial was given the treatment. The trial recruited people with MND and tested multiple doses of the treatment to determine whether it was safe and well tolerated by people with MND. This phase 1 trial found no safety concerns and the treatment was well tolerated at all doses tested.
Phase 2a trial
The phase 2a trial investigated the safety and tolerability of AP-101 in people with MND, as well as looking at how it works in the body. The trial recruited 73 people with MND across multiple international sites. Those in the trial received either AP-101 or a placebo (dummy drug) for up to 48 weeks. Participants were given multiple increasing doses of the treatment to further test the safety and tolerability of the treatment. It also looked at how the drug moves through and out of the body. The trial recruited in Canada, USA, Europe and Korea only. You can find out more about the trial here.
Last updated: 21/01/2026