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.
Phase 2a
Current status: Collecting data
Primary objective: To investigate the safety of AP-101
The phase 2a trial is investigating 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 63 people with MND across multiple international sites. Those in the trial will receive either AP-101 or a placebo (dummy drug) for up to 48 weeks. Participants will be given multiple increasing doses of the treatment, and the trial will further test the safety and tolerability of the treatment, as well as looking at how the drug moves through and out of the body. You can find out more about the trial here.
The trial is recruiting in Canada, USA, Europe and Korea only.
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.
Last updated: 18/04/2024