What does the drug do?
AP-101 is a new antibody produced by the company AL-S PHARMA AG. This antibody is designed to target faulty SOD1 protein which is made in people with MND who have a mutation in the SOD1 gene. These mutations are thought to occur in around 2% of MND cases and lead to clumps of faulty SOD1 protein within neurons which cause damage and cell death. AP-101 has been shown to slow the progression of the disease and prolong survival in mouse models of SOD1 MND.
Phase 2a Trial
AP-101 is being tested in a larger phase 2 study. This phase 2a trial is recruiting 63 people with MND across multiple sites in Canada, USA, Europe and Korea who 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.
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