What does the drug do?
Tegoprubart is an antibody against a protein called CD40 ligand (CD40L) which is present on the surface of some immune cells. Antibodies are proteins that bind to and block a particular target. CD40L plays a role in regulating the immune response and that can trigger inflammation in the spinal cord. Research has shown that the CD40L pathway is overactive in people with MND. By inhibiting CD40L, AT-1501 could block or delay the activation of the damaging inflammatory immune response which could delay the onset or slow the progression of MND and increase survival.
The Phase 1 trial enrolled 8 participants and its primary outcome was safety and tolerability. The trial showed that all participants tolerated all dose levels. The pharmacokinetics (how it is processed in the body) of the drug was found to be as the researchers predicted. You can read more about the results of the trial here.
The Phase 2a trial is recruiting 54 MND patients to take part in the open-label, multidose study in the US. Participants will receive one of three increasing intravenous doses of AT-1501 once every two weeks for 11 weeks. Investigators will continue to monitor them for safety and tolerability for another eight weeks (19 weeks in total). You can find out more about the trial here.
This trial was recruiting in the US and Canada only.
May 2022 - Topline results from the trial were announced showing that it hit the primary endpoints of safety and tolerability. You can read more here.
Feb 2022 - AT-1501 is renamed Tegoprubart. You can read more here.
Jan 2022 - The trial reached full enrollment. You can read more about it here.