Daniel McLoone Major Research Initiative

Published: February 2023

The second part of a Phase 1 clinical trial for the drug SPG302, developed by the US-based company Spinogenix, is due to begin soon in Australia.

SPG302 has been developed to target the synapse in nerve cells. The synapse is the site of contact between two nerve cells or the nerve cell and muscle that enable communication via chemical signals and is key for the brain control of muscles for movement.

One major aspect of MND is the loss of these synapses. This drug is designed to increase the number of synapses in the nerve cell.

The trial is the second part of a Phase 1 trial, which is the first step in testing whether a drug can develop into a viable treatment. The first part of the trial successfully tested how the drug behaves in healthy volunteers to check for any toxic effects and how long it lasts in the body.

This part of the trial will test the drug in MND patients, but only for a very short period (4 weeks), to make sure the drug behaves in the same way in patients as it does in healthy volunteers and to measure any changes in patients that may occur.

There will also be an open-label extension (OLE) of this part of the trial to enable participants to keep receiving the drug for up to a year.

The trial locations are in Adelaide, Sydney and Brisbane and will look to recruit 24 patients in total. The trial is not yet recruiting but further information can be found at clinicaltrials.gov or on our clinical trials page.

It should be noted that although it is exciting to have new drugs being tested and great to have the opportunity for Australian patients to be involved in new trials, this is at a very early stage and it will be a number of years before this drug might make it into the clinic for patients, if successful.