New Australian Clinical Trial to Test a Cancer Drug to Treat Motor Neuron Disease

  • Phase 1 clinical trial of monepantel to treat motor neuron disease (MND) set to proceed
  • Clinical trial enabled by funding from FightMND including the Big Freeze 8 campaign
  • High grade monepantel tablets have been developed specifically for the trial
  • The Phase 1 MND trial set for May/June 2022 commencement


PharmAust Limited is undertaking a Phase 1 clinical trial of monepantel (MPL) for the treatment of motor neuron disease (MND) or Amyotrophic Lateral Sclerosis, enabled by contributions from the Australian Fight MND campaign.

Despite advancements in science, MND remains a terminal disease without effective treatment or cure.  FightMND’s funding of $881,085, is enabling PharmAust to initiate a Phase 1 trial on the effect of its lead drug candidate MPL on MND.  Monepantel is a novel, potent and safe inhibitor of the mTOR pathway that has demonstrated preliminary evidence of efficacy in a previous clinical trial to influence cancers and shown effects in preclinical investigations of neurodegenerative diseases such as MND.

PharmAust recently announced the shipment of its cGMP (current Good Manufacturing Practice) grade MPL tablets specifically designed for the MND trial which will occur at centres in Melbourne and Sydney. These tablets are designed in a particular shape and size to help MND patients swallow them and reach specific blood drug levels targeted for this disease.

The FightMND funded Phase 1 clinical trial on patients with MND is on track to complete formalities in May. PharmAust has appointed a Safety Monitor, has commenced screening patients and will commence recruitment and treatment of patients in June.

The Phase 1 trial will test the safety and tolerability of MPL in patients living with MND, and will look for signs that MPL can slow the progression of MND.  These data, with concurrent animal studies, will determine whether MPL will be tested through larger Phase 2 studies.

PharmAust’s Chief Scientific Officer Dr Richard Mollard stated, “We are pleased with the development of our Phase 1 MPL trial for MND, and look forward to commencing recruitment. We are grateful for the continued support from FightMND and thank everyone for supporting the Big Freeze 8 and Fight MND.”

Find out more about the PharmAust clinical trial