PharmAust Limited, a Perth-based clinical-stage biotechnology company, 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.

Despites 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 with a particular shape and size to help MND patients swallow them and to reach specific blood drug levels targeted for this disease.

Auxiliary preparations for the MND trial commencement are nearing completion awaiting final technical and regulatory sign-offs of the tablet. Recruitment will follow Therapeutic Good Administration Clinical Trials Notification Licence activation, expected this month.

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 supporting the Big Freeze 8 and Fight MND.”

This announcement is authorised by the Board.