PharmAust to proceed with early-stage ALS/MND trial

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PharmAust (ASX:PAA) has announced it will proceed with a new phase one clinical trial to assess monepantel (MPL) in patients with amyotrophic lateral sclerosis/motor neurone disease (ALS/MND).

The company said the trial will be funded and conducted in collaboration with FightMND, Calvary HealthCare Bethlehem (Melbourne) and Macquarie University (Sydney).

ALS/MND is a group of diseases that affect nerve cells controlling vital everyday functions including movement and breathing. ALS/MND is rare and invariably fatal disease. The average life expectancy with MND is just 27 months. 

PharmAust said it has already shown in its preclinical programs that MPL has the potential to activate molecular pathways relevant to the treatment of MND. If effective, MPL could reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem. There are also a number of surrogate clinical endpoints that will also be determined during the trial. 

Monash Health Human Research Ethics Committee (HREC) has approved the monepantel (MPL) clinical trial protocol.

The study is a multi-centre open-label trial that is designed to first test safety in 12 individuals living with ALS/MND according to a conventional dose escalation design, with each level of the dose-escalatione lasting 28 days.