Good things come in small packages, like PharmAust’s (ASX: PAA) newest iteration of their monepantel (MPL) tablets. 

The clinical stage biotechnology company has manufactured smaller versions of their lead asset, MPL, a potent small molecule drug that has been shown to affect key pathways associated with cancer and other diseases. PharmAust have studied MPL for use in many indications, notably motor neurone disease, cancers and COVID-19. 

The Company specialises in repurposing already on-market drugs to treat new indications in both humans and animals. Repurposing of existing drugs lowers the risk associated with clinical trials and approvals as well as the cost of development. 

MPL is the focus of much of PharmAust’s energy, having been looked at in Phase 1 clinical trials in humans and Phase 2 clinical trials in dogs. The drug has shown anticancer activity and is being pursued for the treatment of B-cell lymphoma in dogs.

The drug has also shown promise for the treatment of amyotrophic lateral sclerosis (ALS) and motor neurone disease (MND). ALS and MND are neurodegenerative diseases that cause progressive loss of essential bodily functions such as respiration and movement. Life expectancy after diagnosis is only 27 months. 

PharmAust have initiated a study in people with MND following promising preclinical data showing that MPL has the potential to slow disease progression and the rate of neurodegeneration. 

The Company has also made headway with their COVID-19 program. In conjunction with the Walter and Eliza Hall Institute (WEHI), a leading biomedical research facility, PharmAust has made significant inroads into investigating the compound’s antiviral properties, creating a robust preclinical data package before heading into trials. 

A total of 2,000 smaller MPL tablets have been manufactured for a demonstration batch. The process used is the same as that of larger tablets. 

These smaller tablets will be used for upcoming trials for motor neurone disease and COVID-19 where the dosage required is smaller than that of cancers. 

Chief Scientific Officer of PharmAust, Dr Richard Mollard said: “It is a great outcome to convert from large to small dose tablets so quickly. The smaller dose tablets were also shape-modified to facilitate swallowing for individuals with motor neuron disease. Having the stability set up for the demonstration batch now means that PharmAust is looking forward to having three month accelerated stability studies completed in time to support an early start to the clinical trials once the GMP tablet batch is finalised.” 

As the world rushes to find solutions to the COVID-19 pandemic it remains to be seen if PharmAust will be a competitor with their antiviral. Despite oncology being the most lucrative market by far, the preclinical work in non-cancer diseases could prove invaluable to PharmAust in the long term.