Oncology company PharmAust (ASX: PAA) has posted a strong quarter of business activity, led by a hefty grant last month for a phase 1 clinical trial to investigate the effects of lead drug monepantel on humans with motor neurone disease.
The company received $881,085 from independent funding organisation FightMND to commence the trial, which will be overseen by Dr Susan Mathers of Calvary Health Care in Melbourne, and will include a second trial site headed by Professor Dominic Rowe of the Centre for Motor Neurone Disease Research at Sydney’s Macquarie University.
Preparations for the trial are subject to approval from the Institutional Human Research Ethics Committee, and recruitment of patients is expected to commence in the new year.
The funding agreement allows PharmAust to own all intellectual property generated from the study.
Following in vitro work demonstrating inhibition of the SARS-CoV-2 (COVID-19) virus, PharmAust and Leiden University in the Netherlands agreed to evaluate monepantel’s suitability for ex-vivo human COVID-19 testing, using cultured human airway epithelial tissue.
The studies have started and a final data report is expected by year end.
PharmAust said monepantel may have an advantage over other drugs in development given that it has already been used in human clinical trials and has a high safety profile.
Remdesivir – the only anti-viral drug on the market currently approved for the treatment (but not cure) of COVID-19 infection – is an intravenous therapy, whereas monepantel can be administered orally in tablet form.
This means patients could be treated earlier when they first test positive rather than in a hospital setting.
Cancer cell screening
During the quarter, researchers in the cell death and survival laboratory at Victoria’s Olivia Newton-John Cancer Research Institute conducted an RNA-sequencing screen to investigate how the entire genome of cancer cells respond when treated with monepantel.
The approach identifies which genes are “switched on” and which are “switched-off” by the drug.
One non-cancer cell line was used as control and the three cancer cell lines were melanoma, lung cancer and ovarian cancer.
Researchers found that changes in gene expression in the non-cancer cell genome were relatively modest compared to the changes in the three cancer cell lines and this observation supports previous studies indicating that monepantel does not affect non-cancer cells and has selectivity towards cancer cells.
Specifically, the screen demonstrated that cancer cell genes involved in promoting cell division were suppressed, while those involved in inducing cell death (apoptosis and autophagy) were induced.
Canine cancer studies
Following the conclusion of its phase II canine cancer trial in June, PharmAust confirmed that some veterinarians from the trial have continued to evaluate monepantel in pet dogs with cancer under compassionate use.
During the quarter, these veterinarians voiced their intention to continue working with PharmAust in future formalised studies and PharmAust confirmed it has sufficient monepantel and tablets for all canine trials.
“If our strategy is correct and we can demonstrate that monepantel is consistently effective in tumour regression in most canine patients, this will likely strengthen our negotiating position with third party veterinary and human pharmaceutical companies,” the company said.
The company added it would contact a wider group of global pharmaceutical companies to discuss veterinary collaborations and engage in discussions on identifying the optimal cancers to target.
Human cancer trial
PharmAust continues to make steps towards progressing the evaluation of monepantel in human trials.
During the quarter, the company conducted a tablet re-formulation and pharmacokinetic studies aimed at increasing the uptake of monepantel into the blood and reducing tablet numbers.
It currently has two separate batches of GMP-grade monepantel under stability studies to test the new formulation’s shelf life, with early findings showing a robust tablet to support submission filings to human trial ethics committees.
PharmAust is in discussions with prospective European oncologists with the aim of starting a phase II human cancer trial, as a follow on from the phase I clinical trial undertaken at Royal Adelaide Hospital in 2015.
It has also engaged a third GMP manufacture program to cater for the trial which is expected to commence in 2022.
PharmAust’s cash position at 30 September was $3.9 million.
The company said it would be adequately funded to continue current activities during uncertain times and will continue to “demonstrate appropriate fiscal restraint”.