PharmAust is conducting multiple studies on lead drug monepantel, as it looks to commercialise the drug for multiple applications.

Clinical stage biotech company, PharmAust (ASX:PAA), continued to make progress in the September quarter – advancing studies in lead drug Monepantel (MPL)  as it heads towards commencing human clinical trials in cancer, COVID-19 and Motor Neurone Disease (MND).

In addition, the company is about to advance to a Phase 3 trial in dogs with B-cell lymphoma, following encouraging results from Phase 2.

Data from the Phase 2 trial has shown that combining MPL with prednisolone more than doubles the life expectancy of pet dogs, compared with standard-of-care (prednisolone alone).

PharmAust is also making progress in COVID-19 testing, with data from three independent laboratories showing the capacity of MPL and MPLS (metabolite monepantel sulfone) to protect against cell death in-vitro, following infection with SARS-CoV2.

In moving its programs towards the clinic, PharmAust has also commenced GMP-quality production of MPL, and is in mature negotiations with a European CRO to identify trial sites for testing MPL in people infected with the virus causing COVID-19.

Other viral infection studies are also progressing, with a T-lymphotrophic virus-1 infections study being conducted following a partnership agreement with the Walter and Eliza Hall Institute in Melbourne.

PharmAust continues to take key steps towards progressing the evaluation of MPL in a Phase 2 human cancer trial –  with clinical interest focusing on glioblastoma, esophageal, gastrointestinal and pancreatic cancer.

Commencement of a human cancer Phase II trial on MPL is expected in Q3/4 of CY 2022.

Meanwhile, a Phase I/II Human Trial in Motor Neurone Disease (MND) is on the horizon after the company received a $900k funding commitment from FightMND, the largest independent funder of MND research in Australia.

PharmAust is currently in final negotiations with organisations that will provide the necessary MND trial services.

The company is well funded, with a cash balance of $2.5m at quarter-end.

Phase II Canine Cancer Trial

In the Phase 2 clinical trial, PharmAust has determined an optimum drug plasma range for anticancer activity that produces minimal side effects for canines.

Data from Phase 2 has shown that combining MPL with prednisolone more than doubles the life expectancy of pet dogs, compared with standard-of-care (prednisolone alone).

Of the seven pet dogs treated with drug plasma levels of MPL in the optimum range, six achieved stable disease and one had a partial response (60% regression), with some tumours completely disappearing. Side effects were minimal or not detected.

And of the six pet dogs that achieved stable disease progression, five continued to take MPL after the trial in combination with prednisolone. To date, these five dogs have achieved much higher than expected mean and median survival times, of 125 and 138 days respectively. This 138 day median survival compares favourably with a recently reported 60 day median survival for similar pet dogs treated only with standard-of-care prednisolone.

All pet dog owners reported very high quality of life for their pet dogs while taking MPL. A testimonial page has been added to the PAA website.

In comparison, the most common side effects of a dog being treated with chemotherapy include gastrointestinal effects (vomiting, diarrhea, or loss of appetite) and decreases in blood cell counts. Also, during chemotherapy, owners need to take precautions when handling their pet’s waste. Drugs may be excreted in the urine and faeces, so it is not advisable for children to play with their pets.

“We’ve come a long way in the canine testing, and we’re confident to start Phase 3 trial with aims to commercialise in the near future,” Dr Richard Mollard, PharmAust’s chief scientific officer, told Stockhead earlier.

Phase II Human Cancer Trial

PharmAust continues to take key steps towards progressing the evaluation of MPL in human trials.

The company has identified clinical oncology units in Italy and the United Kingdom to evaluate the company’s new MPL tablet in a Phase II trial, and to discuss the optimal type of cancers to target.

Clinical interest has focused on glioblastoma, esophageal, gastrointestinal and pancreatic cancer.

The company says it will continue to look for further sites to broaden recruitment possibilities, with commencement of a human cancer Phase II trial expected in Q3/4 of CY 2022.

COVID-19 and HTLV-1 Testing

Following its recent patent application, PharmAust is continuing to make progress on MPL to treat viral diseases.

Studies completed by three independent laboratories have finished examining the effects of MPS and MPLS on SARS-CoV2, the causal agent of COVID-19 disease.

All three laboratories have demonstrated that both MPS and MPLS protect against cell death in-vitro, following infection with SARS-CoV2.

A recent breakthrough in the US on antiviral molnupiravir has also shone the spotlight on PharmAust’s own testing of MPL.

In October, US-based Merck and partner Ridgeback Biotherapeutics have called for an early end to their testing of molnupiravir – a potential drug studied  as a COVID-19 antiviral.

The testing was halted  in order to seek regulatory emergency-use authorisation from the US FDA.

According to Merck’s press release, studies showed that only 7.3% of patients who received molnupiravir were hospitalised through Day 29, compared with 14.1% of placebo-treated patients who were hospitalised or died.

Molnupiravir works by disrupting the replication of the virus by mimicking the production of cytidine and uridine  in our body’s RNA (ribonucleic acid).

This study suggests that MPL might work well together with remdesivir as a cocktail treatment.

“We believe that MPL may hopefully be one component of a cocktail (with  remdesivir ) later on down the track, which might include remdesivir or  molnupiravir, for example” Dr. Mollard told Stockhead.

To further understand the anti-viral activity of MPL and to broaden the scope of targets for MPL’s use, PharmAust has teamed up with Walter and Eliza Hall Institute in Melbourne.

The teams will  investigate the effects of MPL upon human T-lymphotrophic virus-1 (HTLV-1) infections in vitro.

The study of HTLV-1 is of particular significance due to the readily available nature of highly relevant in-vitro and in-vivo preclinical virus infection models, potentially providing PharmAust with further data to support future human trials.

Phase I/II Human Trial in Motor Neurone Disease

Following a $900k funding grant from charity FightMND, PharmAust is set to commence the Phase I clinical trial on its lead drug Monepantel, for patients with motor neurone diseases (MND).

Founded in 2014 by former AFL player and coach Neale Daniher,  FightMND is a charity established with the purpose of finding effective treatments and a cure for MND.

The study will look specifically into patients with Amyotrophic Lateral Sclerosis/ Motor Neurone Disease (ALS/MND) – a condition that became widely known following the affliction of renowned physicist, Stephen Hawking.

With success in the clinic, PharmAust expects that in due course MPL will receive orphan drug designation by the US FDA for the indication of MND.

Manufacturing of GMP-grade MPL active ingredient tablets

PharmAust has commenced production of two batches of 10kg each of GMP-grade MPL for research and development (R&D) purposes and clinical trials in humans.

The GMP-grade MPL compound is being manufactured in collaboration with Syngene International, an integrated research, development and manufacturing services company, and Catalent Pharma Solutions (NYSE: CTLT).

These manufactured GMP-grade monepantel tablets will be suitable for use in the upcoming human trials.

Success in the manufacture of this demonstration batch means that PharmAust is now in the process of scaling up a GMP batch ready for the FightMND and COVID-19 trials to commence around April 2022.

Epichem’s waste to fuels technology

During the quarter, PharmAust’s wholly owned subsidiary Epichem paid off its loan that was used to build a major laboratory expansion in Technology Park, Western Australia.

The company had initially sought an export loan from Export Finance Australia (EFA) in 2015, with assistance from PharmAust.

Epichem has built two world class labs at Technology Park, with the equipment and expertise ready to support Epichem’s drug discovery projects.

During the quarter, Epichem  announced a critical milestone in its efforts to develop and promote a disruptive waste-to-fuels technology.

The company has successfully completed building a flow reactor using the  benchtop Oxidative Hydrothermal Dissolution (OHD) technology.

The flow reactor is a world-first, with the potential to turn a wide range of waste and biomass feedstock into valuable energy products.

PharmAust says that potential revenue streams from this technology could come from the removal of organic waste, its conversion to valuable end user products, as well as reduction of landfill.

Data shows that bioplastics will grow into a $28 billion market in five years time, while the bio-ethanol market is expected to double to $65 billion in five years time.

This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.