PharmAust confirms safety of MPL, poised to up dosage in MND trial

PharmAust could soon move patients in the first cohort of its clinical trial into the use of monepantel to treat Motor Neurone Disease to the third cohort with an increased dosage after confirming its safety.

The Trial Safety Committee found that MPL remains well tolerated by all MND patients in Cohort 1 and 2 with no reported safety issues or serious adverse events (SAEs).

Once the ongoing pharmacokinetics (PK) study confirms absorption of the drug, PharmAust (ASX:PAA) will move all six patients in Cohort 1 to Cohort 3 with a corresponding increase in dosage.

The trial is open label and comprises a four week escalating dose of MPL, which had been found in pre-clinical studies to have the potential to activate molecular pathways relevant to the treatment of MND.

Current PK studies are expected to pave the way for Phase 2 of the clinical trial while dose escalation for Cohorts 3 and 4 will allow the company to determine the optimum dose for the Phase-2 trial, which will establish the efficacy of MPL in treating MND.

The Phase-1/2 MND trial remains on track to be completed in the third quarter of this year.

MND is invariably fatal to the more than 350,000 people affected by the disease globally with the average life expectancy of someone who has MND being around 27 months whilst current treatments only prolong survival by up to four months.

More than 100,000 people die from the disease annually.

PharmAust’s Phase 1/2 clinical trial is being funded by a commitment of approximately $900,000 by FightMND, Australia’s largest independent funder of MND research.

FightMND fights the disease

While MND is life-shortening, research has shown that early and timely treatments can help patients lead a better and longer life. One Aussie foundation that’s been at the forefront of this research is the charity organisation, FightMND.

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

A former Essendon player and Melbourne Football Club Coach, Daniher needs little introduction.

Debuting for the Bombers in 1979, Danihner is one of four brothers making up part of the Daniher football dynasty.

His football career, although plagued by injury, was well respected as he won widespread praise and accolades for his actions both on and off the field. But in 2013, Dahiner was diagnosed with MND.

Unable to speak now, Dahiner’s continued determination to live life to the fullest has been a tremendous inspiration not just for MND sufferers, but also for millions of Australians.

CEO of FightMND, Dr. Fiona McInstosh, said her organisation is pulling out all the stops to find a cure.

Since 2014, FightMND has invested nearly $70 million into world-class MND research to find effective treatments and a cure – including 14 clinical trials and 24 drug development projects,” Dr. McIntosh tells Stockkhead

“As a result of this investment, Australians living with MND have more opportunities to participate in research or clinical trials than ever before.

“By funding innovative research and the infrastructure that underpins it, FightMND are helping to accelerate progress towards a cure.

“We are driving discoveries forward by building capacity and capabilities across the whole MND research sector.”

“Beanie On, Play On”

This year will be the ninth consecutive year that FightMND will feature in the AFL’s ‘Big Freeze’ match.

The Queen’s Birthday event is one of the biggest on the football calendar, where AFL athletes and supporters band together to help raise funds for FightMND.

This year’s event will be held during the half time break of the match between Collingwood and Melbourne, played at the AFL’s “home of football”, the Melbourne Cricket Ground.

It will once again feature the “ice challenge”, where past and present athletes take a slider plunge, and splash into a pool of freezing water.

Dr. McIntosh says that while MND is a brutal disease, we still need to find time for moments to laugh and cherish our time together, which is what the Big Freeze is all about.

“It helps us find those moments of fun and humour – we’re playing on and we’re choosing to laugh in the face of this Beast of a disease, as we watch our favourite Australian celebrities plunge into the ice,” she says.

“It’s a special moment of celebration that pushes MND into the spotlight. It’s FightMND’s signal to the world that the fight to find a cure is on.

“Every beanie sold or donation made during the Big Freeze takes us another step closer to cure – so please give generously at fightmnd.org.au and help us rid the world of MND,” Dr McInstosh adds.

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.