PharmAust has been given the green light to progress with an escalated dose of its lead drug monepantel for its Phase 1/2 Motor Neurone Disease (MND) clinical trial.

This follows the Trial Safety Committee granting its approval after an evaluation of the first cohort of patients confirmed that there were no reported safety issues or SAEs (serious adverse effects) as well as established drug absorption.

PharmAust (ASX:PAA) will continue supplying MPL to all six initial patients in Cohort 1 who have elected to remain on treatment and has started patient recruitment for the next MPL dosing level with four patients currently undergoing screening.

PharmAust has developed and manufactured a bespoke MPL tablet for the trial.

MND a multi-billion dollar market

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 of the world’s population, and kills more than 100,000 people every year.

It is invariably fatal, with the average life expectancy of someone who has MND being just 27 months.

Unfortunately, at present there is no known cure to ALS/MND.

The first approved drug treatment for the disease, called Riluzole, became available in 1995. Clinical trials with MND patients showed that Riluzole prolongs survival by around 3-4 months, mainly in those with difficulty swallowing. Riluzole does not reverse the damage already done to motor neurons, and persons taking the drug must be monitored for liver damage and other possible side effects.

The ALS/MND addressable market is US$3.6Bn with Riluzole already reaching ~US$1Bn in annual sales.

Funding and orphan drug hopes

The Phase1/2 study is being funded by a commitment of $881,085 made by FightMND, the largest independent funder of MND research in Australia.

Should the trial be successful, PharmAust is hopeful of receiving orphan drug designation by the US FDA for the indication of motor neurone disease.

The Orphan Drug Act provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor, which will provide a number of financial and supportive benefits.




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.