Patients from the first cohort treated with PharmAust’s lead drug monepantel under the Phase 1/2 Motor Neurone Disease (MND) clinical trial continue to show no signs of material adverse events.

Importantly, the principal investigator has recommended that a patient who remained “stable” after being on MPL for six months be elevated to Cohort 2, which is accompanied by a doubling of the dosage.

The same patient is expected to be moved to Cohort 4 along with the other Cohort 2 patients once the Safety Committee grants PharmAust (ASX:PAA) the green light to move on to Cohorts 3 and 4 – with their corresponding dosage increases to determine the optimum dose level for a possible Phase 2 trial.

Phase 1 of any clinical trial is carried out to evaluate the safety of the drug while Phase 2 will determine its efficacy and further evaluate safety.

“Under the flexible protocol and based on advice from the principal investigator, we are implementing a protocol deviation to allow transfer of a patient from Cohort 1 to Cohort 2, with the associated increase in the dosage of MPL,” executive chairman Dr Roger Aston said.

“The absence of any material adverse events in Cohort 1 to date is highly encouraging as is the potential stability associated with the patient being transferred to Cohort 2.”

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.

What is MND?

A quick primer on Motor Neurone Disease, from the Fight MND website:

Motor Neurone Disease (MND) is the name given to a group of diseases in which the nerve cells (neurones) controlling the muscles that enable us to move around, speak, swallow and breathe fail to work normally and eventually die. With no nerves to activate them the muscles gradually weaken and waste.

With no residual muscles or strength, MND patients are left motionless, mute, and trapped within their once active bodies.

In the majority, the mind and intellect are left intact, with the sufferer bearing witness to it all.

The disease is progressive, meaning the symptoms get worse over time. MND has no cure and there is no effective treatment to reverse its progression.

The patient in PharmAust’s clinical trial has been on MPL for six months with no signs of material adverse events and is “stable” which suggests there is no evidence of disease progression which would be a fantastic outcome.

MPL a promising treatment for MND

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, and has no known cure while the only approved drug treatment – Riluzole – prolongs survival by 3-4 months without reversing the damage already done to motor neurons.

Persons taking Riluzole must also be monitored for liver damage and other possible side effects.

Despite this, Riluzole already reaches about US$1bn in annual sales out of the estimated ALS/MND addressable market of US$3.6bn.

MPL has already been shown to slow disease progression in MND models in pre-clinical studies while the lack of any material adverse events in patients dosed to date is certainly promising.

Should MPL move on to the Phase 2 trial and demonstrate its efficacy while continuing to be well-tolerated by patients, it could open up a sizeable market for PharmAust, particularly when combined with the potential to secure orphan drug designation from the US Food and Drug Administration, which provides a number of financial and supportive benefits.




This article was developed in collaboration with PharmAust (ASX:PAA), 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.