PharmAust’s interim Phase 1 data suggests MPL may inhibit motor neurone disease

An interim analysis of data from a Phase 1 clinical trial of PharmAust’s (ASX: PAA) lead drug candidate monepantel (MPL) has shown that neurofilament light chain (Nfl) protein concentrations in patient plasma do not increase following treatment.

MPL is being developed for the treatment of motor neurone disease and amyotrophic lateral sclerosis (MND/ALS).

Research suggests a correlation between an increase in Nfl and the progression of motor neurone disease however only one patient over three cohorts was reported to have shown any increase over a 10-month period.

The remaining 11 patients displayed no significant change in Nfl, which is believed to correspond to a stabilised progression of MND.

Interim results

Executive chairman Dr Roger Aston said he was impressed with the interim results.

“We are delighted with the fact that 11 of 12 trial participants showed no significant change in Nfl plasma concentrations and are considered to be ‘stable’,” he said.

“MPL has emerged as a leading candidate with enormous potential to aid motor neurone disease therapy development… it is a fantastic result for PharmAust that [the drug] is showing a clinical benefit.”

Pre-clinical potential

PharmAust has previously demonstrated in its pre-clinical programs that MPL has the potential to activate molecular pathways relevant to the treatment of MND.

It said the drug could potentially reduce the rate of degeneration and loss of motor neurons in the brainstem’s anterior horns and motor nuclei.

The company has developed and manufactured a bespoke MPL tablet for the Phase 1 trial and is monitoring several additional surrogate clinical endpoints.

If the trial is successful, it is expected that MPL will receive orphan drug designation for MND by Australia’s Therapeutic Goods Administration and the US Food and Drug Administration.

MND statistics

According to research, MND affects over 350,000 people globally and kills more than 100,000 people a year.

It is progressive and invariably fatal, with independent studies showing that one-third of patients succumb to the disease within 12 months from diagnosis.

MND has no cure and there is currently no effective treatment to reverse its progression.

The global MND/ALS addressable market is approximately $5.6 billion per annum, with sales of approved medication Riluzole already reaching $1.56 billion in annual sales.

As Australia’s largest independent funder of MND research, FightMND has invested $881,085 into PharmAust’s Phase 1/2 trials.