- PharmAust (PAA) reveals its phase one trial using MPL for MND and ALS has slowed disease progression in 11 out of 12 patients
- Analysis shows Nfl protein concentrations in the plasma of blood do not increase following MPL treatment, an indicative sign the drug is working
- The company hopes the results will lead to orphan drug designation from the TGA and FDA for MND
- FightMND, Australia’s largest independent funder of MND research, is investing $881,085 in PAA’s phase I/II MND study
- PAA shares are up 9.59 per cent, trading at 8 cents at 11:40 am AEST
Pharmaust (PAA) has reported that its phase one trial using the lead drug, monepantel (MPL), for Motor Neurone Disease and Amyotrophic Lateral Sclerosis (MND/ALS) has successfully slowed disease progression in 11 out of 12 patients.
An interim analysis has shown that Neurofilament Light Chain (Nfl) protein concentrations in the plasma of participants have not increased following MPL treatment.
The company has announced that the research suggests a correlation between Nfl increases and motor neurone disease progression, with only one patient in all three cohorts displaying increased Nfl.
PAA has been dosing patients with MPL for up to 10 months, where it has shown signs of a reduction in the rate of degeneration and loss of motor neurons in the brainstem, with no signs of material adverse events.
“We are delighted with the results that 11 of 12 trial participants show no significant change in Nfl plasma concentrations and are “stable”,” PAA Executive Chair Dr Roger Aston said.
“MPL has emerged as a leading candidate with enormous potential to aid MND therapy development, and it’s a fantastic result for PharmAust that MPL is showing a clinical benefit.”
The trial uses an MPL tablet curated by the company, which hopes the results will lead to orphan drug designation from the Therapeutic Goods Administration (TGA) and the Food and Drug Administration (FDA) for MND.
FightMND, Australia’s largest independent funder of MND research, is investing $881,085 in PharmAust’s Phase I/II MND study.
MND is responsible for killing more than 100,000 people annually and affects more than 350,000 people, with a life expectancy of 27 months upon diagnosis.
PAA shares were up 9.59 per cent, trading at 8 cents at 11:40 am AEST.