PharmAust’s MND trial successfully completes third patient cohort – Nokturnal

PharmAust Limited (ASX: PAA) has completed its third cohort of six participants in its Phase 1/2 clinical trial of its lead drug candidate monepantel (MPL) in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).

The company has now completed the day 28 dosing of the final patient in the third cohort.

Importantly, all participants from Cohorts 1, 2 and 3 have elected to continue on MPL treatment.

All participants have tolerated the MPL tablets well, and the Safety Monitoring Committee will review data from each dosage level for safety and pharmacokinetic effects.

The Phase 1/2 clinical study is determining the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in patients living with MND. The trial is open-label and comprises a four week escalating dose of MPL. The patients were enrolled at two sites: Calvary Health Care Bethlehem, Statewide Progressive Neurological Disease Service, Caulfield South and The Centre for Motor Neurone Disease Research, Faculty of Medicine and Health Research Macquarie University, Sydney.

Neurofilament Light Chain results

The Principal Investigator for the trial has previously recommended undertaking an interim analysis of preliminary biomarkers and efficacy markers on completion of dosing of the last patient of Cohort 2.

So far, there is very encouraging data coming from the interim analysis. MPL is proving itself with every biomarker having pointed to a benefit.

The results of the highly specialised testing of Neurofilament Light Chain (NfL) are expected in the coming weeks. The most recent advice is that additional replacement parts for the Simoa machine have been received from the US and an engineer from Melbourne is due to install next week. If the Simoa machine is still un-operational, PharmAust has identified another laboratory who will ensure the NfL assay results can be processed in a similar timeline.

MPL a promising treatment for MND

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people every year. The disease is invariably fatal with the average life expectancy of someone who has MND being around 27 months. The MND/ALS addressable market is US$3.6 billin per annum with Riluzole already reaching ~US$1 billion annual sales.

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. PharmAust notes patients have been dosed with MPL for up to nine months in the clinical trial with no signs of material adverse events and appear “stable”.

PharmAust demonstrated in its preclinical programmes that MPL has the potential to activate molecular pathways relevant to the treatment of MND. MPL could potentially reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem. There are also a number of surrogate clinical endpoints to be determined during the trial. PharmAust has developed and manufactured a bespoke MPL tablet for the trial.

With success in the clinic PharmAust hopes that in due course MPL could receive orphan drug designation by the TGA and FDA for motor neurone disease. Such designations come with financial and supportive benefits and this opportunity is being evaluated by PAA.

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