Clinical stage biotech PharmAust’s Phase-1/2 clinical trial is heading in the right direction with patients tolerating its lead drug and electing to continue treatment.

PharmAust (ASX: PAA) has provided a positive update after completing the day 28 dosing of the final patient in the second cohort in its Phase-1/2 clinical trial of its lead drug candidate monepantel (MPL) in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).

Recruitment of six patients for the second cohort was announced on March 30. PAA said All 12 patients from cohorts 1 and 2 have tolerated the MPL tablets well and elected to continue MPL treatment.

Five patients have now surpassed the 6-month-mark on MPL without any safety issues, and one patient has shown stable disease.

The company said 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.

Patients for the trial were enrolled at two sites including Calvary Health Care Bethlehem, Statewide Progressive Neurological Disease Service at Caulfield South in Melbourne and The Centre for Motor Neurone Disease Research, Faculty of Medicine and Health Research Macquarie University, in Sydney.

“Patients electing to continue using Monepantel after participating in the current trial for Motor Neuron Disease, provides increased comfort on our safety data particularly when viewed against the Riluzole (current standard of care) safety data reporting a 14% rejection rate by patients for ongoing use,” PAA executive chairman Dr Roger Aston said.

Interim results due late May

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

Treatment-related changes from baseline in this safety, tolerability, pharmacokinetic and preliminary, efficacy study will include an analysis of functional rating scales, quality of life and cognitive assessment.

Further, prognostic indicators and several disease-related biomarkers will be measured.

The Company expects to proceed to Phase 2 with favourable efficacy biomarker results under the interim analysis

PAA said it will continue with the MPL dose escalation for Cohorts 3 and 4 during the interim trial analysis to determine the optimum dose level for the Phase 2 trial.

Subject to Safety Committee reviews, the patients from Cohort 1 will move to Cohort 3 and the patients from Cohort 2 will move to Cohort 4.

PAA recently took receipt of the roughly 10,000 MPL tablets to ensure continued availability for the Phase-1/2 MND trial and ongoing maintenance dosage along with sufficient supply for other human clinical trials.

MPL a promising treatment for MND

Should PAA 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, 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.

According to the International Alliance of ALS/MND Associations, MND affects more than 350,000 people globally with more than 100,000 people dying from the disease annually.

MND 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 billion per annum, with Riluzole already reaching ~US$1 billion in annual sales.




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.