PharmAust’s interim analysis to point the way forward for MND trial

PharmAust continues to progress the first phase of the clinical trial for its lead drug monepantel to treat Motor Neurone Disease with blood samples collected from all patients for interim analysis.

The samples from the 12 patients in Cohorts 1 and 2 will be analysed for how MPL is absorbed, distributed, metabolised and eliminated by the body with these pharmacokinetics (PK) study results expected within the week.

An analysis evaluating changes in biomarkers and pharmacodynamics is still pending. Pharmacodynamics is basically the activity in treating MND. Pharmacodynamics can be split into (i) actually slowing down progression and (ii) biomarkers indicative of slowing down progression.

PharmAust director, Sam Wright told Stockhead: “Both pharmacokinetics and pharmacodynamics are being determined in this interim analysis with the pharmacokinetics being the first study. It is vitally important that we can demonstrate absorption because without it we would not see an effect.”

All patients had previously been found to have tolerated the drug well and had elected to continue treatment.

PharmAust (ASX:PAA) notes that treatment-related changes from the baseline in this safety, tolerability, pharmacokinetic and preliminary efficacy study will include an analysis of functional rating scales, quality of life and cognitive assessment.

Besides paving the way for Phase-2 of the MND clinical trial, a favourable interim analysis will also pay off by allowing it to prepare for a Phase-2 clinical trial into the use of MPL for the treatment of human cancer.

The company will also continue dose escalation for Cohorts 3 and 4 to determine the optimum dose for the Phase-2 trial, which will establish the efficacy of MPL in treating MND.

Subject to approval at the next Safety Committee meeting patients in Cohort 1 will be escalated to Cohort 3 while further approvals will see Cohort 2 patients move to Cohort 4.

The Phase-1/2 MND trial remains on track to be completed in the third quarter of this year.

Much needed treatment

A successful Phase-2 clinical trial has the potential to significantly improve the quality of life for the more than 350,000 people affected by the disease globally.

According to the International Alliance of ALS/MND Associations, MND is invariably fatal with the average life expectancy of someone who has MND being around 27 months whilst current treatments only prolong survival by up to four months.

More than 100,000 people die from the disease annually.

For PharmAust, this is a significant market when combined with the potential to secure orphan drug designation from the US Food and Drug Administration that could in turn provide a number of financial and supportive benefits.

The MND/ALS addressable market is US$3.6 billion per annum, with existing third-party drug 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.