PharmAust flags ‘steady state’ levels in Motor Neurone Disease clinical trial

In the latest update for PharmAust’s clinical trial of its lead drug candidate monepantel (MPL) in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS), data has shown dosages were well tolerated, and no Serious Adverse Events were observed – implying the drug has a good safety profile.

This is very important as patients are potentially expected to take the product for extended periods as MND is a permanent condition, and patients need to keep taking the drug to slow or stop progression.

The company has also completed the interim pharmacokinetic (PK) analysis of plasma MPL and Monepantel Sulphone (MPLS) levels in MND patients who received MPL at a dosage of 2mg/kg for Cohort 1 and 4mg/kg for Cohort 2.

Usage of MPL tablets for an extended period provides evidence of accumulation of MPLS in plasma, and the data showed that MPL levels appear to reach a “steady state” on Day 1, and MPLS levels appear to reach a steady state before Day 8.

The concept of “steady-state” is a bit of a big deal in pharmacology, because achieving a “steady-state” suggests a dynamic equilibrium where drug concentrations maintain therapeutic levels for long and potentially indefinite periods.

Effective absorption and steady state to boot

The company says the data has provided preliminary evidence for a potential linear relationship between patient dosage and MPL and MPLS plasma concentrations.

“The outcome is excellent news for PAA, our proprietary tablets formulation shows effective absorption of MPL and the achievement of steady-state levels of MPLS, the active metabolite of MPL,” PharmAust (ASX:PAA) executive chairman Dr Roger Aston said.

“We are delighted that patients on our drug for over seven months have shown good tolerance and no Serious Adverse Events.

Additional reporting on the changes in MND biomarkers and pharmacodynamics is still pending, which will determine the activity of MPL and how long MPL stays within therapeutic limits.

“We now eagerly await the analysis of the biomarkers,” Dr Aston said.

Dose escalation approved by Safety Committee

Notably, the trial Safety Committee has approved dose escalation for patients in Cohort 1 to Cohort 3.

And subject to the continued safety of the escalated Cohort 1 patients to level 3, and approval from the Safety Committee, PharmAust will continue with the MPL dose escalation for Cohort 2 to level 4.

With success in the clinic, the company hopes that MPL could receive orphan drug designation by the TGA and FDA for MND.

This article was developed in collaboration with PharmAust Limited, 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.