PharmAust (ASX:PAA) has announced the commencement of Cohort 3 dosing in its current phase 1/2 trial, testing the effects of Monepantel (MPL) in individuals living with motor neurone disease (MND).
The Trial Safety Committee recently approved patients in Cohort 1 to be promoted to Cohort 3, receiving a larger dose of MPL. The company said MPL had displayed a positive safety profile with no serious adverse events observed, and all patients elected to continue with MPL treatment.
PharmAust said that interim pharmacokinetic study results show that the reformulated tablets developed specifically for this trial produce steady-state MPL plasma levels within the predetermined therapeutic range.
The company said that biomarkers being assessed through the current trial are helpful diagnostic tools in MND that can also be used to track disease progression and determine the likely course of the disease.
The current trial's evidence will help inform the design of subsequent human studies and may support a more rapid approval pathway that includes TGA and FDA orphan drug designations.
PharmAust executive chairman Dr Roger Aston said, “The analyses of biomarkers, in conjunction with the recently reported pharmacokinetic study results, are expected to be significant drivers of shareholder value. With good results in the clinic, the company intends to pursue the fastest possible route to approval for the benefit of MND patients and their families.”
“We are delighted with trial progress to date and look forward to confirming MPL’s clinical benefits to MND patients soon.”