PharmAust (ASX:PAA) says it has completed enrolment of the second cohort of six patients 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 said the study aims to determine the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in individuals living with MND. The trial is open-label and comprises four cohorts with escalating MPL doses.
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.
PharmAust said 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 will also 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.
The study is being funded by a commitment of $881,085 by FightMND, the largest independent funder of MND research in Australia.