Ann: Interim analysis recommended in MND trial, page-2

Interim analysis recommended in MND trial
• Protocol amendment in MND trial to evaluate potential indicatory changes in
biomarkers and pharmacodynamics
• Mid-point outcomes resulting from the protocol amendment in the MND trial will
include information on absorption and pharmacokinetics of MPL in patients
• An interim analysis of target engagement and treatment-related changes in
biomarkers is expected, following review after completion of dosing of Cohort 2
• PharmAust will continue with the MPL dose escalation for Cohorts level 3 and 4
to determine the optimum dose level for a Phase 2 trial
• Emerging indicatory evidence for MND severity and progression during the
course of the trial will also be examined
2 March 2023 – Perth, Australia: PharmAust Limited (ASX: PAA & PAAO), a clinical-stage
biotechnology company evaluating the use of Monepantel (MPL) in cancer and motor neurone disease
(MND), is pleased to announce it will undertake an interim analysis of preliminary biomarkers and
efficacy markers on completion of dosing of the last patient of Cohort 2 in its MND trial.
This interim analysis constitutes an approved protocol amendment to the initial approved protocol as
recommended by the Principal Investigator.
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. Furthermore, prognostic indicators and several disease-related biomarkers will be
measured.
Following this protocol amendment, PharmAust will continue with the MPL dose escalation for Cohorts
level 3 and then 4 (subject to Safety Committee reviews).
This process may facilitate determining the optimum dose level or range for a Phase 2 trial.
PharmAust Executive Chairman, Dr Roger Aston commented:
“Subject to trial outcomes under this protocol amendment, PharmAust expects to prepare the ground
for the MND trial to be expedited into Phase 2. The protocol amendment and interim analysis has been
recommended by the Principal Investigator.
PharmAust 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.”
PharmAust acknowledges grant funding from the FightMND charity to support this trial.
The Board authorises this announcement.