The Trial Safety Committee has approved patients in Cohort 1 to be promoted to Cohort 3 and receive an increased dosage. Pic via Getty Images
PharmAust begins Cohort 3 dosing in Phase 1/2 motor neurone disease trial
Health & Biotech
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Following the recent promotion of patients in Cohort 1 to Cohort 3, individuals will now receive a larger 6mg/kg dose of Monepantel treatment to combat motor neurone disease.
In line with expectations, PharmAust (ASX:PAA) says Monepantel (MPL) has demonstrated
an excellent safety profile with no serious adverse events observed and all patients electing to continue with its treatment.
Interim pharmacokinetic study results show that the reformulated MPL tablets developed by PAA, specifically for this trial, produce steady-state MPL plasma levels within the predetermined therapeutic range from Day 1.
These results also show that steady-state therapeutic levels of Monepantel Sulphone (MPLS) – the principal metabolite of MPL – are being reached before Day 8.
PAA believes the detailed understanding being developed about achieving steady-state MPL/MPLS plasma concentrations is central to determining the MPL dose required for therapeutic drug concentrations in a range of human diseases, including cancer.
Biomarker analysis
The biotechnology company is also assessing the use of biomarkers as a helpful diagnostic tool in MND that can also be used to track disease progression and determine the likely course of the disease.
Evidence gathered through the current trial will help to inform the design of subsequent human studies and may support a more rapid approval pathway that includes TGA and FDA orphan drug designations.
“The analyses of biomarkers, in conjunction with the recently reported pharmacokinetic study results, are expected to be significant drivers of shareholder value,” PAA executive chairman Dr Roger Aston says.
“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.”
What’s next?
It is anticipated that biomarker assays being run at each institution will be completed over the coming weeks.
PharmAust will report the combined analysis of these assays once this information is to hand.
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.
