PharmAust is looking to expedite the start of Phase-2 clinical trials of its lead drug monepantel for the treatment of Motor Neurone Disease.

As part of this push, the company plans to carry out an interim analysis of preliminary biomarkers and efficacy markers once it completes dosing of the last patient in the second cohort of its Phase-1 MND trial.

While patients in the first cohort have no reported safety issues or SAEs (serious adverse effects) as well as established drug absorption, which led to the start of dosing of patients for Cohort 2, the interim analysis will include information on absorption and pharmacokinetics (the movement of a drug into, through, and then out of the body) of MPL in patients.

PharmAust (ASX:PAA) noted that the interim analysis, which was recommended by the Principal Investigator, constitutes an approved protocol amendment to the initial approved protocol.

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.

It will also measure prognostic indicators and several disease-related biomarkers.

“Subject to trial outcomes under this protocol amendment, PharmAust expects to prepare the ground for the MND trial to be expedited into Phase-2,” executive chairman Dr Roger Aston said.

“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.”

MPL treatment for MND

The Phase-1/2 clinical study, which is funded by a commitment of $881,085 made by FightMND, is determining the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in MND sufferers.

According to the International Alliance of ALS/MND Associations, MND – an invariably fatal disease – affects over 350,000 of the world’s population, and kills more than 100,000 people every year.

MPL, which has also been tested for canine cancer (B-cell lymphoma), has been shown to slow disease progression in MND models in pre-clinical studies by clearing harmful materials in a motor neuron that stick together and make patients unwell.

A successful trial could allow PharmAust to apply for orphan drug designation by the US FDA for the indication of motor neurone disease.

The Orphan Drug Act provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor, which will provide a number of financial and supportive benefits.

It will also open up a US$3.6Bn market that is currently dominated by Riluzole which prolongs survival by around 3-4 months but does not reverse the damage already done to motor neurons.




This article was developed in collaboration with PharmAust (ASX:PAA), a Stockhead advertiser at the time of publishing.


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