PharmAust is moving on to the next stage of its Phase 1/2 Motor Neurone Disease (MND) trial after its lead drug monepantel was found to be well tolerated by patients at the first dosing level.

Further highlighting the success of the treatment to date, all six of the patients in the first cohort have chosen to continue with the monepantel (MPL) treatment for MND – also known as Lou Gehrig’s disease.

PharmAust (ASX:PAA) noted that subject to satisfying safety, progressive cohorts of patients will receive escalating doses of MPL.

The company will also start patient recruitment at the next dosing level later this month and will continue to evaluate safety and efficacy in MND patients treated with rising dose levels of MPL that may enable progress to a Phase 2 study to determine its efficacy.

Additionally, the safety and efficacy data from the MND trial will be used to facilitate a Phase 2 study in COVID 19 patients given that the same drug is used for both studies.

MND a global issue

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

It is invariably fatal, with the average life expectancy of someone who has MND being just 27 months.

The ALS/MND addressable market is US$3.6Bn with Riluzole already reaching ~US$1Bn in annual sales.

MPL, which is 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.

The Phase1/2 study is being funded by a commitment of $881,085 made by FightMND, the largest independent funder of MND research in Australia.

PharmAust also expects success in the trial to lead to MPL receiving 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.




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


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