PharmAust moves closer to clinical trial of MPL in motor neuron disease
PharmAust 计划于 5 月正式开始 MPL 在 MND 的 1 期临床试验。
Clinical-stage biotech PharmAust (ASX: PAA) is closer to starting a phase 1 clinical trial evaluating its monepantel (MPL) drug in motor neuron disease (MND) after receiving its first instalment of a funding commitment and appointing Alithia Life Sciences to manage the study.
FightMND is the largest independent funder of motor neurone disease research in Australia and committed $881,085 in September 2020 to the upcoming clinical study.
The organisation has now paid the first instalment of $201,615, which was dependent on PharmAust completing the first milestone.
The first milestone of PharmAust manufacturing MPL to current good manufacturing practice grade (cGMP) and this has now been achieved.
A second instalment of $99,230 is payable to PharmAust upon completion of the one-month GMP accelerated stability study of the newly prepared MPL tablets, with this study already underway.
FightMND will pay the third instalment of $173,034.80 once the trial has begun, which is scheduled for next month.
Trial manager
PharmAust has appointed Alithia to oversee and support the phase 1 trial, including providing project and site management.
The company’s appointment to oversee the study is fully funded through FightMND’s drug development grant.
PharmAust chief scientific officer Dr Richard Mollard said PharmAust was “delighted” to work with the “highly experienced” Alithia team.
“We look forward to trial commencement and providing positive outcomes for people enrolled in the trial,” Dr Mollard added.
MPL study in MND
The upcoming trial will test the safety and tolerability of MPL in motor neurone patients.
It will also identify any signs that MPL can slow down the disease.
This data will be combined with animal studies to determine whether to proceed to a larger phase 2 clinical study.
PharmAust describes MPL as a novel, potent and safe inhibitor of the mTOR pathway. This pathway has key influences in cancer growth and neurodegenerative diseases.
The drug has already undergone evaluation in phase 1 clinical trials in humans and dogs, with preliminary evidence indicating it has anticancer activity in humans. In dogs, MPL demonstrated objective anticancer activity.
MND is the name for a group of diseases that affect motor nerves, or motor neurons.
In this disease, the neurons die, which causes the muscles to weaken and eventually results in paralysis.