Clinical-stage biotechnology company PharmAust Limited (ASX: PAA) has received Trial Safety Committee approval to progress with an escalation of the MPL tablet dosing for Motor Neurone Disease (MND) patients.
The trial patients living with MND/Amyotrophic Lateral Sclerosis (MND/ALS) were evaluated for adverse events and pharmacokinetic information of MPL absorption. The Trial Safety Committee confirmed there were no reported safety issues or SAEs. The PK data also confirmed drug absorption.
PharmAust will continue to supply MPL tablets to all six patients in Cohort 1 that elected to remain on the treatment. The trial is open label and comprises a four-week escalating dose of MPL.
Patient recruitment at the next MPL dosing level has commenced with four new patients currently undergoing screening.
According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people every year. The disease is invariably fatal, with average MND life expectancy of about 27 months. The MND/ALS addressable market is US$3.6Bn per annum. While there is no cure for MND/ALS, Riluzole is the only drug licensed for treatment with ~US$1 billion annual sales.
PharmAust demonstrated in its preclinical programmes that MPL has the potential to activate molecular pathways relevant to the treatment of MND. MPL could potentially reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem. There are also a number of surrogate clinical endpoints to be determined during the trial. PharmAust has developed and manufactured a bespoke MPL tablet for the trial.
This Phase1/2 study is being funded by $881,085 by FightMND, the largest independent MND research funder in Australia.
With success in the clinic PharmAust is hopeful MPL could receive orphan drug designation by the US FDA for MND. Such designations come with financial and supportive benefits.