Clinical-stage biotechnology company PharmAust (ASX:PAA) reports it has completed its first cohort of patients for its Phase 1/2 Motor Neuron Disease (MND) and Amyotrophic Lateral Sclerosis (ALS) clinical trial.
This clinical trial aims to determine the tolerability, safety, pharmacokinetics, and the preliminary efficacy of oral monepantel (MPL) in MND sufferers. The trial is open label and comprises a 4-week escalating dose of MPL.
The company says 6 patients were recruited in December, 2022 and enrolled at the Calvary Health Care Bethlehem in Parkdale, Victoria, as well as the Faculty of Medicine and Health Research at Macquarie University in Sydney. PharmAust states all 6 patients in the first cohort have elected to continue on MPL treatment.
PharmAust reports the MPL tablets were ‘well’ tolerated by patients in the first cohort, and states the Safety Monitoring Committee will review data from each dosage level for safety and pharmacokinetics. This review will take into account factors including serious adverse events (SAEs), adverse events (AEs), safety blood results, ECG, vital signs, pharmacokinetic results, and cerebrospinal fluids.
The company says the progressive elevation of MPL levels as it progresses through the pharmacokinetic evaluation of MND will be indicative of the safe dosing levels for its planned COVID trial.
In the current trial, PharmAust reports levels of MPL are determined in serum after dosing over a 28-day period. The company states patient recruitment at the next dosing level of MPL is expected to begin later this month.
According to the International Alliance of ALS and MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people every year. It states the disease is invariably fatal, with the average life expectancy of a MND patient being about 27 months.
PharmAust reports that in preclinical programs, MPL has the potential to activate molecular pathways relevant to the treatment of MND, and could potentially reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem.
In addition, the company also states there are a number of surrogate clinical endpoints to be determined during the trial. Pending the outcome of the trial, the company states it hopes to receive orphan drug designation by the US Food and Drug Administration (FDA) for the indication of motor neuron disease.
PharmAust reports the clinical trial is being funded by a commitment of $881,085 made by independent MND research funder, FightMND.
PharmAust is an ASX-listed, clinical-stage biotechnology company developing therapeutics for humans and animals. The company specialises in repurposing marketed drugs which it states lowers the risks and costs of development.
The company’s lead drug candidate is MPL, a novel, potent, and safe inhibitor of the mTOR pathway which influences cancer growth and neurodegenerative diseases.