PharmAust gets ethics committee green light for open-label MND extension study
Health & Biotech
Health & Biotech
Special Report: PharmAust will begin an open-label extension (OLE) study for monepantel (MPL) in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS) at Calvary Health Care Bethlehem in Melbourne after receiving approval from Monash Health Human Research Ethics Committee (HREC).
Clinical-stage biotech PharmAust (ASX:PAA) says the OLE multicentre study will span 12 months and is designed to enable the 12 participants from the Phase 1 MEND study to continue receiving MPL treatment.
Over the course of the study, patients will be administered a daily dose of 10 mg/kg body weight of MPL.
The OLE study is scheduled to start in February at Calvary Health Care Bethlehem and will led by Associate Professor Susan Mathers, with patients at Macquarie University, under the leadership of Professor Dominic Rowe, to soon follow.
PAA says all 12 patients have continued treatment with MPL to be eligible for the OLE study through a compassionate-use program administered by their treating physician, either Mathers or Rowe.
The first group of six patients enrolled in the Phase 1 MEND Study have all received treatment with MPL for more than 15 months now.
PAA’s Phase I MEND study involved 12 patients and was completed in December with top-line results on track to be released before the end of this current quarter.
The company also this week announced the US Food and Drug Administration (FDA) Office of Orphan Products Development has requested additional data to support the
MPL ODD application for the treatment of MND/ALS.
The company plans to submit results of its Phase I MEND study to the FDA to support its ODD request.
Figures from the International Alliance of ALS/MND Associations shows MND impacts over 350,000 individuals worldwide, resulting in more than 100,000 annual fatalities.
The disease is uniformly fatal, lacking any cure or effective treatment to halt its advancement.
On average, individuals diagnosed with MND can expect a life expectancy of ~27 months, with independent research indicating that a third of patients succumb within the first 12 months after diagnosis.
The OLE study will further investigate the hypothesis that MPL administration to individuals with MND/ALS can safely diminish disease-associated protein accumulation in motor neurons, providing therapeutic benefits.
The primary goal of the study is to evaluate the long-term safety and tolerability of MPL.
Secondary objectives include the assessment of biomarkers such as serum neurofilament/light chain and urinary p75ECD levels.
Efficacy endpoint efficacy include disease severity, cognitive function, respiratory health, and overall quality of life with assessments using established scales such as ALS Functional Rating Scale-Revised, Edinburgh Cognitive & Behavioural ALS Screen, Slow Vital Capacity, and ALSSQOL-R, respectively.
In pre-clinical programs, PAA has already shown that MPL can activate molecular pathways relevant to its development as a MND therapeutic drug.
If effective, MPL would reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem.
PAA chief executive Dr Michael Thurn says it is very positive that all 12 patients who completed the Phase I MEND study are in the position to continue treatment with monepantel for a further 12 months.
“The OLE study allows us to capture safety and efficacy data within a quality framework suitable for regulatory submission,” he says.
“We are thrilled to work again with experienced investigators Associate Professor Susan Mathers and Professor Dominic Rowe.
“Together, we hope our efforts bring about a much-needed new therapy for MND/ALS.”
This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.