PharmAust (ASX:PAA) has received approval from Monash Health Human Research Ethics Committee (HREC) to commence an Open-Label Extension (OLE) study of monepantel (MPL) in patients with Motor Neurone Disease(MND)/Amyotrophic Lateral Sclerosis (ALS) at Calvary Health Care Bethlehem, Melbourne.
The OLE study is a multicentre, 12-month study designed to allow the 12 patients who participated in the Phase 1 MEND study to continue to receive treatment with MPL.
Patients will receive a daily dose of 10 mg/kg body weight of MPL for 12 months. The study is expected to commence at Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers, in February 2024, with patients at Macquarie University, led by Professor Dominic Rowe, to follow soon after.
PharmaAust said the OLE study will further test the hypotheses that MPL administration to individuals living with MND/ALS will safely reduce disease-associated protein accumulation in motor neurons and provide therapeutic benefits.
The study's primary objective is to assess the long-term safety and tolerability of MPL. The secondary objective is to assess the biomarkers and efficacy endpoints.
PharmAust CEO Dr Michael Thurn said, “We are delighted to receive ethics approval for this Open-Label Extension study of monepantel in MND/ALS patients. It is very positive that all 12 patients who completed the Phase 1 MEND study are pleased and available to continue treatment with monepantel for a further 12 months. This study allows us to capture safety and efficacy data within a quality framework suitable for regulatory submission."