PharmAust receives approval for open-label MND extension study

PharmAust Limited (ASX: $PAA), a clinical-stage biotechnology company, has obtained approval from Macquarie University 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 Macquarie University, Sydney. The study aims to collect ongoing data from the 12 patients who completed the Phase 1 MEND study, with the primary objective being to assess the long-term safety and tolerability of MPL.

PharmAust CEO's commentary on the approval

We are delighted to receive ethics approval from Macquarie University HREC and excited to now be able to offer all Phase 1 MEND patients the opportunity to enrol in the Open-Label Extension study. It's incredibly rewarding to know that 9 patients have already been enrolled at Calvary Health Care Bethlehem and the remaining 3 patients can now continue receiving benefit from treatment with monepantel. We are very thankful to the patients and our investigators, Associate Professor Susan Mathers and Professor Dominic Rowe, for their ongoing involvement as we continue working towards a much-needed new therapy for MND/ALS.

Summary of PharmAust's open-label MND extension study approval

PharmAust Limited has received final ethics committee approval to conduct an Open-Label Extension (OLE) study of monepantel (MPL) in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS) at Macquarie University, Sydney. The study aims to assess the long-term safety and tolerability of MPL, as well as the biomarkers and efficacy endpoints related to the disease. The company's CEO, Dr Michael Thurn, expressed delight at the approval and gratitude towards the patients and investigators involved. PharmAust's focus on developing therapeutics for neurodegenerative diseases aligns with the growing market for such treatments, with positive top-line results for its Phase 1 study in patients with MND/ALS paving the way for a pivotal registration adaptive Phase 2/3 clinical study in H2 CY 2024. The company anticipates accelerated approval with the US Food and Drug Administration in 2026, contributing to the expanding Neurodegenerative Disease Market.