PharmAust Information

PharmAust Limited is listed on the Australian Securities Exchange (ASX Code: PAA). PAA is a clinical-stage biotechnology company developing therapeutics for human health applications. The company is focused on repurposing monepantel (MPL) for human neurodegenerative diseases. MPL is a potent and safe inhibitor of the mTOR pathway. This pathway plays a central role in degenerating neurons. The mTOR pathway regulates the cellular “cleaning process”, where toxic protein is broken down into macromolecules to be reused. This autophagic process is disrupted in most neurodegenerative diseases, including motor neurone disease (MND/ALS). PAA’s lead MPL program is for the treatment of MND/ALS, a rare, incurable disease. PAA has announced positive top-line results from a Phase 1 study in MND/ALS, and anticipates starting an adaptive Phase 2/3 clinical study in H2 CY 2024 that could lead to accelerated approval with the US Food and Drug Administration in 2026.