Kazia Therapeutics and the Hebrew University of Jerusalem Receives a Grant from The Michael J. Fox Foundation to Evaluate the Therapeutic Potential of Paxalisib as a Treatment for Parkinson's Disease

Core Insights - Kazia Therapeutics Limited has received a research grant from The Michael J. Fox Foundation for Parkinson's Research to investigate the therapeutic potential of paxalisib for Parkinson's disease [1][2] - The research will be conducted in collaboration with The Hebrew University of Jerusalem, focusing on preclinical studies to establish a link between a specific pathway in Parkinson's disease and paxalisib [2][3] - Paxalisib is a blood-brain barrier-penetrating inhibitor targeting class IA phosphatidylinositol 3-kinase (PI3K), with the aim of addressing the underlying pathophysiology of Parkinson's disease [2][3] Company Overview - Kazia Therapeutics is an oncology-focused drug development company based in Sydney, Australia, with paxalisib as its lead program [4] - Paxalisib has been involved in ten clinical trials for various brain cancers, including a completed Phase 2/3 study in glioblastoma reported in 2024 [4] - The company has received multiple designations from the FDA for paxalisib, including Orphan Drug Designation and Fast Track Designation for glioblastoma and other conditions [4] Research Objectives - The primary goal of the research is to explore the therapeutic potential of paxalisib in treating Parkinson's disease by inhibiting the AKT phosphorylation reaction of α-SynA53T [3] - Preliminary findings from Professor Ronit's lab suggest a significant role of the PI3K/AKT/mTORC pathway in the pathogenesis of Parkinson's disease [3] - The research will assess the impact of paxalisib on mouse survival, motor and non-motor performances, and various disease biomarkers [2][3]