Kazia Therapeutics and Hebrew University: Exploring Paxalisib's Potential in Parkinson's Disease

Kazia Therapeutics, an oncology-focused drug development company, has received a research grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to explore the therapeutic potential of paxalisib as a treatment for Parkinson's disease (PD). In collaboration with The Hebrew University of Jerusalem (Hebrew University), Kazia will conduct preclinical studies to establish a link between a specific pathway in the pathophysiology of PD and paxalisib.

Paxalisib, a unique, blood-brain barrier-penetrating inhibitor for class IA phosphatidylinositol 3-kinase (PI3K), has shown promise in treating various forms of brain cancer. The grant will fund collaborative preclinical studies at Professor Ronit Sharon's lab (Hebrew University) to assess the impact of paxalisib on mouse survival, motor and non-motor performances, as well as specific biochemical, pathological, and molecular disease biomarkers in the brains of treated mice.

The collaboration between Kazia Therapeutics and Hebrew University leverages three critical advantages: first, paxalisib's unique blood-brain barrier penetration capability, which is crucial in CNS drug development; second, the preliminary data suggesting PI3K/AKT/mTORC pathway involvement in Parkinson's disease, providing a strong scientific rationale for the research; and third, the backing of The Michael J. Fox Foundation, which adds credibility and potential access to additional resources and expertise in Parkinson's disease research.

The main goal of this research is to delve into the therapeutic potential of paxalisib, a drug that can enter the brain and inhibit class IA phosphatidylinositol 3-kinase (PI3K). Preliminary findings from Professor Ronit's lab obtained in PD brains and supported by in-vivo data in a mouse model for PD collectively emphasize the role of the canonical PI3K/AKT/mTORC pathway in the pathogenesis of PD. The preclinical research from this grant builds upon earlier data obtained in PD brains, α-SynA53T tg mouse brains, and cell models for PD, which collectively emphasize a distinct role for α-Syn in hyper-activating the PI3K/AKT/mTORC2 pathway to facilitate α-Syn's activity in neuronal lipid metabolism.

The success of paxalisib in treating Parkinson's disease could have significant implications for the future of PD treatment and the market for neurodegenerative disease therapies. Early detection and intervention could lead to improved quality of life for patients and reduced healthcare costs associated with advanced stages of the disease. Targeted therapy with paxalisib could potentially reduce the need for invasive procedures like therapeutic surgery on the brain. The global Parkinson's disease therapeutics market, valued at approximately $5.3 billion, represents a significant commercial opportunity with substantial unmet medical needs. Success in PD treatment could open up new development pathways and expand the market for neurodegenerative disease therapies.

In conclusion, the collaboration between Kazia Therapeutics and Hebrew University, backed by The Michael J. Fox Foundation, holds promise for exploring the therapeutic potential of paxalisib in treating Parkinson's disease. The research findings could have a significant impact on the future of PD treatment, enabling earlier detection, targeted therapy, and market expansion. These findings also present an opportunity for Kazia Therapeutics to diversify its portfolio and explore potential partnerships in the neurodegenerative disease market.