Kazia Therapeutics Limited

Kazia Therapeutics, an oncology-focused drug development company, leads with paxalisib, an investigational brain-penetrant inhibitor targeting the PI3K/Akt/mTOR pathway, aimed at addressing various forms of brain cancer. Acquired from Genentech in late 2016, paxalisib has been the focus of ten clinical trials in this domain. Notably, a Phase 2 study in glioblastoma unveiled promising clinical activity in 2021. Presently, a pivotal study, GBM AGILE, continues, with anticipated final data in CY2023. Concurrently, clinical trials in brain metastases, diffuse midline gliomas, and primary CNS lymphoma are underway, several of which have reported encouraging interim results.

Paxalisib has garnered significant regulatory attention, securing Orphan Drug Designation for glioblastoma in February 2018, followed by Fast Track Designation (FTD) for glioblastoma in August 2020. Further, FTD was granted in July 2023 for solid tumour brain metastases harboring PI3K pathway mutations, in combination with radiation therapy. Moreover, it received Rare Pediatric Disease Designation and Orphan Drug Designation for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid/rhabdoid tumours (AT/RT) in June and July 2022, respectively.

Beyond paxalisib, Kazia is advancing EVT801, a small-molecule VEGFR3 inhibitor, acquired from Evotec SE in April 2021. Preclinical assessments have demonstrated EVT801’s efficacy across various tumour types, suggesting synergy with immuno-oncology agents. In November 2021, a Phase I study commenced recruitment, marking progress in its development.

Kazia was removed from the ASX at the close of trading on Wednesday, 15 November 2023.