Paxalisib is under clinical development by Kazia Therapeutics and currently in Phase III for Glioblastoma Multiforme (GBM). According to GlobalData, Phase III drugs for Glioblastoma Multiforme (GBM) have a 30% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Paxalisib’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Paxalisib overview

Paxalisib is under development for the treatment of high-grade glioblastoma or glioma, breast cancer metastases to brain, pediatric diffuse intrinsic pontine glioma, solid tumors with brain metastases and leptomeningeal metastases, primary CNS lymphoma and anaplastic astrocytoma, rhabdoid/teratoid tumors (AT/RT) and metastatic melanoma. It is a small molecule administered orally as a capsule. It acts by targeting the phosphoinositide 3-kinase and mTOR. The drug candidate was under development for the treatment of patients recurrent high-grade glioblastoma or glioma.

Kazia Therapeutics overview

Kazia Therapeutics (Kazia) is a clinical stage biotechnology company which focuses on the discovery and development of anti-cancer drugs. The company’s product pipeline includes paxalisib (formerly GDC-0084) and Cantrixil (TRX-E-002-1). Paxalisib is an inhibitor of PI3K /AKT /mTOR pathway to treat glioblastoma multiforme It is in phase II clinical trials. TRX-E-002-1 (Cantrixil) is a third generation benzopyran molecule that acts against cancer stem cells. Cantrixil is in phase I clinical trials for the treatment of ovarian cancer. The company has collaborations with various research institutes, hospitals, and universities including St Jude Children’s Hospital, University of Newcastle, Dana Farber Cancer Institute and Memorial Sloan Kettering Cancer Centre to develop its pipeline. Kazia is headquartered in Sydney, New South Wales, Australia.

For a complete picture of Paxalisib’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.