Kazia Therapeutics (Nasdaq: KZIA) is a late-stage oncology company. Its lead program, paxalisib, is a brain-penetrant PI3K inhibitor that was licensed from Genentech. In addition to glioblastoma, for which paxalisib is in an international Phase III study, Kazia has a substantial focus on childhood brain cancer. Paxalisib is being evaluated in a multinational Phase II study in diffuse intrinsic pontine glioma (DIPG). Preclinical data supporting trials of the drug in this disease were recently presented by associate professor Matt Dun of the Hunter Medical Research Institute at the International Symposium on Pediatric Neuro-Oncology (ISPNO) 2022 Annual Conference. Paxalisib has previously been granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) for this disease.
In addition, a team from Johns Hopkins Medical School have recently presented promising preclinical data in atypical rhabdoid/teratoid tumors (AT/RT), a rare and aggressive childhood brain cancer with a five-year survival rate of around 32%. Approximately 600 people are living with the cancer in the United States, with around 60 new cases reported each year. Kazia recently received ODD in AT/RT. ODD accords seven years of market exclusivity in the United States on approval, in addition to possible grant funding and tax credits.
In this webinar, Kazia’s team and associate professor Matt Dun of the Hunter Medical Research Institute discuss the new data they presented at the ISPNO 2022 conference.