Kazia to Present DIPG Data at ISPNO and Publish in European Journal of Cancer

Kazia Therapeutics Limited, a biotechnology firm focused on oncology and listed on NASDAQ under the ticker KZIA, has announced new data from its leading program, paxalisib, that will be presented at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024). The symposium is scheduled to take place from June 29 to July 2, 2024, in Philadelphia, PA. Additionally, an article highlighting the necessity of evaluating mutation-specific, CNS-penetrant inhibitors for pediatric patients with Diffuse Midline Glioma (DMG) has been published in the European Journal of Cancer.

During ISPNO 2024, there will be three presentations related to paxalisib. One of the key presentations will focus on data from the Phase 2 PNOC DMG-ACT study, which examines the safety and efficacy of paxalisib in combination with ONC201. Researchers will discuss survival data, pharmacokinetics, and tumor biomarkers from 132 patients with diffuse midline glioma who participated in the study. The study is divided into three cohorts: Cohort 1 includes patients newly diagnosed and enrolled before radiation therapy (n=33), Cohort 2 includes those newly diagnosed and enrolled post-radiation therapy (n=69), and Cohort 3 includes patients with relapsed disease, enrolled after progression (n=30). The median overall survival reported was 13.2 months for Cohort 1, 15.8 months for Cohort 2, and 8.8 months for Cohort 3.

The second presentation will showcase preclinical data involving the addition of a novel HDAC inhibitor to paxalisib in DMG models. The third presentation, led by researchers from Johns Hopkins University, will discuss preclinical data on the combination therapy of paxalisib and gemcitabine for patients with relapsed or recurrent atypical teratoid/rhabdoid tumors (AT/RT). Based on these promising findings, the Pacific Pediatric Neuro-Oncology Consortium plans to incorporate this combination therapy into its upcoming international clinical trial for AT/RT.

The European Journal of Cancer publication, arising from a two-day forum held at Dana Farber Cancer Institute in April 2023, underscores the importance of evaluating mutation-specific, CNS-penetrant PI3-K inhibitors in treating children with DMG. The forum included patient advocates, regulators, researchers, and pediatric clinicians, and it concluded that innovative regulatory approaches are required due to the rarity of the patient population.

Kazia Therapeutics Limited, based in Sydney, Australia, is known for its lead program, paxalisib, an investigational inhibitor of the PI3K/Akt/mTOR pathway designed to penetrate the brain. Licensed from Genentech in late 2016, paxalisib has been involved in ten clinical trials focusing on various forms of brain cancer. A completed Phase 2 study in glioblastoma showed early signs of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, has recently completed enrollment with final data expected soon. Other ongoing clinical trials are exploring its efficacy in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several reporting encouraging interim results. The FDA has granted paxalisib several designations, including Rare Pediatric Disease Designation and Orphan Drug Designation for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid/rhabdoid tumors in June 2022 and July 2022.

In summary, Kazia Therapeutics is making significant strides in the development of paxalisib for various pediatric brain cancers, with promising new data to be presented at ISPNO 2024 and an important publication in the European Journal of Cancer, emphasizing the need for targeted CNS-penetrant therapies in pediatric oncology.