KAZIA THERAPEUTICS LTD

Kazia Therapeutics Limited is an emerging oncology-focused biotechnology company primarily engaged in pharmaceutical drug development. The company has a portfolio of development candidates across several distinct technologies, aiming to produce new and improved treatments for various oncology indications. Kazia's main program is Paxalisib, an investigational drug designed to treat brain cancer. Paxalisib, previously known as GDC-0084, is a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway. Kazia obtained a worldwide exclusive license for Paxalisib in October 2016 from Genentech, Inc. Genentech had completed extensive pre-clinical development and a phase I clinical trial in 47 patients with advanced recurrent grade III and grade IV glioma (NCT01547546). In this trial, the most common adverse events were oral mucositis and hyperglycemia. According to ANO criteria, 40% of patients showed a best observable response of stable disease, and 26% demonstrated a metabolic partial response on FDG-PET. The World Health Organisation granted Paxalisib the International Non-Proprietary Name (INN) in December 2019, and the USAN Council confirmed it as the United States Adopted Name ("USAN") in April 2020. Paxalisib is administered orally as a 15mg capsule. It is currently under IND 112,608 with the U.S. Food and Drug Administration ("FDA"). Paxalisib is a potent and selective inhibitor of all four isoforms of phosphoinositide-3-kinase (PI3K) and a moderate inhibitor of the mammalian target of rapamycin ("mTOR"). The PI3K / Akt / mTOR signaling axis is dysregulated in approximately 85-90% of glioblastoma cases, according to the Cancer Genome Atlas. Five PI3K inhibitors have been approved by the FDA for various hematological malignancies and solid tumors.