Multicenter study to evaluate paxalisib plus gemcitabine for children and young adults with recurrent or progressive AT/RT, where no approved therapies currently exist

Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or the "Company"), an oncology-focused biotechnology company developing therapies that selectively reprogram cancer biology, restore anti-tumor immunity and overcome treatment resistance, today announced that the first patient has been dosed in Arm A of PNOC035, a Phase 2 platform study evaluating paxalisib in combination with gemcitabine for patients ages 1 to 39 with recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT). AT/RT is widely recognized as one of the most aggressive forms of pediatric brain cancer.

PNOC035 is conducted by the Pediatric Neuro-Oncology Consortium (PNOC), an open-label, multi-center platform study. In Arm A, patients receive oral, once-daily paxalisib in combination with intravenous gemcitabine on Days 1, 8 and 15 of each 28-day treatment cycle.

The combination increased median survival nearly four-fold (22 to 82.5 days; p<0.0001) in an orthotopic AT/RT preclinical model and demonstrated complementary anti-tumor activity across multiple AT/RT cell lines. These preclinical findings provided the rationale for advancement into the multicenter PNOC035 clinical study evaluating paxalisib plus gemcitabine in children and young adults with recurrent or progressive AT/RT. These findings, previously presented at ISPNO, AACR and Neuro-Oncology meetings, provided the scientific rationale for PNOC035 and extend paxalisib's pediatric neuro-oncology development beyond diffuse midline glioma into another area of significant unmet need.

Paxalisib has been granted both Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for AT/RT. These designations do not constitute FDA approval or endorsement of any therapeutic claim. If paxalisib were to receive marketing approval for this indication, which would require successful completion of clinical development and FDA regulatory approval, Kazia could become eligible to receive a pediatric priority review voucher from the FDA.

"AT/RT typically affects infants and very young children, and families facing this devastating diagnosis have limited treatment options," said Dr. John Friend II, Chief Executive Officer of Kazia Therapeutics. "Despite decades of research, there are still no approved therapies for recurrent or progressive AT/RT, underscoring the urgent need for new treatment options. We've remained committed to pediatric brain cancer because this is an area where the need is especially great and the options are often fewest. Reaching this milestone with the PNOC035 team is a meaningful step forward, and we look forward to working closely with PNOC as enrollment progresses."