Sydney – September 13, 2026 -- Kazia Therapeutics Limited (NASDAQ: KZIA) has dosed the first patient in Arm A of PNOC035, a Phase 2 platform study testing paxalisib combined with gemcitabine for recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT), an aggressive pediatric brain cancer with no approved therapies.
Preclinical data showed the combination nearly quadrupled median survival in an AT/RT model
In an orthotopic AT/RT preclinical model, the paxalisib-gemcitabine combination extended median survival from 22 to 82.5 days (p0.0001) and showed complementary anti-tumor activity across multiple AT/RT cell lines. These findings, previously presented at ISPNO, AACR and Neuro-Oncology meetings, formed the scientific basis for advancing into the multicenter PNOC035 trial.
The trial enrolls patients ages 1 to 39 across a multi-center platform
PNOC035 is conducted by the Pediatric Neuro-Oncology Consortium (PNOC), an open-label platform study allowing multiple treatment arms within shared trial infrastructure. In Arm A, patients receive oral, once-daily paxalisib alongside intravenous gemcitabine on Days 1, 8 and 15 of each 28-day cycle. The study is registered under ClinicalTrials.gov identifier NCT07447076.
Paxalisib holds two FDA designations for AT/RT ahead of clinical proof
The FDA has granted paxalisib both Orphan Drug Designation and Rare Pediatric Disease Designation for AT/RT. Neither designation constitutes approval or endorsement of a therapeutic claim. Should paxalisib eventually receive marketing approval for this indication, Kazia could become eligible for a pediatric priority review voucher from the FDA, contingent on successful completion of clinical development and regulatory review.
Kazia's CEO frames the dosing as a step forward for an underserved patient population
Dr. John Friend II, Chief Executive Officer of Kazia Therapeutics, said families facing AT/RT diagnoses have limited treatment options despite decades of research, with no approved therapies currently available for recurrent or progressive disease. He described reaching this dosing milestone with the PNOC035 team as a meaningful step forward for enrollment progress.
The AT/RT program extends paxalisib's pediatric neuro-oncology development beyond its existing work in diffuse midline glioma, addressing a second area of significant unmet clinical need.