Kazia Therapeutics Begins First Patient Dosing in PNOC035 Study for AT/RT

Kazia Therapeutics, an oncology-focused biotechnology company, announced the dosing of the first patient in its Phase 2 PNOC035 clinical study. The trial evaluates paxalisib in combination with gemcitabine for children and young adults with recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT), a highly aggressive form of pediatric brain cancer.

PNOC035 is a multicenter, open-label, multi-center platform study conducted by the Pediatric Neuro-Oncology Consortium (PNOC). In Arm A of the study, patients aged 1 to 39 will receive oral, once-daily paxalisib in combination with intravenous gemcitabine on Days 1, 8, and 15 of each 28-day treatment cycle. This combination therapy has shown significant promise in preclinical models, with a nearly four-fold increase in median survival time from 22 to 82.5 days.

"AT/RT typically affects infants and very young children, and families facing this diagnosis have limited treatment options," said Dr. John Friend II, CEO of Kazia Therapeutics. "Despite decades of research, there are still no approved therapies for recurrent or progressive AT/RT. This trial represents a meaningful step forward and underscores our commitment to developing new treatment options for this devastating disease."

Paxalisib, the investigational brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway, 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 guarantee approval but can provide potential benefits if the drug receives marketing clearance.

The study is part of Kazia's ongoing efforts to address the significant unmet need in pediatric brain cancer. "Reaching this milestone with the PNOC035 team is a significant achievement," Friend II added. "We look forward to working closely with PNOC as enrollment progresses and hope to bring new hope to patients and families affected by this disease."

The Phase 2 platform trial, PNOC035, is being conducted by the Pediatric Neuro-Oncology Consortium for patients aged 1 to 39 with recurrent or progressive AT/RT. Additional information about the study is available on ClinicalTrials.gov (NCT07447076).

Kazia Therapeutics, based in Sydney, Australia, is developing multiple clinical-stage programs, including paxalisib, which has shown promising results across various cancer types. The company's other ongoing trials include advanced breast cancer, brain metastases, diffuse midline gliomas, and primary central nervous system lymphoma.