Oncology-focused biotechnology company Kazia Therapeutics Limited (NASDAQ: KZIA) declared on Monday that the first patient has been dosed in the Arm A of PNOC035, a Phase 2 platform study evaluating paxalisib plus gemcitabine in patients aged 1 to 39 with recurrent or progressive atypical teratoid/rhabdoid tumour (AT/RT).

The multicentre study, conducted by the Pediatric Neuro-Oncology Consortium, will assess once-daily oral paxalisib combined with intravenous gemcitabine in 28-day treatment cycles. Preclinical testing showed the combination increased median survival nearly four-fold in an orthotopic AT/RT model.

Paxalisib has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for AT/RT. The designations do not constitute regulatory approval or endorsement of the treatment.

Kazia Therapeutics is developing paxalisib across multiple cancer indications, alongside EVT801, an investigational VEGFR3 inhibitor, and preclinical programmes targeting immunotherapy resistance.