Kazia Therapeutics doses first patient in PNOC035 Phase 2 paxalisib-gemcitabine trial for recurrent pediatric AT/RT
KZIA•Regulatory status for paxalisib
Paxalisib holds FDA Orphan Drug status and Rare Pediatric Disease designation for AT/RT, positioning the program for a priority review voucher if approved.
First patient dosed in PNOC035 Arm A
Kazia Therapeutics said it has started dosing patients in PNOC035 Arm A, a Phase 2 study of paxalisib with gemcitabine in recurrent atypical teratoid/rhabdoid tumor (AT/RT).
The trial targets patients ages 1 to 39, addressing a setting with no approved therapies, and expands paxalisib beyond diffuse midline glioma.
Preclinical support for the combination
Kazia said earlier preclinical findings supporting the combination had already been presented at ISPNO, AACR, and Neuro-Oncology meetings.
Preclinical work showed the regimen extended survival in models, supporting a potential improvement in outcomes if replicated clinically.
