Kazia Therapeutics Announces First Patient Dosed in PNOC035 Clinical Study Evaluating Paxalisib for Recurrent Pediatric AT/RT Brain Cancer

Kazia Therapeutics (KZIA) announced that Sovargen Co. dosed the first patient in a Phase 1b/2a trial of paxalisib for rare seizure disorders. The milestone triggers a $2 million payment to Kazia, with potential for up to $17 million more. Paxalisib is being developed for intractable epilepsy associated with FCD T2 and TSC, with Kazia retaining oncology focus.

Kazia Therapeutics (KZIA) dosed the first patient in a Phase 2 study of paxalisib combined with gemcitabine for pediatric brain cancer. The trial, conducted by the Pediatric Neuro-Oncology Consortium, follows preclinical data showing increased survival rates. Paxalisib has FDA designations for this indication.

Kazia Therapeutics (KZIA) reported preclinical data showing its lead asset, paxalisib, reduced tumor burden in immunotherapy-resistant colorectal cancer by 52% as a monotherapy and by an additional 50% when combined with Pembrolizumab. The treatment was well tolerated, and the company plans to advance paxalisib into a Phase 2 clinical study. KZIA's stock closed at $13.77, up 1.40%, and was at $13.90 in pre-market trading.

Kazia Therapeutics (KZIA) reported paxalisib reduced tumor burden by 52% in a colorectal cancer model. The company plans a Phase 2 trial in 2027, evaluating paxalisib alone and with immunotherapy. Kazia is developing biomarkers and has filed a patent for colorectal cancer applications.

Kazia Therapeutics reported preclinical data showing paxalisib's activity in HR+/HER2- breast cancer models. The company will expand its clinical trial to evaluate paxalisib combinations in metastatic breast cancer, with patient enrollment expected to begin before the end of 2026. Kazia filed a provisional patent related to the program and expects full enrollment by the end of 2027, with a complete data readout in 2028.

BTIG raised its price target for Kazia Therapeutics (NASDAQ:KZIA) to $40 from $34, citing positive trial data for paxalisib in metastatic triple-negative breast cancer. The stock has surged nearly 100% year-to-date to $12.71. Kazia also announced a $40 million public offering. BTIG noted the data showed an 83% overall response rate and no serious adverse events.