Kazia Therapeutics begins clinical study for pediatric brain cancer

Kazia Therapeutics Limited has commenced a new clinical study, designated PNOC035, to assess the efficacy of paxalisib in combination with gemcitabine for pediatric and young adult patients diagnosed with recurrent or progressive atypical teratoid/rhabdoid tumors (AT/RT).

AT/RT is a rare and aggressive form of brain cancer that primarily affects young children. Currently, there are no approved therapies for this condition, and treatment outcomes are often poor. This study aims to address the unmet medical need in this patient population.

The multicenter study has dosed its first patient, marking a significant step in the evaluation of this potential new treatment. The trial will investigate the safety and efficacy of the combination therapy in individuals for whom standard treatment options have been exhausted.

Paxalisib is an orally available small molecule inhibitor of the PI3K pathway, developed by Kazia Therapeutics. The company is pursuing its development for various oncology indications, with this study focusing on a critical pediatric cancer with limited therapeutic alternatives.