SYDNEY - Kazia Therapeutics Limited (NASDAQ: KZIA), an Australian biotechnology firm, has announced the early conclusion of a Phase I clinical trial due to positive safety and clinical response findings. The study investigated the combination of paxalisib, an oral PI3K/mTOR dual inhibitor, with radiation therapy in treating brain metastases with PI3K pathway mutations from solid tumors.
The trial's first part established the maximal tolerated dose of paxalisib combined with radiation therapy, demonstrating promising clinical activity in nine evaluable patients. The second part, an expansion cohort, aimed to further assess the safety and efficacy of the treatment in up to 12 additional patients. The investigators have concluded that the primary endpoint of the study has been met, with continued encouraging clinical responses observed in the expansion cohort.
Dr. John Friend, CEO of Kazia, expressed optimism about the trial's findings and announced plans to engage with the FDA for guidance on a pivotal registration study, with the goal of advancing paxalisib's development.
Activation of the PI3K pathway, common in brain metastases, has been linked to tumor resistance to radiotherapy. This has underpinned the rationale for combining paxalisib with radiotherapy to potentially improve disease control. The detailed findings from the second part of the study are to be submitted for presentation at a forthcoming global scientific meeting.
Last year, paxalisib received Fast Track Designation from the FDA for the treatment of solid tumor brain metastases with PI3K pathway mutations in combination with radiation therapy. With approximately 200,000 cancer patients in the U.S. developing brain metastases annually, and the variable efficacy of current radiotherapy treatments, there is a significant need for new therapeutic options.
Kazia is also developing EVT801, a VEGFR3 inhibitor, with preliminary data expected in CY2024. Paxalisib continues to be evaluated in multiple clinical trials for various forms of brain cancer.
This report is based on a press release statement from Kazia Therapeutics Limited.
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