Kazia announces early conclusion of two-part Phase I trial evaluating paxalisib

Kazia Therapeutics announced the early conclusion based on positive safety and promising clinical response findings observed to date of an important two-part Phase I trial. This investigator-initiated trial evaluated the use of paxalisib, an oral PI3K/mTOR dual inhibitor, with radiation therapy for the treatment of patients with PI3K pathway mutation brain metastases from solid tumors. Part I of the study established the maximal tolerated dose (MTD) of paxalisib in combination with radiation therapy, while also demonstrating promising signs of clinical activity in all nine evaluable patients. Part II was a follow-on expansion cohort to further evaluate safety and efficacy of the MTD (45mg daily) combined with radiation therapy in up to 12 additional patients. After reviewing the Part II patient data generated to date, the three lead investigators have determined that the primary endpoint of the study has been reached. Last year, paxalisib was awarded Fast Track Designation on the basis of the Part I clinical data by the FDA for the treatment of solid tumor brain metastases harboring PI3K pathway mutations in combination with radiation therapy. Approximately 200,000 cancer patients develop brain metastases in the U.S. each year.