On June 5, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or the "Company"), an oncology-focused biotechnology company developing innovative therapies for difficult-to-treat cancers, reported that the first patient has been dosed in a Phase 1b clinical trial sponsored by Kazia (Press release, Kazia Therapeutics, JUN 5, 2025, View Source [SID1234653739]). The study evaluates paxalisib, the Company’s dual PI3K/mTOR inhibitor, in combination with olaparib or pembrolizumab for patients with advanced breast cancer.
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This multi-center, open-label, randomized trial is designed to assess the safety, tolerability, and preliminary efficacy of multiple paxalisib-based treatment combinations. The study also includes deep biomarker profiling to support future development and early signals of clinical activity.
"The start of patient dosing in this Kazia-sponsored study marks an important milestone in the evolution of paxalisib beyond brain cancer and into broader solid tumor applications," said Dr. John Friend, Chief Executive Officer of Kazia. "By leveraging the dual inhibition of PI3K and mTOR, this trial builds on compelling preclinical data showing epigenetic modulation in aggressive breast cancer pre-clinical models. We believe the combinations explored here may provide a more effective therapeutic strategy by simultaneously targeting tumor metabolism, DNA repair, and immune evasion."
About the Study
This Phase 1b trial (ACTRNXXX) will enroll patients with advanced breast cancer into two treatment arms:
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Arm A: Patients are randomized to receive either 15mg or 30mg of paxalisib (once daily) in combination with olaparib (300mg orally, twice daily), administered in 28-day cycles.
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Arm B: Patients are randomized to receive paxalisib (15mg or 30mg once daily) and pembrolizumab (200mg IV every 21 days) alongside standard-of-care chemotherapy: either nanoparticle albumin-bound paclitaxel or a gemcitabine-carboplatin regimen, depending on clinical indication.
Participants will be evaluated for:
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Adverse events and overall tolerability
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Changes in circulating tumor cells ("CTCs") and CTC clusters
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Immune cell signatures and overall immune functio
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Clinical activity and tumor response signals
Strategic Significance
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For investors, this trial expands the clinical footprint of paxalisib into solid tumors beyond the central nervous system, targeting a significant commercial opportunity in advanced and metastatic breast cancers, including triple-negative breast cancer.
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For potential partners and acquirers, the novel biomarker-driven design provides an early window into how paxalisib may enhance or sensitize tumors to immune checkpoint inhibitors and DNA repair-targeted therapy.
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For the scientific community, the study is structured to generate translational data that may clarify the mechanistic interactions between dual PI3K/mTOR inhibition, immune modulation, and chemotherapy-induced cytotoxicity.
"The integration of paxalisib into combination regimens reflects our strategy of building value through differentiated science and high-quality collaborations," added Dr. Friend. "As this study progresses, we aim to share interim updates that may further underscore the potential of paxalisib to impact multiple indications with poor prognoses."