Paxalisib is a highly differentiated, brain-penetrant oral PI3K/mTOR inhibitor with origins in neuro-oncology. The program has advanced across multiple solid tumor indications, demonstrating our commitment to expanding our impact in cancer therapy.
| Study / Indication | Trial ID | Preclinical | Phase 1 | Phase 2 | Phase 3 | Market | Sponsor |
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| Breast Cancer | |||||||
| PaxPlus-ABC TNBC: Triple-Negative Breast Cancer & HER2- | ACTRN12624001340527 |
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Kazia Therapeutics |
| Neuro-Oncology | |||||||
| GBM AGILE Glioblastoma | NCT03970447 |
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Global Coalition for Adaptive Research (GCAR) |
| 5G-PEARL Glioblastoma | NCT07391215 |
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Institute of Cancer Research (UK) |
| Paxalisib & Diet Combination Glioblastoma | NCT05183204 |
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Weill Medical College of Cornell University |
| LUMOS2 Gliomas | ACTRN12623000096651 |
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University of Sydney |
| OPTIMISE Pediatric Brain Cancer | NCT06208657 |
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University of Sydney |
| PNOC022 DIPG: Diffuse Intrinsic Pontine Glioma | NCT05009992 |
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Pediatric Neuro-Oncology Consortium (PNOC) |
| PNOC035 AT/RT: Atypical Teratoid Rhabdoid Tumor | NCT07447076 |
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Pediatric Neuro-Oncology Consortium (PNOC) |
| Genomically-Guided Brain Metastases Brain Metastases | NCT03994796 |
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Alliance for Clinical Trials in Oncology |
The PI3K/mTOR pathway is one of the most frequently dysregulated signaling networks in cancer, playing a critical role in tumor cell growth, proliferation, survival, metabolism, and treatment resistance. Dual inhibition of PI3K and mTOR provides comprehensive blockade of this pathway, disrupting key oncogenic signals that drive cancer progression and disease maintenance.
Beyond its established role in pathway inhibition, emerging research has demonstrated that paxalisib can influence downstream transcriptional, epigenetic, and immune regulatory processes that shape tumor behavior. At de-escalated doses, this approach has the potential to modulate cancer cell behavior associated with immune evasion, metastatic progression, and therapeutic resistance.
This concept forms the foundation of our functional epigenetic reprogramming strategy, which seeks to leverage selective modulation of disease-driving biology to restore immune responsiveness, alter tumor cell behavior, and improve treatment outcomes. We believe this differentiated mechanism represents an important evolution beyond traditional kinase inhibition and may create new opportunities across multiple cancer types.
Explore our posters and publications to access our latest scientific data, clinical insights, and contributions to advancing oncology research.
View Our Posters & Publications