Kazia Therapeutics Achieves Significant Breakthrough in HER2-Positive Breast Cancer Treatment with Paxalisib

Kazia Therapeutics Achieves Milestone in Cancer Therapy

Kazia Therapeutics Limited, a company specialized in drug development for oncology, recently shared groundbreaking results from its collaborative research led by Professor Sudha Rao at QIMR Berghofer. The findings emphasize the medicinal impact of their investigational drug, Paxalisib, particularly among patients suffering from Stage IV HER2-positive metastatic breast cancer (mBC).

The Challenge of HER2-Positive Breast Cancer

HER2-positive breast cancer represents approximately 15-20% of all breast cancer cases. It poses significant treatment challenges, even with advancements in HER2-targeted therapies. Despite such innovations, many patients continue to experience resistance, recurrence, or metastasis, highlighting the urgent need for novel therapeutic strategies, especially considering the limited effectiveness of existing immunotherapies for this type of cancer.

In a recent ex vivo study, blood samples of patients with Stage IV HER2-positive mBC were analyzed. Researchers focused on understanding how Paxalisib, a PI3K-mTOR inhibitor, could affect the metastatic burden of the disease. The results were nothing short of promising; it disclosed a statistically significant drop in isolated circulating tumor cells (CTCs), achieving complete disruption (100%) of CTC clusters that contained three or more cells, which are recognized as indicators of an aggressive form of cancer and its potential spread.

A Promising Future with Paxalisib

Dr. John Friend, CEO of Kazia Therapeutics, emphasized the importance of these findings during the announcement, stating, "This monotherapy ex-vivo result extends our understanding of Paxalisib's potential beyond triple-negative breast cancer into HER2-positive disease. The ability to disrupt circulating tumor cell clusters [...] represents a transformative therapeutic avenue."

Kazia's findings not only expand the potential applications of Paxalisib but also align with precision medicine principles where biomarkers could be utilized to gauge the progression of the disease and inform subsequent treatment decisions. This highlights Kazia's dedication to meeting the high unmet needs of patients grappling with advanced breast cancer, who often find themselves with few options.

Moreover, this recent advancement complements the ongoing Phase 1b trial targeting Stage IV triple-negative breast cancer (TNBC). The preliminary data released earlier in July 2025 illustrated significant reductions in CTCs and clusters, reinforcing the broader prospects for Paxalisib in the fight against metastatic breast cancer.

Future Prospects

In light of these developments, comprehensive datasets outlining metastatic signatures in the context of disrupted progenitor populations of HER2-positive breast cancer are slated for presentation at a global oncology meeting in 2025. The medical community eagerly awaits this information, which holds the potential for reshaping treatment paradigms in cancer therapy.

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About Kazia Therapeutics

Founded in Sydney, Australia, Kazia Therapeutics (NASDAQ: KZIA) is driven by a mission to revolutionize cancer treatment. Their flagship program features Paxalisib, a brain-penetrant PI3K/Akt/mTOR pathway inhibitor undergoing multiple clinical trials across various malignancies. With a portfolio that also includes promising candidates like EVT801, Kazia is at the forefront of creating innovative cancer therapies that aim to address significant gaps in current treatment regimens.

As these advancements unfold, Kazia Therapeutics continues to commit to unearthing new possibilities in oncology, with the ultimate goal of enhancing treatment options for patients worldwide.