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Kazia Therapeutics

An Australia-founded oncology drug developer with operations in Sydney and the United States. The company runs global clinical development through external clinical sites and specialist investigator networks, with a primary emphasis on CNS oncology.

Founding and History

Kazia is publicly traded on Nasdaq (KZIA). The company’s strategy has been built around in-licensing and developing targeted small molecules for hard-to-treat cancers, led by its brain-penetrant PI3K/Akt/mTOR pathway inhibitor paxalisib (licensed from Genentech in 2016). In 2024–2025, Kazia repositioned its glioblastoma program around results generated in the GBM AGILE platform trial and subsequent FDA interactions, while expanding paxalisib into combination studies beyond CNS disease.

Therapy Areas and Focus

Kazia focuses on oncology, with priorities in:

Primary brain tumors, particularly glioblastoma
Hard-to-treat solid tumors where pathway inhibition may enhance immunotherapy response
The company has also supported exploratory research outside oncology where paxalisib’s mechanism may be relevant (for example, preclinical work in Parkinson’s disease).

Technology Platforms and Modalities

Kazia is a small-molecule oncology company with two principal assets:

Paxalisib (oral, brain-penetrant PI3K/Akt/mTOR inhibitor)
Designed to achieve therapeutic exposure in the brain, supporting development in glioblastoma and other CNS-involved cancers. The program has been evaluated in a Phase II/III setting through GBM AGILE and is being developed in additional clinical settings, including combination immuno-oncology in metastatic triple-negative breast cancer.

EVT801 (selective VEGFR3 inhibitor)
A small molecule targeting tumor-associated lymphangiogenesis and aspects of angiogenesis biology. EVT801 has completed Phase I evaluation and is positioned for follow-on development in selected solid tumors, including settings where combination with immunotherapy may be rational.

Strategic Partnerships

Kazia’s model combines in-licensed assets with external development and manufacturing partners. The EVT801 program is associated with Evotec as a development partner. Kazia has also relied on investigator-sponsored and collaborative frameworks (for example GBM AGILE) to generate clinically relevant datasets efficiently.

FAQ Section

Kazia develops targeted small-molecule therapies for cancers with high unmet need, with a particular emphasis on CNS oncology. The company’s core value proposition is brain-penetrant pathway inhibition (paxalisib) alongside a second oncology asset targeting VEGFR3 biology (EVT801).

The lead focus is glioblastoma. Kazia also evaluates paxalisib in other solid tumors, including immuno-oncology combinations, where PI3K-pathway modulation may improve anti-tumor immune response.

Key disclosed clinical programs include:

Paxalisib in glioblastoma: supported by GBM AGILE Phase II/III experience and subsequent planning for a registrational pathway (Phase III design discussions and FDA engagement disclosed in 2025)
Paxalisib in metastatic triple-negative breast cancer: Phase I b combination study with pembrolizumab and chemotherapy (ongoing)
EVT801: Phase I completed; preparing for next-stage studies based on tolerability and early activity signals

Recent updates include:

January 2026: a clinical update from the ongoing Phase I b study of paxalisib in metastatic triple-negative breast cancer, including preliminary responses in treated patients
November 2025: a company business update highlighting an immune-complete response reported in the TNBC combination study and outlining a continued FDA engagement strategy for glioblastoma
May 2025: disclosure of FDA alignment on key elements of a proposed registrational Phase III study design for paxalisib in glioblastoma
July 2024: reporting of GBM AGILE Phase II/III results for paxalisib in glioblastoma, including outcomes in prespecified subgroups

Kazia’s most substantive dataset is from GBM AGILE (Phase II/III), where paxalisib was evaluated against standard-of-care in glioblastoma, with the company emphasizing survival findings in defined patient subsets. In TNBC, disclosures have been interim and exploratory, focused on early clinical response signals in a combination regimen.

Near-term milestones are driven by:

Continued FDA engagement on a potential registrational strategy for paxalisib in glioblastoma (including meeting outcomes and confirmatory study requirements)
Further maturing of the Phase I b TNBC combination dataset (dose refinement, expansion, and response durability)
Definition of next-stage clinical development plans for EVT801 following Phase I completion

Kazia positions paxalisib around CNS exposure and feasibility for brain tumor development, addressing a common limitation of many pathway inhibitors. The company also frames combination development (notably with checkpoint blockade) as a route to broaden utility beyond CNS tumors where tumor-intrinsic signaling intersects with immune suppression.

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