Pipeline

A Pipeline that Harnesses Next-Generation Science

Kazia is a biotechnology company specializing in oncology innovation. With a diverse portfolio of development candidates, we are committed to advancing novel cancer therapies designed to deliver first-in-class and best-in-class outcomes across multiple oncology indications.

Our Diversified Pipeline

Targeting Cancer's Core Control Layers

Our pipeline spans multiple oncology candidates designed to target diverse cancer types and mechanisms.

Paxalisib

Study / Indication	Trial ID	Preclinical	Phase 1	Phase 2	Sponsor
Breast Cancer
PaxPlus-ABC TNBC: Triple-Negative Breast Cancer & HER2-	ACTRN12624001340527	Preclinical	Phase 1		Kazia Therapeutics
Neuro-Oncology
GBM AGILE Glioblastoma	NCT03970447	Preclinical	Phase 1	Phase 2	Global Coalition for Adaptive Research (GCAR)
5G-PEARL Glioblastoma	NCT07391215	Preclinical	Phase 1	Phase 2	Institute of Cancer Research (UK)
Paxalisib & Diet Combination Glioblastoma	NCT05183204	Preclinical	Phase 1	Phase 2	Weill Medical College of Cornell University
LUMOS2 Gliomas	ACTRN12623000096651	Preclinical	Phase 1	Phase 2, 50% complete	University of Sydney
OPTIMISE Pediatric Brain Cancer	NCT06208657	Preclinical	Phase 1	Phase 2, 50% complete	University of Sydney
PNOC022 DIPG: Diffuse Intrinsic Pontine Glioma	NCT05009992	Preclinical	Phase 1	Phase 2	Pediatric Neuro-Oncology Consortium (PNOC)
PNOC035 AT/RT: Atypical Teratoid Rhabdoid Tumor	NCT07447076	Preclinical	Phase 1	Phase 2, 15% complete	Pediatric Neuro-Oncology Consortium (PNOC)
Genomically-Guided Brain Metastases Brain Metastases	NCT03994796	Preclinical	Phase 1	Phase 2	Alliance for Clinical Trials in Oncology

NDL2

Study / Indication	Trial ID	Preclinical	Phase 1	Phase 2	Phase 3	Market	Sponsor
Advanced Solid Tumors		Preclinical					Kazia Therapeutics

MSETC

Study / Indication	Trial ID	Preclinical	Phase 1	Phase 2	Phase 3	Market	Sponsor
Advanced Solid Tumors		Preclinical					Kazia Therapeutics

EVT801

Study / Indication	Trial ID	Preclinical	Phase 1	Phase 2	Phase 3	Market	Sponsor
Advanced Solid Tumors	NCT05114668	Preclinical	Phase 1				Kazia Therapeutics

Paxalisib

Potentially Best-in-Class Dual PI3K/mTOR Inhibitor

Paxalisib was originally discovered by Genentech and targets PI3K, a key regulator of cell growth and division implicated in many forms of cancer.

Paxalisib is an oral, once-a-day, brain-penetrant small-molecule dual PI3K/mTOR inhibitor for solid tumor indications, including brain and breast cancer, evaluated to date in >550 adult and pediatric patients across Phase 1-3 clinical trials and expanded access programs.

Learn More About Paxalisib

PD-L1 Protein Degrader

First-in-Class Nuclear Protein Degrader

We’re developing NDL2, a first-in-class PD-L1 protein degrader designed to redefine how immune checkpoint pathways are targeted in cancer treatment.

Our PD-L1 protein degrader platform lead compound, NDL2, is an optimized bicyclic peptide PD-L1 degrader designed to target cancer cell-enriched nuclear PD-L1 protein, thereby restoring anti-tumor immune responses even in cases of immune evasion or checkpoint inhibitor resistance.

Learn More About PD-L1 Protein Degrader

SETDB1 Inhibitor

First-in-Class SETDB1 Inhibitor

The SETDB1 program centers on a chromatin-modifying enzyme linked to drug resistance, immune evasion, and metastasis. The program leverages extensive peptide screening and optimization to develop intracellular targeting capabilities.

Our SETDB1 inhibitor platform lead compound, MSETC, is an optimized, bicyclic peptide designed to target SETDB1, a key epigenetic regulator of immune evasion.

Learn More About SETDB1 Inhibitor

EVT801

Highly Selective VEGFR-3 Inhibitor

EVT801 is an oral, highly selective small-molecule inhibitor of VEGFR-3 designed to modulate lymphangiogenesis and metastatic spread.

Learn More About EVT801

Clinical Trials

Our clinical trials are progressing through a series of structured, globally-coordinated trials aimed at demonstrating the therapeutic potential of our oncology portfolio.

View Our Clinical Trials