What Kazia Therapeutics does

Kazia Therapeutics Limited is an Australia-incorporated, Nasdaq-listed (KZIA) clinical-stage oncology biotechnology company headquartered in Sydney. Founded in 2016 as a spin-out from Novogen, the company develops drug candidates targeting cancers that are poorly served by existing therapies, including glioblastoma, triple-negative breast cancer (TNBC), renal cell carcinoma, liver cancer, and rare pediatric brain tumors such as DIPG and AT/RT. The pipeline is anchored by paxalisib (GDC-0084), a brain-penetrant dual PI3K/mTOR inhibitor with FDA Rare Pediatric Disease and Orphan Drug designations, currently in Phase 1b/2 development. EVT801, a small-molecule VEGF receptor inhibitor for renal and liver cancers, is in Phase 1 trials. The preclinical pipeline has been expanded through the in-licensing of NDL2, a first-in-class nuclear PD-L1 protein degrader showing 49-73% tumor reduction in TNBC models, and MSETC, a SETDB1-targeted epigenetic bicyclic peptide platform acquired from QIMR Berghofer in April 2026. First-in-human trials for NDL2 are planned for 2027.

The company operates a pre-revenue business model, funding development through private placements and equity raises (over $54 million in 2025-2026 alone) rather than product sales, with anticipated future revenue from licensing deals or post-approval commercialization. Its go-to-market approach is enterprise-focused: scientific engagement through peer-reviewed publications and major oncology conferences (AACR, ASCO, Society for Neuro-Oncology), regulatory interaction through Type C meetings and the FDA's Project FrontRunner pathway, and clinical-trial-based drug distribution across US, Canadian, Australian, and European sites. The company previously traded on the ASX but delisted in late 2023 to consolidate on Nasdaq, where it regained full compliance following a $50 million private placement in December 2025. Leadership was refreshed in 2026 with the appointments of Dr. Sudha Rao (CSO, April 2026) and James Levine (CFO, June 2026), alongside CEO Dr. John Friend (appointed May 2023).

Name

Kazia Therapeutics

Legal name

Kazia Therapeutics Limited

Website

https://kaziatherapeutics.com

Company type

Public

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Short description

Kazia Therapeutics is a clinical-stage Australian oncology biotech developing brain-penetrant PI3K/mTOR inhibitor paxalisib for glioblastoma and TNBC, plus preclinical programs in protein degradation and epigenetic immunotherapy, targeting cancers poorly served by existing therapies.

Ownership category

akta.pro rank

Product category

Oncology Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

akta.pro secondary industry

Personalized Therapeutic Design (neoantigen vaccines, individualized cell/gene therapies) (HLAAANAK)

Keywords

• Oncology drug development
• Glioblastoma therapeutics
• PI3K inhibitor research
• Protein degradation therapy
• Cancer epigenetics

Headquarters

HQ city

Hornsby

HQ country

Australia

HQ region

Oceania

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. Drug Development and Commercialization: Kazia Therapeutics is a pre-revenue clinical-stage biotech company developing oncology drugs. The company has no commercialized products and does not generate product revenue. Funding comes from private placements, institutional financing, and potential future partnerships or licensing deals.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

Kazia Therapeutics is a clinical-stage oncology company developing proprietary drug candidates for cancers with significant unmet medical need. Its portfolio centers on Paxalisib, a brain-penetrant dual PI3K/mTOR inhibitor for glioblastoma and triple-negative breast cancer, alongside the EVT801 VEGF receptor inhibitor, the NDL2 nuclear PD-L1 protein degrader, and the MSETC SETDB1-targeted epigenetic platform acquired from QIMR Berghofer.

Product overview

Kazia Therapeutics is a clinical-stage oncology company developing a pipeline of cancer drug candidates. The company's portfolio includes the lead asset Paxalisib (a brain-penetrant PI3K/mTOR inhibitor) in clinical development for glioblastoma and triple-negative breast cancer, EVT801 in clinical trials for advanced cancers, and preclinical programs including NDL2 (a nuclear PD-L1 protein degrader) and MSETC (a SETDB1-targeted epigenetic platform). The company focuses on treatments for brain cancer, renal cancer, and liver cancer that are poorly served by existing therapies.

Differentiator

Problem solved

Functional benefit

Brands

• Paxalisib: Lead brain-penetrant PI3K/mTOR inhibitor being developed for glioblastoma, triple negative breast cancer, and other oncology indications
• NDL2
• MSETC
• EVT801

Products and services

• Paxalisib Brain-penetrant dual PI3K/mTOR inhibitor being developed for glioblastoma, triple-negative breast cancer, and other oncology indications including pediatric brain cancers such as DIPG and AT/RT. It is Kazia's lead clinical-stage drug candidate.
• EVT801 VEGF receptor inhibitor candidate in clinical development for advanced renal cell carcinoma and liver cancers. A small-molecule oncology therapy intended to address tumor angiogenesis.
• NDL2 First-in-class nuclear PD-L1 protein degrader designed to address immunotherapy resistance and metastatic progression, with preclinical TNBC data showing 49% primary tumor reduction as monotherapy and 73% in combination with anti-PD-1 therapy.
• MSETC Lead candidate bicyclic peptide from the SETDB1-targeted epigenetic drug development platform in-licensed from QIMR Berghofer, designed to restore immune signaling in tumors resistant to immunotherapy.

Quantifiable outcome

• NDL2 reduced primary tumor volume by 49% as monotherapy and 73% in combination with anti-PD-1 therapy in TNBC models
• +4 more outcomes

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Three partnerships are on record, tiered core.

• QIMR Berghofer Medical Research InstitutecoreStrategic or Co-development Partner · 13 April 2026Kazia Therapeutics in-licensed a first-in-class SETDB1-targeted epigenetic drug development platform from QIMR Berghofer for approximately $1.39 million upfront, plus a tiered revenue-sharing structure with no clinical or regulatory milestone obligations. The platform includes lead candidate MSETC, a bicyclic peptide designed to restore immune signaling in tumors resistant to immunotherapy. Dr. Sudha Rao, CSO, is the scientific originator of this platform.
• QIMR Berghofer Medical Research InstitutecoreStrategic or Co-development PartnerOngoing collaboration with QIMR Berghofer for epigenetic platform development. The platform targets SETDB1 proteins to restore immune signaling in immunotherapy-resistant tumors. Combined cost to advance both SETDB1 and PD-L1 degrader programs to IND readiness expected to be approximately $6 million over 18 months, with substantial portion eligible for Australian R&D tax incentives.
• QIMR Berghofer Medical Research InstitutecoreStrategic or Co-development PartnerPartnership for development of MSETC and follow-on compounds from the SETDB1 program. The transaction extends Kazia's oncology strategy alongside existing paxalisib program and PD-L1 platform.

Scale indicators10 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• CNS Pharmaceuticals: Clinical-stage oncology biotech developing brain-penetrant therapies for glioblastoma multiforme (berubicin). Directly comparable clinical-stage GBM-focused peer with similar patient population and regulatory pathway.
• Novocure: Commercial-stage oncology company whose Tumor Treating Fields (TTFields) therapy is approved for glioblastoma. Direct GBM treatment peer operating in the same indication Kazia's lead asset targets.
• Northwest Biotherapeutics: Clinical-stage immuno-oncology company developing personalized dendritic cell therapies (DCVax) for glioblastoma and other solid tumors. Closely comparable clinical-stage GBM peer with similar regulatory pathway.
• Denovo Biopharma: Clinical-stage oncology company with biomarker-driven drug development programs including glioblastoma. Comparable clinical-stage biotech pursuing novel oncology mechanisms with similar platform strategy.

Emerging players

• Arvinas: Clinical-stage biotech pioneering targeted protein degradation (PROTAC platform). Directly comparable to Kazia's NDL2 nuclear PD-L1 degrader program given shared focus on protein degradation oncology modalities.
• Cellectar Biosciences: Clinical-stage oncology company developing targeted phospholipid drug conjugates. Comparable clinical-stage oncology profile and former employer of Kazia's CEO Dr. John Friend.
• Cardiff Oncology: Clinical-stage oncology company developing onvansertib for KRAS-mutated cancers. Comparable clinical-stage small-cap oncology peer and former employer of Kazia's CFO James Levine.
• VBL Therapeutics: Clinical-stage oncology company developing gene-therapy-based treatments for solid tumors including GBM (ofra-vec). Comparable clinical-stage oncology profile with overlapping GBM indication focus.
• Mustang Bio: Clinical-stage cell and gene therapy company with multiple oncology programs including GBM (MB-101). Comparable clinical-stage small-cap biotech profile running several parallel oncology programs.
• Kymera Therapeutics: Clinical-stage biotech developing targeted protein degradation therapies using heterobifunctional small molecules. Directly relevant peer for Kazia's NDL2 nuclear PD-L1 degrader given shared focus on oncology protein degradation.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

• Xx.com/kaziatx
• LinkedInlinkedin.com/company/kaziatx

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds9 records

Investors6 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Kazia Therapeutics