SYDNEY, June 11,
2025 /PRNewswire/ -- Kazia Therapeutics Limited
(NASDAQ: KZIA), a clinical-stage biotechnology company pioneering
next-generation oncology therapeutics, today announced the
publication of transformative preclinical research in the journal
Molecular Cancer Therapeutics, a journal of the American
Association for Cancer Research that underscores the powerful
potential of its lead asset, paxalisib, in reshaping the treatment
landscape for triple-negative breast cancer (TNBC), one of the most
aggressive and treatment-resistant cancer subtypes.
The preclinical program was conducted by Professor Sudha Rao at the renowned QIMR Berghofer Medical
Research Institute, revealing how paxalisib can reprogram the tumor
microenvironment and enhance immune response, showing substantial
synergy with immune checkpoint inhibitors. The data provide strong
scientific and translational rationale for continued development of
paxalisib as part of immunotherapy-based regimens.
Key Findings
- Dual targeting of PI3K and mTOR but not PI3K alone inhibits
cancer cell proliferation and migration in vitro.
- Paxalisib remodels the TNBC tumor microenvironment, increasing
CD4+ and CD8+ T cell infiltration and activation
- The combination of paxalisib with KEYTRUDA® (pembrolizumab)
demonstrated synergistic antitumor activity in advanced breast
cancer, resulting in robust tumor regression and prolonged survival
in preclinical models
- The data validate a mechanistic rationale for ongoing clinical
exploration of paxalisib in combination with checkpoint inhibitors
and PARP inhibitors
"This landmark study offers a mechanistic and translational
foundation for our newly launched Phase 1b clinical trial of paxalisib in advanced breast
cancer," said Dr. John Friend, CEO
of Kazia. "It not only extends the therapeutic potential of
paxalisib beyond brain cancers but also positions it at the
forefront of innovative immunotherapy combinations in solid
tumors."
The publication, "Combination of the PI3K/mTOR inhibitor
paxalisib with immune checkpoint inhibitors enhances antitumor
activity in preclinical models of triple-negative breast cancer,"
is available online at
https://aacrjournals.org/mct/article/doi/10.1158/1535-7163.MCT-24-0693/762929/Depleting-the-action-of-EZH2-through-PI3K-mTOR.
Clinical Milestone
Kazia recently announced that the
first patient has been dosed in the Phase 1b trial evaluating paxalisib in combination with
checkpoint inhibitors and chemotherapy in advanced breast cancer,
marking a critical step toward clinical translation of these
findings.
For investor and media, please contact Alex Star, Managing
Director LifeSci Advisors LLC
About Kazia Therapeutics Limited
Kazia Therapeutics
Limited (NASDAQ: KZIA) is an oncology-focused drug development
company, based in Sydney,
Australia. Our lead program is paxalisib, an investigational
brain penetrant inhibitor of the PI3K / Akt / mTOR pathway, which
is being developed to treat multiple forms of brain cancer.
Licensed from Genentech in late 2016, paxalisib is or has been the
subject of ten clinical trials in this disease. A completed Phase
2/3 study in glioblastoma (GBM-Agile) was reported in 2024 and
discussions are ongoing for designing and executing a pivotal
registrational study in pursuit of a standard approval. Other
clinical trials involving paxalisib are ongoing in brain
metastases, diffuse midline gliomas, and primary CNS lymphoma, with
several of these trials having reported encouraging interim data.
Paxalisib was granted Orphan Drug Designation for glioblastoma by
the FDA in February 2018, and Fast
Track Designation (FTD) for glioblastoma by the FDA in August 2020. Paxalisib was also granted FTD in
July 2023 for the treatment of solid
tumour brain metastases harboring PI3K pathway mutations in
combination with radiation therapy. In addition, paxalisib was
granted Rare Pediatric Disease Designation and Orphan Drug
Designation by the FDA for diffuse intrinsic pontine glioma in
August 2020, and for atypical
teratoid / rhabdoid tumours in June
2022 and July 2022,
respectively. Kazia is also developing EVT801, a small molecule
inhibitor of VEGFR3, which was licensed from Evotec SE in
April 2021. Preclinical data has
shown EVT801 to be active against a broad range of tumour types and
has provided evidence of synergy with immuno-oncology agents. A
Phase I study has been completed and preliminary data was presented
at 15th Biennial Ovarian Cancer Research Symposium in September 2024. For more information, please
visit www.kaziatherapeutics.com or follow us on X @KaziaTx.
Forward-Looking Statements
This announcement may
contain forward-looking statements, which can generally be
identified as such by the use of words such as "may," "will,"
"estimate," "future," "forward," "anticipate," or other similar
words. Any statement describing Kazia's future plans, strategies,
intentions, expectations, objectives, goals or prospects, and other
statements that are not historical facts, are also forward looking
statements, including, but not limited to, statements regarding:
the timing for results and data related to Kazia's clinical and
preclinical trials, Kazia's strategy and plans with respect to its
programs, including paxalisib and EVT801, the potential results of
its Phase 1b clinical trial
evaluating paxalisib in combination with olaparib or KEYTRUDA®
(pembrolizumab), developed by Merck & Co., Inc. for patients
with advanced breast cancer, the potential benefits of paxalisib as
an investigational PI3K/mTOR inhibitor, timing for any regulatory
submissions or discussions with regulatory agencies, the potential
market opportunity for paxalisib and Kazia's intent and efforts to
regain and/or maintain compliance with the applicable Nasdaq
continued listing requirements and standards. Such statements are
based on Kazia's current expectations and projections about future
events and future trends affecting its business and are subject to
certain risks and uncertainties that could cause actual results to
differ materially from those anticipated in the forward-looking
statements, including risks and uncertainties: associated with
clinical and preclinical trials and product development, related to
regulatory approvals, related to the impact of global economic
conditions, and related to Kazia's ability to regain and/or
maintain compliance with the applicable Nasdaq continued listing
requirements and standards. These and other risks and uncertainties
are described more fully in Kazia's Annual Report, filed on form
20-F with the SEC, and in subsequent filings with the United States
Securities and Exchange Commission. Kazia undertakes no obligation
to publicly update any forward-looking statement, whether as a
result of new information, future events, or otherwise, except as
required under applicable law. You should not place undue reliance
on these forward-looking statements, which apply only as of the
date of this announcement.
This announcement was authorized for release by Dr. John Friend, CEO.
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