SYDNEY, Feb. 20,
2025 /PRNewswire/ -- Kazia Therapeutics Limited
(NASDAQ: KZIA) ("Kazia" or "the Company"), an oncology-focused drug
development company, today announced a research grant awarded from
The Michael J. Fox Foundation for Parkinson's Research (MJFF) to
fund research between The Hebrew University of
Jerusalem (Hebrew University)
and Kazia to explore the therapeutic potential of paxalisib as a
treatment for Parkinson's disease (PD).
The grant will fund collaborative preclinical studies at
Professor Ronit Sharonʼs lab
(Hebrew University) aimed at
establishing an operational link between a specific pathway in the
pathophysiology of PD and paxalisib. The research will assess the
impact of paxalisib on mouse survival, motor and non-motor
performances, as well as specific biochemical, pathological and
molecular disease biomarkers that will be determined in brains of
treated mice. Paxalisib is a unique, blood-brain
barrier-penetrating inhibitor for class IA phosphatidylinositol
3-kinase (PI3K), and data generated from this research is expected
to provide valuable insights into its potential activity for the
treatment of PD.
"We are pleased to enter into this exciting collaboration with
Dr. Ronit Sharon and The
Hebrew University of Jerusalem, whose
groundbreaking research has led to important discoveries related to
neuronal degeneration in Parkinson's disease and related
synucleinopathies," said Dr. John
Friend, Kazia's CEO. "Overexpression of human α-synuclein
A53T (α-SynA53T) in dopamine neurons is an underlying hallmark and
contributor of Parkinson's disease. As a brain-penetrant PI3K
inhibitor, we believe paxalisib may have the potential to address
the underlying pathophysiology of PD by inhibiting AKT
phosphorylation reaction of α-SynA53T, and the preclinical models
we intend to explore will help answer this critical
question."
The main goal of this research is to delve into the therapeutic
potential of paxalisib, a drug that can enter the brain, and
inhibit class IA phosphatidylinositol 3-kinase (PI3K). The
preliminary findings from Professor Ronit's lab obtained in PD
brains and supported by in-vivo data in a mouse model for PD,
collectively emphasize the role of the canonical PI3K/AKT/mTORC
pathway in the pathogenesis of PD. The preclinical research from
this grant builds upon earlier data obtained in PD brains,
α-SynA53T tg mouse brains and cell models for PD, which
collectively emphasize a distinct role for α-Syn in
hyper-activating the PI3K/AKT/mTORC2 pathway to facilitate α-Synʼs
activity in neuronal lipid metabolism. More information can
be found at
https://www.michaeljfox.org/grant/exploring-paxalisib-novel-therapeutic-parkinsons-disease.
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, potential results of
research between Hebrew University and
Kazia regarding the therapeutic potential of paxalisib for the
treatment of Parkinson's disease, the potential benefits of
paxalisib as an investigational PI3K/mTOR inhibitor, timing for any
regulatory submissions or discussions with regulatory agencies, and
the potential market opportunity for paxalisib. 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, and related to the impact of global economic
conditions. 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.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/kazia-therapeutics-and-the-hebrew-university-of-jerusalem-receives-a-grant-from-the-michael-j-fox-foundation-to-evaluate-the-therapeutic-potential-of-paxalisib-as-a-treatment-for-parkinsons-disease-302381417.html
SOURCE Kazia Therapeutics Limited
