SYDNEY, Oct. 6, 2022
/PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA; ASX:
KZA), an oncology-focused drug development company, today announced
that an ongoing phase II study of paxalisib, an investigational
drug for the treatment of diffuse intrinsic pontine glioma (DIPG)
and other diffuse midline gliomas (DMGs), sponsored by the Pacific
Pediatric Neuro-Oncology Consortium (PNOC), has expanded
internationally, with two sites opening recently in Australia. This is the first time that
paxalisib has been the subject of a clinical trial in Australia.
Key Points
- DMGs are a group of childhood brain cancers which include DIPG,
and which represent approximately 15% of brain tumours in children.
Life expectancy from diagnosis is estimated to be 9-11 months, and
there are currently no FDA-approved drug treatments.
- The ongoing PNOC022 study (NCT05009992) combines paxalisib with
ONC201, an experimental dopamine receptor D2 (DRD2) antagonist
manufactured by Chimerix, Inc. (Durham,
NC).
- To date, recruitment to the study has been robust since it
opened in November 2021. After
initially recruiting entirely at the University of California, San Francisco, the study
is currently active at nine sites in the
United States and two in Australia. Activation in other countries is
under discussion.
"We are very pleased with progress in the study," commented Dr
James Garner, Chief Executive
Officer at Kazia. "Brain cancer is the leading cause of childhood
cancer death, and DMGs represent one of the most challenging types
of tumour. The preclinical data supporting the combination of
paxalisib and ONC201 is substantial and persuasive. We look forward
to seeing initial data from this ongoing clinical study next
year."
Clinical Trial Design
PNOC022 enrols children and young adults with DMGs, a category
of brain tumours that includes DIPG. The study will include
separate cohorts comprising patients with newly diagnosed disease,
patients who have completed initial radiotherapy, and patients who
have experienced disease progression after treatment.
All patients will receive some combination of ONC201 with
paxalisib. The study employs an adaptive design, in which different
arms will be opened and closed based on emerging preclinical and
clinical data. The primary endpoint will be the proportion of
patients who are progression-free at six months (PFS6) for newly
diagnosed patients, and overall survival at seven months (OS7) for
recurrent patients.
The design of the PNOC022 study has been informed by laboratory
research in DIPG, and in particular, by research undertaken at the
University of Newcastle, Hunter
Medical Research Institute (HMRI) in Australia by Associate Professor Matt Dun and colleagues, and funded by RUN DIPG,
the DIPG Collaborative, Chad Tough Defeat DIPG Foundation, and the
McDonald Jones Foundation.
Professor Dun's work was the subject of an oral presentation at
the International Symposium on Pediatric Neuro-Oncology (ISPNO) in
Hamburg, Germany, in June 2022. In the ISPNO presentation, Professor
Dun outlined an extensive body of preclinical work demonstrating
the synergy between ONC201 and paxalisib and described two patients
from compassionate use experience who had demonstrated "dramatic
reductions in tumor volume and complete resolution of disease
symptoms, extending overall survival".
Next Steps
Kazia anticipates initial data from the ongoing PNOC022 study in
CY2023.
The GBM AGILE pivotal study of paxalisib in glioblastoma is
ongoing and is anticipated to produce final data in 2H CY2023.
Depending on the results of the study, Kazia may use such data to
support a new drug application to the FDA for marketing
authorisation.
In addition, six other clinical trials of paxalisib in other
forms of brain cancer are ongoing, with multiple data read-outs
anticipated throughout CY2023.
For More Information, Please Contact:-
Jane Lowe
IR Department
jane.lowe@irdepartment.com.au
Phone: +61 411 117 774
About Kazia Therapeutics
Limited
Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA) is an
oncology-focused drug development company, based in Sydney, Australia.
Our lead program is paxalisib, a brain-penetrant inhibitor of
the PI3K / Akt / mTOR pathway, which is being developed to treat
glioblastoma, the most common and most aggressive form of primary
brain cancer in adults. Licensed from Genentech in late 2016,
paxalisib commenced recruitment to GBM AGILE, a pivotal study in
glioblastoma, in January 2021. Seven
additional studies are active in various forms of brain cancer.
Paxalisib was granted Orphan Drug Designation for glioblastoma by
the US FDA in February 2018, and Fast
Track Designation for glioblastoma by the US FDA in August 2020. In addition, paxalisib was granted
Rare Pediatric Disease Designation and Orphan Designation by the US
FDA for DIPG in August 2020, and for
atypical teratoid / rhabdoid tumours (AT/RT) 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
compelling evidence of synergy with immuno-oncology agents. A phase
I study commenced recruitment in November
2021.
For more information, please visit www.kaziatherapeutics.com or
follow us on Twitter @KaziaTx.
This document was authorized for release to the ASX by
James Garner, Chief Executive
Officer, Managing Director.
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," "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 of Kazia's clinical
trials, and Kazia's strategy and plans with respect to paxalisib.
Such statements are based on Kazia's expectations and projections
about future events and future trends affecting our 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 trials and product development and 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 to SEC.
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.
Actual results could differ materially from those discussed in this
announcement.
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SOURCE Kazia Therapeutics Limited