SYDNEY, Nov. 11, 2021 /PRNewswire/ -- Kazia Therapeutics
Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug
development company, is pleased to announce that PNOC022
(NCT05009992), a multi-drug phase II study in DIPG and diffuse
midline gliomas, has been initiated at the University of California, San Francisco, with the
first patient successfully enrolled to the study.
PNOC022 includes Kazia's investigational drug, paxalisib among
the experimental arms and is expected to provide valuable data on
the potential use of the drug in this highly aggressive form of
childhood brain cancer.
Key Points
- Diffuse intrinsic pontine glioma (DIPG) is the most common of a
group of childhood brain cancers known as diffuse midline gliomas
(DMGs). The disease has no FDA-approved drug treatments and average
survival from diagnosis is approximately 10 months.
- The PNOC022 study is sponsored by the Pacific Pediatric
Neuro-Oncology Consortium (PNOC), an international consortium
focused on the development of new therapies for brain cancer. It is
an adaptive platform study that will examine multiple therapies
concurrently, both as single agents and in combination, to
determine the optimal approach to treatment.
- The lead investigator in the study is Professor Sabine Mueller, a board-certified neurologist
and a pediatric neuro-oncologist with a research program focused on
novel therapies for pediatric brain tumors.
- Design of the study has been informed by research performed in
Australia at the Hunter Medical
Research Institute under the leadership of Associate Professor
Matt Dun, who serves as a scientific
advisor to the study.
- The study has the potential to recruit up to several hundred
patients, with the actual number dependent on emerging
results.
- Kazia's support of the project will consist primarily in
provision of study drug.
The Principal Investigator for the study, Professor Sabine Mueller, commented, "No individual drug
therapy has ever shown convincing efficacy in this group of
patients. We plan to apply novel trial design methodologies to
understand the potential for combination therapy, using some of the
most promising agents in the global pipeline, to change the outcome
in this devastating disease."
Kazia CEO, Dr James Garner,
commented, "We are proud to be part of this important and
innovative clinical study. Preclinical data supports the potential
for paxalisib to combine well with certain other therapies, and we
are keen to see if this approach is similarly promising in the
clinic. There remains a desperate need for new treatment options in
this disease, and we hope that paxalisib may be able to contribute
to better outcomes for patients and their families."
Clinical Trial Design
PNOC022 will enrol children and young adults with diffuse
midline gliomas, 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.
At the outset, all patients will be treated with ONC201,
combined with either paxalisib or panobinostat. 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 (OS) for recurrent patients.
The lead investigator, Professor Sabine
Mueller, is a board-certified neurologist and paediatric
neuro-oncologist whose research focuses on novel therapies in
childhood brain cancer. Professor Mueller holds an academic
appointment in the Department of Neurology, Neurosurgery and
Pediatrics at the University of California,
San Francisco (UCSF) and serves as head of the clinical
program at the DMG Centre at the Children's Hospital of the
University of Zurich. She obtained
her medical degree from the University of Hamburg and also holds a PhD in molecular
biology.
The design of the PNOC022 study has been extensively 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.
The HMRI team has conducted laboratory research with paxalisib for
several years and has generated a powerful body of data combining
paxalisib with other investigational drugs. This research has been
partly funded by RUN DIPG, a not-for-profit organisation led by
Associate Professor Dun, the DIPG Collaborative, Chad Tough Defeat
DIPG Foundation, and the McDonald Jones Foundation.
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. Eight
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.
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.
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SOURCE Kazia Therapeutics Limited