SYDNEY, Nov. 15,
2023 /PRNewswire/ -- Kazia Therapeutics Limited
(NASDAQ: KZIA), an oncology-focused drug development company, is
pleased to announce that the Society of Neuro-Oncology 2023 Annual
Meeting late breaking abstract entitled "PNOC022: a combination
therapy trial using an adaptive platform design for patients with
diffuse midline gliomas (DMGs) at initial diagnosis, post-radiation
therapy and at time of therapy" was released by the conference
organizers on 10 November 2023. The
preliminary results from a single cohort in this Pacific Pediatric
Neuro-Oncology Consortium (PNOC) sponsored cooperative group
clinical study will be presented on Sunday, 19 November 2023 in Vancouver, Canada. Sixty-eight children and
young adults with DMG who completed standard of care radiation
treatment received paxalisib (an investigational PI3K-mTOR dual
inhibitor) and ONC201 (an investigational dopamine receptor D2
(DRD2) and ClpP agonist). The abstract reported that median overall
survival (OS) from time of diagnosis was 16.5 months.
"We are very excited to see the OS preliminary results of 16.5
months from this cohort of patients, keeping in mind there are two
other cohorts of patients in this study for which data is
anticipated in 2024. The average (median) OS rate for children with
DMG receiving standard of care is less than 1 year, generally
ranging from 8-11 months," stated John
Friend, MD, CEO Kazia
Therapeutics. "The PNOC022 study has exceeded expectations
in terms of enrolment rates and has partnered with 29 leading
children's cancer centers and physicians across the globe."
Key Points from the Abstract
- Sixty-eight patients with biopsy-proven DMG were enrolled
between November 2021 and
June 2023 (median age 9 years [range
3-37], n=41 female [60%])
- Median OS from time of diagnosis was 16.5 months (lower 95%
confidence interval (CI) 11.6 months) with a median follow-up time
of 9.9 months (95% CI: 8.5, 11.4)
- Most common grade 3 and above treatment-related adverse events
were decreased neutrophil count (n=4); mucositis (n=3); and,
colitis, drug reaction with eosinophilia and systemic symptoms,
decreased lymphocyte count, hyperglycemia, and hypokalemia
(n=2)
About the PNOC022 Phase 2 study
The PNOC DMG Adaptive Combination Trial (PNOC022) Phase 2
study is an adaptive platform study that is examining paxalisib in
combination with ONC201, an experimental DRD2 antagonist developed
by Chimerix, Inc. (Durham, NC).
PNOC022 is enrolling children and young adults with DMG, a category
of brain tumours that includes diffuse intrinsic pontine glioma
(DIPG). The study includes three cohorts comprising of newly
diagnosed patients, patients who have completed initial
radiotherapy, and patients who have experienced disease progression
after treatment. The primary endpoint will be the proportion of
patients who are progression-free at six months for newly diagnosed
patients, and OS for recurrent patients.
Rare Pediatric Disease Designation
In 2020, the United States Food and Drug Administration (FDA)
awarded Rare Pediatric Disease Designation (RPDD) to paxalisib for
the treatment of Diffuse Intrinsic Pontine Glioma (DIPG). With RPDD
granted, Kazia may now be eligible to receive a 'priority review
voucher' (PRV) if paxalisib is first approved for DIPG. A PRV
grants the holder an expedited six-month review of a new drug
application by FDA. PRVs can be sold to other companies and have
historically commanded prices in excess of US$100 million.
This announcement was authorized for release by Dr. John Friend, CEO.
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, a 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 study
in glioblastoma reported promising signals of clinical
activity in 2021, and a pivotal study, GBM AGILE, is ongoing, with
final data expected in CY2023. Other clinical trials are ongoing in
brain metastases, diffuse midline gliomas,
and primary CNS lymphoma, with several of these having
reported encouraging interim data.
Paxalisib was granted
Orphan Drug Designation for glioblastoma by the US
Food and Drug Administration (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 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 1
study in advanced solid
tumors commenced recruitment in November 2021.
For more information, please
visit www.kaziatherapeutics.com or follow
us on Twitter @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, and Kazia's strategy and plans with respect
to its programs, including paxalisib and EVT801, as well as any
potential future indications and timing for the release of interim
or final data for such programs. 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, including
the risk that preliminary or interim data may not reflect final
results, 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 United States Securities and Exchange
Commission (SEC), and in subsequent filings with the 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.
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