SYDNEY, Dec. 31,
2024 /PRNewswire/ -- Kazia Therapeutics Limited
(NASDAQ: KZIA), an oncology-focused drug development company, today
provided a regulatory update on paxalisib for the treatment of
glioblastoma (GBM) following its Type C clinical meeting with the
United States Food and Drug Administration (FDA).
In July 2024, the Company reported
topline results from the GBM-AGILE study in which newly diagnosed
unmethylated (NDU) patients with glioblastoma treated with
paxalisib showed a clinically meaningful improvement in a
prespecified secondary analysis for overall survival (OS) compared
to standard of care. Based on these results and the totality of
data from all completed paxalisib clinical studies, Kazia requested
a meeting with the FDA to discuss potential clinical and regulatory
paths forward.
Following discussions with the FDA and feedback from Kazia's
recent Type C meeting, the FDA's current position is that data on
OS would generally not be appropriate for accelerated approval, but
could be considered to support a traditional/standard approval. The
Agency further commented that the secondary endpoint OS data from
the GBM-AGILE study are supportive and informative for designing
and executing a pivotal registrational study in pursuit of a
standard approval. Importantly, the Company aligned with the
FDA on key aspects of the design of a proposed
registrational/pivotal phase 3 study, including patient population,
primary endpoint, and the comparator arm to be used.
"We appreciate the extensive and thoughtful feedback from the
FDA, which provides us with added clarity with respect to
paxalisib's potential registration pathway for the treatment of
patients with NDU glioblastoma," commented Dr. John Friend, Kazia's CEO "We believe data from
the GBM-AGILE trial, including the prespecified secondary endpoint,
which demonstrated a 3.8-month OS improvement, provides evidence
supporting a clinically meaningful efficacy signal that merits
further testing paxalisib in this patient population in a larger,
pivotal study."
"As we evaluate our next steps in NDU glioblastoma, paxalisib
continues to be tested in a number of other key indications,
including pediatric brain cancer and brain metastases. We have
received Orphan Drug and Rare Pediatric Disease Designations for
both DIPG and AT/RT, which could make us eligible to receive
pediatric review vouchers at the time of product approval. We
recently presented very exciting data at the San Antonio Breast
Cancer meeting highlighting synergistic activity between a novel
combination of paxalisib and immunotherapy, and we believe
paxalisib shows potential to be evaluated in breast cancers where
iPI3K pathway mutations are known to drive tumor growth. The Kazia
team, in conjunction with the Board of Directors, is continuing to
evaluate several options, and we expect to provide an outline for
our path forward to maximize shareholder value by the end of
January 2025."
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 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 on behalf of the
Board of Directors by Dr John
Friend, CEO.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/kazia-therapeutics-provides-update-on-paxalisib-regulatory-pathway-following-type-c-meeting-with-fda-302340490.html
SOURCE Kazia Therapeutics Limited