SYDNEY, July 6, 2022
/PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA; ASX:
KZA), an oncology-focused drug development company, is pleased to
announce that the United States Food and Drug Administration (FDA)
has awarded Rare Pediatric Disease Designation (RPDD) to Kazia's
paxalisib for the treatment of atypical rhabdoid / teratoid tumors
(AT/RT), a rare and highly-aggressive childhood brain cancer.
- Rare Pediatric Disease Designation (RPDD) is granted to drugs
which are under development for rare childhood diseases.
- RPDD means that the sponsor may be entitled to receive a
pediatric priority review voucher (pPRV) if the drug is initially
approved for that rare childhood disease. A PRV grants the holder
an expedited six-month review of a new drug application. PRVs are
tradeable and have historically commanded prices in excess of
US$ 100 million.
- FDA's award of RPDD follows the presentation of promising
preclinical data for paxalisib in AT/RT, which was presented by
Professor Jeffrey Rubens and
colleagues at the American Association of Cancer Research (AACR)
Annual Meeting in New Orleans, LA,
in April 2022.
- Paxalisib was previously granted orphan drug designation (ODD)
for AT/RT by FDA on 16 June
Kazia CEO, Dr James Garner,
commented, "this is the second time that paxalisib has been granted
RPDD, and it demonstrates the importance of childhood brain cancer
in the overall paxalisib development program. Brain cancer is the
most common cause of cancer death in children, and outcomes in many
forms of childhood brain cancer have not improved in decades. We
very much hope that paxalisib can make a difference to families
affected by both DIPG and AT/RT, and we will be working closely
with clinicians, researchers, and FDA to determine the optimal way
to move the drug forward."
Rare Pediatric Disease Designation
The Food and Drug Administration Safety and Innovation Act
(2012) established FDA's RPDD initiative. RPDD may be granted to
drugs in development for diseases which primarily affect children
(under the age of 18 years), have an incidence of less than 200,000
new cases per annum in the United
States, and which are serious or life-threatening.
A sponsor of a drug with RPDD may request a Rare Pediatric
Disease Priority Review Voucher (PRV) at the time of a marketing
application to FDA. In effect, the PRV shortens the FDA review
period for a future marketing application of any drug from 12
months to 6 months. PRVs can be sold to other companies and have
historically been transacted at prices in the tens to hundreds of
millions of dollars. For a large company launching a billion-dollar
drug, the six-month acceleration in regulatory review can be of
substantial economic value. In 2019, five pediatric PRVs were
granted by FDA.
A phase II clinical trial of multiple drug therapies, including
paxalisib, is ongoing, under the sponsorship of the Pacific
Pediatric Neuro-Oncology Consortium (PNOC) (NCT05009992). This
study combines several investigational drugs in the treatment of
patients with diffuse midline gliomas (DMGs), a category which
includes DIPG. Initial data from this study is anticipated in
A phase I study of paxalisib in DIPG, led by St Jude Children's
Research Hospital in Memphis, TN
(NCT03696355), is nearing completion, and final data is expected to
be submitted for publication by the end of CY2022.
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
AT/RT in June 2022.
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
For more information, please visit www.kaziatherapeutics.com or
follow us on Twitter @KaziaTx.
This announcement may contain forward-looking statements, which
can generally be identified as such by the use of words such as
"may," "intend," "potential," "prospective," 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. 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.
This document was authorized for release to the ASX by
James Garner, Chief Executive
Officer, Managing Director.
View original content to download
SOURCE Kazia Therapeutics Limited