SYDNEY, Feb. 23, 2018 /PRNewswire/ -- Kazia Therapeutics
Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused
biotechnology company, is pleased to announce that the United
States Food and Drug Administration (FDA) has granted Orphan Drug
Designation (ODD) to Kazia's investigational new drug, GDC-0084,
for the treatment of glioblastoma multiforme, the most common and
most aggressive form of primary brain cancer. The company received
written notification from FDA on Friday 23rd
February 2018.
Key Points
- Orphan Drug Designation (ODD) is a special status accorded to
drugs which are considered promising potential treatments for rare
('orphan') diseases, generally defined as those which affect less
than 200,000 cases per annum in the
United States
- ODD can provide drug developers with up to seven years of
Orphan Drug Exclusivity (ODE), extending the effective life of a
commercial product. It also provides opportunities for grant
funding, protocol assistance, and financial benefits, such as a
waiver of New Drug Application fees, and tax credits
- Licensed from Genentech in October
2016, GDC-0084 is due to commence a phase II clinical trial
in glioblastoma in late March or early April of 2018
Glioblastoma multiforme (GBM) is an area of significant unmet
medical need. More than 130,000 patients are diagnosed worldwide
each year, and the prognosis remains poor, with median survival of
12-15 months on best available care. Existing drug treatments are
largely ineffective in almost two-thirds of patients, and there
remains an urgent need for new therapies.
GDC-0084 was licensed from Genentech in late 2016, after
demonstrating favourable results in a phase I study of 47 patients
with advanced brain cancer. Kazia intends to shortly commence an
international phase II clinical study to provide definitive
evidence of clinical efficacy. This phase II study will initially
be conducted predominantly at leading centres in the United States, and is anticipated to
provide an initial data read-out in early calendar 2019.
Kazia CEO, Dr James Garner,
commented, "we are very pleased to have successfully completed this
important regulatory step in the development of GDC-0084. We share
FDA's recognition of the need for new treatments in this very
challenging disease, and we believe that GDC-0084 has great promise
as a potential new therapy. We anticipate an imminent start of the
phase II clinical study, and look forward to working closely with
the participating clinicians."
About Kazia Therapeutics Limited
Kazia Therapeutics Limited (ASX: KZA, NASDAQ: KZIA) is an
innovative oncology-focused biotechnology company, based in
Sydney, Australia. Our pipeline
includes two clinical-stage drug development candidates, and we are
working to develop therapies across a range of oncology
indications.
Our lead program is GDC-0084, a small molecule inhibitor of the
PI3K / AKT / mTOR pathway, which is being developed to treat
glioblastoma multiforme, the most common and most aggressive form
of primary brain cancer. Licensed from Genentech in late 2016,
GDC-0084 is due to enter a phase II clinical trial in late March or
early April of 2018. Initial data is expected in early calendar
2019.
TRX-E-002-1 (Cantrixil), is a third-generation benzopyran
molecule with activity against cancer stem cells, and is being
developed to treat ovarian cancer. TRX-E-002-1 is currently
undergoing a phase I clinical trial in Australia and the
United States. Initial data is expected in the first half of
calendar 2018.
For more information, please visit
www.kaziatherapeutics.com.