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 paxalisib (formerly GDC-0084) for the treatment of malignant 
glioma, which includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and 
highly aggressive childhood brain cancer.

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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 
- Receipt of ODD follows award of Rare Pediatric Disease Designation (RPDD) for 
DIPG on 7 August 2020

Kazia CEO, Dr James Garner, commented, "Taken together, RPDD and ODD provide a 
powerful suite of incentives, opportunities, and protections for the 
development of paxalisib in DIPG. We look forward to seeing initial data from 
the ongoing phase I study in DIPG at St Jude Children's Research Hospital 
during the second half of calendar 2020. In parallel, we are working closely 
with collaborators, advisors, and researchers to determine the best path 
forward for paxalisib in this devastating disease."

He added, "This award of ODD concludes a program of regulatory optimisation 
that Kazia has initiated for paxalisib over the past six months. As we orient 
paxalisib towards commercialization, these special designations from FDA will 
allow us to move forward in the swiftest and most effective way possible."

Orphan Drug Designation

ODD exists to recognise the development of a drug for a rare disease, which may 
affect adults or children. ODD provides an additional period of 7.5 years data 
exclusivity (for a paediatric disease), which allows companies to better defend 
their products against competition. It also results in a waiver by FDA of fees 
for a marketing application, under the Prescription Drug User Fees Act (PDUFA 
fees), which are just under US$ 3 million in FY2020. In addition, drugs with 
ODD may be eligible for orphan grants by FDA. 

Kazia previously received ODD for paxalisib in glioblastoma in February 2018.

Summary of Paxalisib Regulatory Status
                                     
Glioblastoma                                         DIPG
                                     Most common and most aggressive adult 
brain cancer   Highly aggressive childhood brain cancer
Orphan Designation                   February 
2018                                        August 2020
Fast Track Designation               August 2020
Rare Pediatric Disease Designation   
n/a                                                  August 2020

Next Steps

Kazia expects to present further data from its ongoing phase II study of 
paxalisib in glioblastoma at the Society for Neuro-Oncology (SNO) Annual 
Meeting in November 2020.

Initial efficacy data from the ongoing phase I study of paxalisib in DIPG at St 
Jude Children's Research Hospital is expected during 2H CY2020. Precise timing 
remains uncertain due to pandemic-related disruption in conference schedules, 
but Kazia expects to provide an update to investors at the earliest opportunity.

Paxalisib has been selected to join the international GBM AGILE pivotal study 
in glioblastoma, and recruitment is expected to begin in 2H CY2020.

SOURCE: Kazia therapeutics Limited