Full Press Release Details
US FDA AWARDS ORPHAN DRUG DESIGNATION (ODD) TO PAXALISIB
FOR AT/RT, A RARE FORM OF CHILDHOOD BRAIN CANCER
Sydney, 17 June 2022 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 Orphan Drug Designation (ODD) to Kazia s paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive
childhood brain cancer.
Kazia CEO, Dr James Garner, commented, childhood brain cancer has emerged as an important area of focus for the
paxalisib program. We have been working for some years with several world-leading researchers in DIPG, one of the most aggressive childhood cancers. Recent data presented at the AACR conference by Dr Jeffery Rubens and colleagues from Johns Hopkins
Medical School has shown the potential of the drug to also add benefit in AT/RT, another form of childhood brain cancer that is very poorly served by existing treatments. This represents an important new opportunity for paxalisib, and one that we
continue to explore enthusiastically with our collaborators and advisors.
Mr Iain Ross Chairman, Non-Executive Director
Mr Bryce Carmine Non-Executive Director
Mr Steven Coffey Non-Executive Director
Dr James Garner Chief Executive Officer, Managing Director
Three International Towers, Level 24, 300 Barangaroo Avenue, Sydney NSW 2000
Orphan Drug Designation
ODD exists to recognise and encourage 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 over US$ 3 million in FY2022. In addition, drugs with ODD may be eligible for orphan grants by FDA.
received ODD for paxalisib in glioblastoma in February 2018, and for malignant glioma in August 2020.
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 CY2023.
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.
Kazia Therapeutics will participate in a webinar to discuss the company s activities
in childhood brain cancer on Wednesday 22nd June 2022. Registration details are available via the company website.
For More Information, Please
| In the United States: | In Australia: | |
| Joe Green | Jane Lowe | |
| Edison Investor Relations | IR Department | |
| jgreen@edisongroup.com | jane.lowe@irdepartment.com.au | |
| Phone: +1 646-653-7030 | Phone: +61 411 117 774 |
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 Orphan Designation 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 2021.
This document was authorized for release to the ASX by James Garner, Chief Executive Officer, Managing Director.