About the PNOC022 Phase 2 study
The PNOC DMG Adaptive Combination Trial (PNOC022) Phase 2 study is an adaptive platform study that is examining paxalisib in combination with ONC201, an
experimental DRD2 antagonist developed by Chimerix, Inc. (Durham, NC). PNOC022 is enrolling children and young adults with DMG, a category of brain tumours that includes diffuse intrinsic pontine glioma (DIPG). The study includes three cohorts
comprising of newly diagnosed patients, patients who have completed initial radiotherapy, and patients who have experienced disease progression after treatment. The primary endpoint will be the proportion of patients who are progression-free at six
months for newly diagnosed patients, and OS for recurrent patients.
Rare Pediatric Disease Designation
In 2020, the United States Food and Drug Administration (FDA) awarded Rare Pediatric Disease Designation (RPDD) to paxalisib for the treatment of Diffuse
Intrinsic Pontine Glioma (DIPG). With RPDD granted, Kazia may now be eligible to receive a priority review voucher (PRV) if paxalisib is first approved for DIPG. A PRV grants the holder an expedited
six-month review of a new drug application by FDA. PRVs can be sold to other companies and have historically commanded prices in excess of US$100 million.
This announcement was authorized for release by Dr. John Friend, CEO.
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, a 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
study in glioblastoma reported promising signals of clinical activity in 2021, and a pivotal study, GBM AGILE, is ongoing, with final data expected in CY2023. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and
primary CNS lymphoma, with several of these having reported encouraging interim data.
Paxalisib was granted Orphan Drug Designation for glioblastoma by
the US Food and Drug Administration (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 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 1
study in advanced solid tumors commenced recruitment in November 2021.
For more information, please visit www.kaziatherapeutics.com or follow us
on Twitter @KaziaTx.