Background
The phase II study of paxalisib (NCT03522298) opened to recruitment in May 2018. It was designed to establish the most appropriate dose for use in
newly-diagnosed patients, and to seek initial indications of potential clinical efficacy.
The study had previously determined a maximum tolerated dose
(MTD) of 60mg, administered once daily. This determination was based primarily on safety findings, which suggested increased toxicity at a higher dose. Today’s PK data corroborates this finding, and shows that increased doses provide limited
additional benefit in terms of drug exposure. In effect, these two independent variables both point to a dose of 60mg, giving a high degree of confidence that this is appropriate for future studies and for commercialisation.
Interim analyses of efficacy data from this study have previously shown encouraging signals of clinical efficacy, with a median overall survival (OS) of 17.5
months and a median progression-free survival (PFS) of 8.4 months reported at the most recent data cut. These figures compare very favourably to historical controls for temozolomide, which provide an OS of 12.7 months and a PFS of 5.3 months.
The phase II study remains ongoing, with a number of patients in follow-up, and is expected to deliver final data in 2H CY2021.
In January 2021, paxalisib opened to recruitment in the GBM AGILE pivotal study, which is expected to provide the basis for registration in the US and other
territories.
About Kazia Therapeutics Limited
Kazia
Therapeutics Limited (ASX: KZA, 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 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 other 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. In March 2021, Kazia licensed Greater China rights
for paxalisib to Simcere Pharmaceutical Company.
For more information, please visit www.kaziatherapeutics.com.
This document was authorized for release to the ASX by James Garner, Chief Executive Officer, Managing Director.