Kazia Therapeutics Announces Phase II/III Clinical Trial Results for Paxalisib in Glioblastoma
GBM AGILE trial data shows clinically meaningful improvement in a prespecified secondary analysis for overall survival in paxalisib-treated, newly diagnosed unmethylated patients with glioblastoma
GBM AGILE STUDY
GBM AGILE is an adaptive phase II/III global trial sponsored by the
Paxalisib is the third drug candidate to complete its evaluation in the study and was evaluated in newly diagnosed glioblastoma patients with unmethylated MGMT promoter status as well as in patients with recurrent disease.
GBM AGILE Paxalisib Results
Kazia CEO, Dr
A total of 313 newly diagnosed unmethylated (NDU) patients and recurrent patients being treated at top US cancer hospitals were randomized in Stage 1 to either a paxalisib treatment arm (60 mg/day) or the SOC concurrent control arm from
For the primary analysis the median Overall Survival (OS) was 14.77 months for paxalisib-treated NDU patients (n=54) versus 13.84 months for cumulative SOC NDU patients (n=75).
For a prespecified secondary analysis in the NDU patients, median OS was 15.54 months in the paxalisib arm (n=54) versus 11.89 months for concurrent SOC (n=46). In addition, a prespecified sensitivity analysis in NDU patients showed similar median OS difference between paxalisib treated patients (15.54 months) and concurrent SOC patients (11.70 months).
The secondary analysis results are consistent with the previously reported Company-sponsored phase II study, where median OS was 15.7 months (n=27) for paxalisib treated NDU patients compared to 12.7 months historically reported with temozolomide in this patient group (Wen 2022).
Paxalisib was well tolerated in GBM-AGILE, and no new safety signals were identified in this patient population.
An efficacy signal was not detected in the recurrent disease population (median OS of 9.69 months for concurrent SOC (n=113) versus 8.05 months for paxalisib (n=100). Similar results in this population have been reported in the other two drug candidates that have completed the GBM AGILE trial. Kazia is currently pursuing further analyses of this data to elucidate potential signals for further consideration.
Based on the totality of data available from all completed paxalisib clinical studies in newly diagnosed unmethylated GBM patients, Kazia will request a meeting with the
Paxalisib has previously received orphan drug designation and fast track designation from the FDA for glioblastoma in unmethylated MGMT promoter status patients, following radiation plus temozolomide therapy.
Full data including secondary endpoints from the paxalisib arm of the GBM AGILE study is expected to be presented at a scientific meeting later this year.
About
Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from
Paxalisib was granted Orphan Drug Designation for glioblastoma by the FDA in
Kazia is also developing EVT801, a small-molecule inhibitor of VEGFR3, which was licensed from Evotec SE in
For more information, please visit www.kaziatherapeutics.com or follow us on X @KaziaTx.
Forward-Looking Statements
This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, the potential benefits of Kazia's product candidates, including paxalisib, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the
This announcement was authorized for release by Dr
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