Kazia Hits Barrier in Glioblastoma Platform Trial

Australian biotech Kazia Therapeutics hit a snag in its GBM AGILE pivotal study, a global adaptive clinical trial platform designed to evaluate multiple therapies for glioblastoma.

Photo: James Garner, CEO of Kazia

The Global Coalition for Adaptive Research (GCAR), the sponsor of the study, advised Kazia that the first stage of the trial arm for paxalisib, the company’s lead experimental therapy, did not meet pre-defined criteria for continuing to a second stage, and patients enrolled in the first stage of the paxalisib arm will therefore continue on treatment as per protocol, and in follow-up, until completion of the final analysis, which Kazia anticipates receiving in the second half of 2023.

Given that recruitment is completed, the study will not open to the paxalisib arm in Germany or China. Kazia will work with its licensing partner to determine the way forward in China, given that country’s general requirement for local data to register a new pharmaceutical product.

All Kazia personnel continue to be blinded to efficacy and safety data from the ongoing study, as required by regulatory authorities, which means the company remains unable to provide analysis or interpretation of the study until follow-up is complete and final data is available.

“GBM AGILE was designed as an adaptive study, with the potential to follow a range of different paths to completion. Today’s news defines the remaining trajectory of the study, with modestly positive implications for both costs and timelines, and with some specific consequences for regulatory strategy in China,” said James Garner, CEO of Kazia. “It does not allow us to draw any meaningful inferences about the outcomes of the study, and indeed it is critical for regulatory purposes that we remain blinded to the evolving data. In the meantime, we are excited by some of the emerging data in diffuse intrinsic pontine glioma (DIPG) and brain metastases, which have become increasingly important areas of focus for the company and look forward to sharing more detail on those activities in due course.”

Kazia Therapeutics’s paxalisib is 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, Fast Track designations by the U.S. Food and Drug Administration for glioblastoma. In addition, paxalisib was granted Rare Pediatric Disease and Orphan designations by the FDA for DIPG and for AT/RT.

Author: Rare Daily Staff