A year and a half after enrollment began, Kazia Therapeutics' glioblastoma treatment paxalisib has been nixed from a global umbrella trial intended to swiftly investigate a multitude of potential therapies.
The company—formerly known as Novogen—says that although enrollment is now completed, the paxalisib arm did not meet “predefined criteria” for moving on to the second stage of development, according to an announcement Monday. Those criteria were decided by the Global Coalition for Adaptive Research, a collaboration between an evolving number of pharmas and biotechs to simultaneously test treatments for glioblastoma, an aggressive brain cancer. Other participants include Bayer, Biohaven, Kintara and Vigeo.
The GBM Agile trial launched in 2019, and, when treatments are found to be ineffective, they’re dropped from the roster. Simultaneously, potential new therapies are added. Think of it as a track meet where all the runners are competing against the speed of the disease rather than each other.
When asked for additional details, Kazia’s chief financial officer Karen Krumeich would not elaborate on what the “predefined criteria” were. The trial record says that treatments would move onto stage 2 if they reached “an efficacy threshold for graduation” in stage 1. The trial's primary endpoint is overall survival assessed over both study stages.
For Kazia, however, the initial data weren’t promising. But Kazia says it can’t “provide analysis or interpretation of the study until follow-up is complete,” and its personnel remains blinded in the trial. Patients given paxalisib in the first stage will continue on treatment, though, and a full analysis is expected in the second half of 2023.
With recruitment complete, Kazia says it will not conduct new investigations in China, and it is now working with its Chinese licensing partner Simcere to figure out a clinical path forward in the country.
In a statement, Kazia CEO James Garner put a positive spin on the results, saying that had “positive implications” for both costs and timelines. But he acknowledged that leaving GBM Agile had “consequences” for the company’s regulatory strategy in China. Kazia is also testing paxalisib in other brain cancers including diffuse intrinsic pontine glioma, a form of childhood brain cancer, and primary CNS lymphoma.