Kazia Therapeutics’s stocks have skyrocketed by more than 250% after succeeding in a Phase II/III trial in glioblastoma.
The company announced positive results from an investigator-led platform trial, which was investigating its lead candidate, paxalisib, versus standard of care (SOC) for patients with glioblastoma.
The GBM-AGILE trial (NCT03970447), fronted by Global Coalition for Adaptive Research (GCAR), a nonprofit research organisation, is investigating a variety of agents for glioblastoma.
Prespecified secondary analysis of newly diagnosed unmethylated (NDU) patients, showed a median OS of 15.54 months in the paxalisib arm versus 11.89 months for concurrent standard of care (SOC).
Kazia CEO Dr John Friend stated: "We are excited to have shown a 3.8-month improvement in overall survival, an approximate 33% improvement, for newly diagnosed unmethylated patients with GBM compared to the concurrent standard of care arm. Having comparable overall survival data across two independent studies is a compelling outcome in this difficult-to-treat glioblastoma population. We look forward to discussing possible approaches for an accelerated approval pathway for paxalisib with the FDA."
Investors also agreed with Friend's statement, with the company’s stock price skyrocketing by 260% from $0.30 opening on 10 July to $1.08 at its peak after the announcement, before stocks settled at $0.67 at closing, a 123% increase.
Kazia said that secondary analysis results are consistent with the Phase II study, where the median OS was 15.7 months for paxalisib-treated NDU patients compared to 12.7 months in patients on temozolomide chemotherapy.
Despite the big success, the primary analysis was not as favourable, with data showing an overall survival (OS) rate of 14.77 months for paxalisib versus 13.84 months for SOC in NDU patients.
The drug performed worse than the placebo in recurrent disease, with a median OS of 9.69 months for concurrent SOC versus 8.05 months for paxalisib. Kazia said it will be further pursuing this data.
Paxalisib was well tolerated in GBM-AGILE, and no new safety signals were identified in this patient population.
Paxalisib acts by targeting the phosphoinositide 3-kinase (PI3K) and mTOR. The drug elicits anti-neoplastic activity by inhibiting and suppressing the signalling within this pathway that causes tumour growth.
Kazia will now look to arrange a meeting with the US Food and Drug Administration (FDA) in hopes of accelerating an approval pathway for the candidate. The candidate received orphan drug and fast track designations from the FDA for glioblastoma in unmethylated MGMT promoter status patients, following radiation plus temozolomide therapy.
The GBM-AGILE trial is also investigating candidates, including Bayer’s Stivarga (regorafenib), Kintara Therapeutics’s VAL-083 and Biohaven Pharmaceuticals troriluzole, among others.
Full data from the paxalisib arm of the trial is expected later this year.
