Top-line Phase 1 Results for Cantrixil Confirm Earlier Efficacy, Safety Signals

Kazia Therapeutics reported final data from a phase 1 study of cantrixil, a cyclodextrin-encapsulated, third-generation super-benzopyran (SBP) compound with potential antineoplastic activity.

In a statement, the company reported that the trial achieved it's primary objective, determining the maximum-tolerated dose of 5 mg/kg. The study involved 25 patients with advanced metastatic ovarian cancer at 6 sites in the United States and Australia; 11 took part in a dose-escalation arm and 14 took part in a dose-expansion arm.

Overall, 16 patients were evaluable for efficacy, with 1 patient demonstrating a complete response (CR) and 2 demonstrating a partial response, based on RECIST criteria, for an overall response rate of 19%. The patient with the CR remains in remission 3 years after the last dose of cantrixil.

The drug’s main adverse events were abdominal pain, vomiting, and nausea.

"This was a heavily pre-treated population, comprising patients with very advanced disease,” said the Australian lead investigator, Associate Professor Jim Coward, MBBS, PhD, of Icon Cancer Centre. “Existing treatment options for such patients are limited, and there remains an urgent need for new therapies. My colleagues and I are excited by the potential for cantrixil to provide benefit here, and we look forward to seeing the drug move forward in its development.”

Full data from the phase 1 study will be presented at a future scientific meeting, according to the statement from Kazia.