Race Oncology (ASX:RAC) has announced that the investigator-sponsored Phase 1b/2 trial of bisantrene in combination with clofarabine and fludarabine in relapsed or refractory acute myeloid leukaemia patients has been successfully concluded having achieved its primary endpoint for efficacy.
The trial was conducted at the Chaim Sheba Medical Centre in Israel under the supervision of Professor Arnon Nagler, a key opinion leader.
The trial has investigated intravenous bisantrene, clofarabine, and fludarabine (Bis/Clo/Flu) in cohorts of adult patients with refractory acute myeloid leukaemia (R/R AML) using Simon’s 2-stage design. This included a Phase 1b lead-in dose escalation stage to establish the maximum tolerated dose or recommended Phase 2 dose of Bis/Clo/Flu and a Phase 2 expansion stage to determine efficacy and confirm the safety of the Bis/Clo/Flu regimen at the RP2D in up to 17 subjects.
The company said six of the 15 evaluable patients in the Phase 2 efficacy stage responded to the Bis/Clo/Flu treatment, with three clinical responders having active extramedullary disease. Five of the six treatment-responsive patients were able to be bridged to a potentially curative stem cell transplant within one to three months of treatment.
Of the five stem-cell transplanted patients, three have since died - one from graft-versus-host disease, one from relapse four months after transplant, and one from infection after two years.
Race Oncology said the patient complete response rate exceeded the trial’s prespecified 2-stage Simon efficacy threshold of 3 or more complete responses.
Race Oncology CEO Dr Daniel Tillett said, “I would like to extend my sincere thanks to Professor Nagler and his dedicated team at the Chaim Sheba Medical Centre, who have spent the last 5 years meticulously investigating the utility of bisantrene for patients with relapsed/refractory Acute Myeloid Leukaemia.
"The Sheba team’s dedication and commitment has added to the strong historical evidence of bisantrene’s efficacy in treating patients with AML and has stimulated further clinician interest in taking the drug forward in new AML trials. We extend our gratitude to all patients and their families whose participation make the development of new AML treatments such as bisantrene possible.”