The company’s two previously announced Phase II trials are studying primary brain cancer and melanoma that has spread to the brain.
This third Phase II trial is designed to determine the safety profile of Azixa and to assess the extent of its ability to improve the overall survival of patients. The primary endpoint of the trial is a comparison of the overall survival of patients treated with Azixa alone to those treated with temozolomide, or the combination of Azixa plus temozolomide. Secondary endpoints include progression-free survival and a quality of life assessment of patients.
The trial is an adaptive, open-label, multiple-dose study. Patients will be randomized into one of three treatment arms. The study may enroll up to 150 patients per arm.
“Due to Azixa’s demonstrated ability to cross the blood-brain barrier and its activity in drug-resistant tumors, along with the compelling safety data from our Phase I trials, we have decided to accelerate the development of Azixa through this additional Phase II trial,” said Adrian Hobden, president of Myriad Pharmaceuticals.
Myriad says that in Phase I trials, one in patients with refractory solid tumors that may also have had brain metastases and the other in patients with known brain metastases, Azixa appeared to have a biological effect on patients’ metastases from many different primary tumors, including non-small-cell lung cancer, which is consistent with the mechanism of the drug candidate.
Patients in the studies had typically failed from five to seven previous chemotherapy drugs and were determined by their physicians to be in the terminal stage of the disease, without further approved therapeutic options remaining. Of the 32 patients that received a therapeutic dose of Azixa, 38% responded as determined by tumor shrinkage, stable disease or disruption of the vasculature of the tumor. Two patients with brain metastases were still alive and stable after 15 months of treatment with Azixa.