Myriad Genetics Begins Study of Treatment for Metastatic Brain Cancer

Myriad Genetics announced it has initiated a Phase II human clinical trial of its therapeutic candidate Azixa in patients with melanoma that has spread to the brain.

The trial is designed to determine the safety profile of Azixa and the extent of its ability to improve the overall survival of patients with melanoma skin cancer and brain metastases. The trial will compare the survival of patients treated with Azixa with those treated with temozolomide or the combination of Azixa plus temozolomide.

The trial is designed as an adaptive, open-label, multiple-dose study in patients with metastatic melanoma. The first stage of the trial is designed to determine the safety and the maximum-tolerated dose of Azixa in combination with temozolomide. The format for Stage 1 is a six-patient cohort to be given daily temozolomide and weekly Azixa for six weeks. The maximum-tolerated dose of Azixa alone was determined during the earlier trials.

The second stage of the trial will then assess the overall survival of patients treated with Azixa alone compared with temozolomide alone or the Azixa-temozolomide combination therapy. Patients will be randomized into one of the three treatment arms. The study may enroll up to 150 patients per arm.

"In earlier studies, Azixa showed biological activity in a broad range of tumors, and we hope to further define that activity in this second Phase II trial," Adrian Hobden, president of Myriad Pharmaceuticals, said. "We believe that Azixa has the potential to treat multiple forms of primary and metastatic brain cancer, and we look forward to continue developing this potential in this second of a total of three separate Phase II studies."

Azixa has a dual mode of action: It acts both as a cytotoxin and a vascular disrupting agent (VDA). VDAs kill tumor cells by reducing the blood supply to a tumor. The disruption of the tumor vasculature results in acute ischemia followed by massive tumor cell death. Azixa is believed to selectively disrupt tumor vasculature, and not healthy tissue, by inhibiting the formation of microtubules.