CytRx reports positive interim results from aldoxorubicin Phase 2 trial for treatment of GBM

Jan 6 2015

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced positive interim results from its ongoing Phase 2 clinical trial with aldoxorubicin for the treatment of unresectable glioblastoma multiforme (GBM), a deadly form of brain cancer. The open-label, multisite trial is designed to investigate the preliminary efficacy and safety of aldoxorubicin in patients whose tumors have progressed following prior treatment with surgery, radiation and temozolomide. Preliminary results in 12 patients show both prolonged stable disease and tumor shrinkage in several patients, including one patient who demonstrated no microscopic evidence of tumor when tissue was examined after resection, representing a complete response. These observations suggest that aldoxorubicin allows doxorubicin to cross the tumor's blood-brain barrier in humans.

"Aldoxorubicin appears to be the first anthracycline to cross the blood-brain barrier in GBM, potentially creating a new approach to attacking brain tumors," said Steven Kriegsman, Chairman, President and CEO of CytRx. "These findings are early but important, and suggest that binding to albumin may play a crucial role in transporting chemotherapies that normally cannot cross the tumor blood-brain barrier into the malignancy. They indicate that aldoxorubicin has the potential to address the unmet therapeutic needs of patients suffering from brain cancer. We currently expect to have more definitive data in the first half of this year, which we intend to submit for possible presentation at ASCO 2015."

The primary objective of this Phase 2 trial is to determine progression-free survival (PFS) at 6 months and overall survival (OS) in patients with recurrent glioblastoma multiforme. The principal secondary objective is to evaluate the safety of aldoxorubicin in study patients as assessed by the frequency and severity of adverse events. Only patients who have not received prior treatment with bevacizumab (Avastin®) are eligible to participate in the trial. The clinical trial is expected to enroll up to 28 patients randomly assigned equally to receive either 350 mg/m2 (260 mg/m2 doxorubicin equivalent) or 250 mg/m2 (185 mg/m2 doxorubicin equivalent) of aldoxorubicin intravenously on Day 1, and every 21 days thereafter until evidence of tumor progression, unacceptable toxicity or withdrawal of consent. Tumor response is monitored every 6 weeks by MRI until disease progression occurs. The trial is being conducted at the John Wayne Cancer Center/Sarcoma Oncology Center in Santa Monica, CA, City of Hope in Duarte, CA, the Louisiana State University Health Sciences Center in New Orleans, LA, and Texas Oncology in Dallas, TX.

This Phase 2 study follows positive confirmatory results reported in 2013 from a preclinical study in which aldoxorubicin demonstrated statistically significant efficacy (p<.0001) in the treatment of rapidly growing human brain (glioblastoma) cancer in the brains of animals. In that study, animals treated with aldoxorubicin had median survival of more than 63 days, compared with approximately 25 days for animals treated with doxorubicin or saline. In addition, because aldoxorubicin uptake was confined to the tumor in the brain rather than normal brain tissue, the principal investigator concluded that aldoxorubicin has the potential to safely shrink glioblastoma tumors, which could dramatically prolong patient survival.