(NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced that the first patient has been dosed in the Company’s 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.
“With the dosing of the first patients we are on schedule with this important Phase 2 GBM trial, and look forward to reporting preliminary results in the second half of this year,” said CytRx President and CEO Steven A. Kriegsman. “Aldoxorubicin has been shown to have the unique ability to cross the blood-brain barrier in animal models of human glioblastoma, potentially creating a new approach to attacking brain tumors. Should the data from this trial be positive, we will pursue the rapid development of aldoxorubicin for unresectable GBM, including filing for ‘breakthrough therapy’ designation with the U.S. Food and Drug Administration, which could expedite marketing approval.”
The primary objective of this Phase 2 trial is to determine progression-free survival (PFS) and overall survival (OS) according to RANO Working Group Criteria. The principal secondary objective is to evaluate the safety of aldoxorubicin in study patients as assessed by the frequency and severity of adverse events. The clinical trial is expected to enroll up to 28 patients randomly assigned equally to receive either 350 mg/m
doxorubicin equivalent) or 250 mg/m
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 will be 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, Calif., City of Hope in Duarte, Calif. and the Louisiana State University Health Sciences Center in New Orleans.
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.