CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced initiation of a Phase 2 clinical trial with the Company’s aldoxorubicin for the treatment of unresectable glioblastoma multiforme (GBM), a deadly form of brain cancer. The open-label, multi-center study is designed to investigate the preliminary efficacy and safety of aldoxorubicin in subjects with unresectable GBM whose tumors have progressed following prior treatment with surgery, radiation and temozolomide. The clinical trial is expected to enroll up to 28 subjects randomly assigned equally to two groups that will be administered either 350 mg/m 2 (260 mg/m 2 doxorubicin equivalent) or 250 mg/m 2 (185 mg/m 2 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. The primary objective of the clinical trial is to determine progression-free survival (PFS) and overall survival (OS), and the principal secondary objective is an evaluation of the safety of aldoxorubicin in the study subjects. This Phase 2 study follows positive, confirmatory results reported earlier this year 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 only to the tumor in the brain and did not enter normal brain tissue, the principal investigator concluded that aldoxorubicin has the potential to safely shrink glioblastoma tumors, which could dramatically prolong patient survival. This study was replicated by Crown Biosciences, an independent research laboratory.
“We were highly encouraged by aldoxorubicin’s apparent ability to cross the blood-brain barrier, potentially creating a new approach to attacking brain tumors. We are on track with the rapid development of aldoxorubicin for unresectable GBM, and look forward to having preliminary results from this Phase 2 trial in 2014,” said CytRx President and CEO Steven A. Kriegsman. “Should the data from this trial be positive, we plan to file for breakthrough therapy designation with the U.S. Food and Drug Administration, which could expedite marketing approval."Dr. Brian Boulmay of the LSU Health Sciences Center and the principal investigator in the study commented, “Patients with unresectable GBM who have failed prior surgery, radiation and chemotherapy have an extremely poor prognosis, with progression-free survival of around 16 weeks and median overall survival of approximately 31 weeks following treatment. Our Phase 2 study will provide important data on aldoxorubicin’s potential to effectively treat these patients.” About Glioblastoma Multiforme Glioblastoma multiforme (GBM) is the most common and most malignant brain tumor in adults and afflicts more than 12,000 new patients in the U.S. annually. Despite surgical resection, radiotherapy and chemotherapy, the median survival after diagnosis is about 12 to 14 months. Although treatment failure may be due to several factors, limited efficacy of chemotherapeutic agents has been attributed to several contributing factors including insufficient drug delivery to the tumor site through the blood-brain barrier. About Aldoxorubicin The widely used chemotherapeutic agent doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 500 mg/m 2. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. This allows for greater doses of doxorubicin to be administered while reducing its toxic side effects. In studies thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2 g/m 2. About CytRx Corporation CytRx Corporation is a biopharmaceutical research and development company specializing in oncology. CytRx currently is focused on the clinical development of aldoxorubicin (formerly known as INNO-206), its improved version of the widely used chemotherapeutic agent doxorubicin. CytRx is conducting a global Phase 2b clinical trial with aldoxorubicin as a treatment for soft tissue sarcomas, has completed its Phase 1b/2 clinical trial primarily in the same indication and a Phase 1b study of aldoxorubicin in combination with doxorubicin in patients with advanced solid tumors, and has completed a Phase 1b pharmacokinetics clinical trial in patients with metastatic solid tumors. CytRx plans to initiate under a special protocol assessment a potential pivotal Phase 3 global trial with aldoxorubicin as a therapy for patients with soft tissue sarcomas whose tumors have progressed following treatment with chemotherapy. CytRx also is initiating Phase 2 clinical trials with aldoxorubicin in patients with late-stage glioblastoma (brain cancer) and AIDS-related Kaposi’s sarcoma. CytRx plans to expand its pipeline of oncology candidates based on a linker platform technology that can be utilized with multiple chemotherapeutic agents and may allow for greater concentration of drug at tumor sites. CytRx also has rights to two additional drug candidates, tamibarotene and bafetinib. CytRx completed its evaluation of bafetinib in the ENABLE Phase 2 clinical trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL), and plans to seek a partner for further development of bafetinib. For more information about CytRx Corporation, visit www.cytrx.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks relating to the outcome, timing and results of CytRx's clinical trials, the risk that any future human testing of aldoxorubicin, including the Phase 2 study of aldoxorubicin for the treatment of unresectable glioblastoma multiforme (GBM), might not produce results similar to those seen in past human or animal testing, including the mouse study described in this press release, risks related to CytRx's ability to manufacture its drug candidates in a timely fashion, cost-effectively or in commercial quantities in compliance with stringent regulatory requirements, risks related to CytRx's need for additional capital or strategic partnerships to fund its ongoing working capital needs and development efforts, including the Phase 3 clinical development of aldoxorubicin, and the risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.