CytRx’s (CYTR) Aldoxorubicin Shrinks Tumors and Prolongs Survival

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced that aldoxorubicin, its more potent version of the widely used chemotherapeutic agent doxorubicin, demonstrated statistically significant efficacy (p<.0001) in the treatment of rapidly growing human brain (glioblastoma) cancer in the brains of animals. Complete results from this favorable confirmatory trial, which was conducted in collaboration with Louisiana State University (LSU) School of Medicine, will be presented at the European Society for Medical Oncology being held September 29-October 1 2013 in Amsterdam.

“We are surprised and excited about the effectiveness demonstrated by aldoxorubicin in this particularly difficult-to-treat cancer,” said Om Prakash, Ph.D., the study’s principal investigator and Research Professor of Medicine, Stanley S. Scott Cancer Center, Louisiana State University Health Sciences Center, New Orleans. “It has been well documented that doxorubicin, although active against glioblastoma cancer cells in tissue culture, does not cross the blood-brain barrier, the body’s natural defense system protecting the brain, to effectively treat patients with brain tumors. In fact, in our study doxorubicin was no more effective than saline in suppressing glioblastoma tumor growth. We have shown that aldoxorubicin uptake is confined only to the tumor in the brain and does not enter normal brain tissue. Thus, we would expect toxicity in the central nervous system to be negligible. Our conclusion from this trial is that aldoxorubicin has the potential to safely shrink glioblastoma tumors which could dramatically prolong the average survival time in patients. We initially had observed a similar effect of aldoxorubicin on glioblastoma in a preliminary study, and are quite pleased to have confirmed the result in a larger, well-controlled study that included native doxorubicin.”

Dr. Prakash’s main focus of his research efforts in the last few years has been to understand the pathogenesis and treatment of glioblastoma multiforme, the most malignant and the most deadly type of brain tumor. He is the corresponding author on several poster presentations in national/international meetings. More recently, he is the first author on a publication, Gliomas and Seizures in the Medical Hypothesis Journal (Prakash et al. 2012; 79:622).

“This trial produced remarkable results in a deadly cancer that virtually always returns regardless of whether treated with surgery, radiation, chemotherapy or a combination of methods,” said CytRx President and CEO Steven A. Kriegsman. “Animals treated with aldoxorubicin survived on average more than twice as long as those treated with saline or doxorubicin.

“Aldoxorubicin could provide an exciting new approach in how we attack brain tumors. These outstanding results support our plan to initiate a Phase 2b clinical trial with aldoxorubicin in patients with relapsed glioblastoma. We remain on track with expanding our aldoxorubicin clinical development activities and expect our progress to accelerate in the coming months and year,” he added. If the data from the company’s planned Phase 2b clinical trial for glioblastoma are positive, it plans to file for breakthrough therapy designation with the U.S. Food and Drug Administration, which could expedite marketing approval for aldoxorubicin.

Aldoxorubicin has shown to be superior to doxorubicin in seven different tumor types and animal models of cancer, including ovarian, lung, breast and pancreatic cancer, as well as multiple myeloma, and has demonstrated activity in human trials for the treatment of soft tissue sarcomas and other cancers. Aldoxorubicin is the first drug candidate CytRx is developing based on a novel linker technology that has proven ability to allow attachment of multiple chemotherapeutic agents and is designed to provide both greater anti-cancer activity and to mitigate the toxicity that limits these agents’ use.

About Glioblastoma Multiforme

Glioblastom 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 12-14 months. Although the reason for treatment failure may depend upon 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 CytRx Corporation

CytRx Corporation is a biopharmaceutical research and development company specializing in oncology. The CytRx oncology pipeline is focused on the clinical development of aldoxorubicin (formerly known as INNO-206), its improved version of the widely used chemotherapeutic agent doxorubicin. CytRx has initiated an international 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 is conducting a Phase 1b pharmacokinetics clinical trial in patients with metastatic solid tumors. The Company is initiating a Phase 3 pivotal trial under a special protocol assessment (SPA) with aldoxorubicin as a therapy for patients with soft tissue sarcomas whose tumors have progressed following treatment with chemotherapy. CytRx is expanding its pipeline of oncology candidates based on a novel linker platform technology that can be utilized with multiple chemotherapeutic agents and could allow for greater concentration of drug at tumor sites. The Company also has rights to two additional drug candidates, tamibarotene and bafetinib. The Company 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, and is evaluating further development of tamibarotene. 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 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.