CytRx Receives FDA Orphan Drug Designation for INNO-206 for the Treatment of Soft Tissue Sarcomas

Company plans to initiate INNO-206 Phase 2b clinical trial in advanced soft tissue sarcomas in the second half of 2011

LOS ANGELES--(BUSINESS WIRE)-- CytRx Corporation (Nasdaq: CYTR), a biotechnology company specializing in oncology, today announced that the Office of Orphan Product Development of the U.S. Food and Drug Administration (FDA) has granted INNO-206 orphan drug designation for the treatment of patients with soft tissue sarcomas. Soft tissue sarcomas are cancers that are formed in the muscle, fat, fibrous tissue, blood vessels or other supporting tissue of the body. CytRx holds the exclusive worldwide development and commercialization rights to INNO-206.

INNO-206 is a novel conjugate of doxorubicin, a commonly prescribed chemotherapeutic, and was designed to improve efficacy and reduce adverse events through controlled release and preferential targeting of tumors. Doxorubicin is currently the only FDA-approved drug on the market for soft tissue sarcoma, and is a standard chemotherapeutic treatment for a variety of other cancers. In April 2011, the FDA granted INNO-206 orphan drug designation for treating patients with pancreatic cancer.

CytRx plans to begin a Phase 2b clinical trial with INNO-206 in patients with late-stage soft tissue sarcomas in the second half of 2011, following completion of an ongoing open-label Phase 1b clinical trial in patients with advanced solid tumors who have failed standard therapies. The Phase 1b clinical trial, which consists mostly of patients with soft tissue sarcomas, is evaluating the safety of administering doses of INNO-206 that are more than two to four times the standard dose of doxorubicin.

“Our strategy to move quickly into a Phase 2b trial with INNO-206 in soft tissue sarcomas is further supported by the FDA’s approval of orphan drug designation," said Steven A. Kriegsman, President and CEO of CytRx. “We envision a significant opportunity in this indication due to the objective clinical responses seen with INNO-206 in patients with sarcomas in an earlier Phase 1 trial as well as preclinical data.”

Patients with advanced soft tissue sarcomas who can no longer be treated with surgery have a poor prognosis. Progression-free survival for this group is around six to seven months, and median overall survival is approximately 18 months with less than one-third of these patients living past three years. Combinations of the chemotherapy drugs ifosfamide and doxorubicin appear to offer the highest response rates and longest time to progression in these patients; however, these regimens have not significantly improved survival.

In the United States, under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition, which is generally a disease that affects fewer than 200,000 individuals in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA requirements. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee.

About CytRx Corporation

CytRx Corporation is a biopharmaceutical research and development oncology company engaged in the development of high-value human therapeutics. The CytRx oncology pipeline includes three programs in clinical development for cancer indications: INNO-206, tamibarotene and bafetinib. With its tumor-targeted doxorubicin conjugate INNO-206, CytRx plans to initiate a Phase 2b clinical trial as a treatment for soft tissue sarcomas, following a Phase 1b dose escalation safety trial. The Company is evaluating bafetinib in the ENABLE Phase 2 clinical trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL) and the PROACT Phase 2 clinical trial in advanced prostate cancer, and is conducting a pharmacokinetic clinical trial in brain cancer. CytRx's pipeline also includes tamibarotene, which it is testing in a double-blind placebo-controlled Phase 2 clinical trial in patients with non-small-cell lung cancer, and which is in a registration clinical trial as a treatment for acute promyelocytic leukemia (APL). For more information on the Company, visit

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 or uncertainties related to the outcome, timing and results of CytRx's clinical testing for INNO-206 in patients with soft tissue sarcomas, uncertainties regarding regulatory approvals for current and future clinical testing of INNO-206 and the scope of the clinical testing that may eventually be required by regulatory authorities for INNO-206, the significant time and expense that will be incurred in developing any of the potential commercial applications for INNO-206, including for soft tissue sarcomas, the risk that any future human testing of INNO-206 for might not produce results similar to those seen in animals, risks related to CytRx's ability to manufacture its drug candidates, including INNO-206, 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 any future clinical development of INNO-206, 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.


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