CytRx’s INNO-206 Receives FDA Approval for Orphan Drug Designation for Pancreatic Cancer

– CytRx Plans to Advance Phase 2 Clinical Development for Tumor-Targeting Pro-Drug Candidate as a Treatment for Pancreatic Cancer –

– In Earlier Animal Study, Treatment with INNO-206 Resulted in Statistically Significant Reduction in Pancreatic Tumor Size, Outperforming Doxorubicin and Gemcitabine –

LOS ANGELES--(BUSINESS WIRE)-- CytRx Corporation (Nasdaq: CYTR), a biopharmaceutical company specializing in oncology, today announced that its tumor-targeting pro-drug candidate INNO-206 has been approved for orphan drug designation for the treatment of patients with pancreatic cancer by the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA). CytRx holds the exclusive worldwide development and commercialization rights to INNO-206.

“This designation represents an important and exciting step in the overall development program for INNO-206. We are delighted with the FDA’s decision to grant INNO-206 this special status, particularly given that treatment with INNO-206 resulted in a statistically significant, three-fold reduction in the average primary tumor size in an animal model of pancreatic cancer,” said Steven A. Kriegsman, President and CEO of CytRx. “Only a handful of drugs have shown any benefit for the treatment of patients suffering from this rapidly progressing, deadly cancer, and INNO-206 outperformed both doxorubicin and the current standard of care gemcitabine in the animal trial. We are now arranging advancement of INNO-206’s development for pancreatic cancer in a Phase 2 clinical trial.”

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.

INNO-206 is designed to control the release of the commonly prescribed chemotherapeutic doxorubicin and to preferentially target tumors, which the Company believes may make it more effective and less toxic in cancer patients than doxorubicin. Objective clinical responses have been reported in patients with sarcoma, breast and small cell lung cancers. CytRx is currently conducting a Phase 1b safety and dose escalation study with INNO-206 in patients with advanced solid tumors who have failed standard therapies and in the second half of this year, anticipates moving into Phase 2 clinical testing for patients suffering from soft tissue sarcoma.

In July 2009, CytRx announced positive results from an animal trial with a human model of pancreatic cancer. In this trial, treatment with INNO-206 resulted in a statistically significant (p<0.005) three-fold reduction in the average primary tumor size, compared to the control. Treatment with doxorubicin showed only a 30% primary tumor reduction, which was not statistically significant. In a parallel experiment, treatment with gemcitabine, the approved and most commonly prescribed drug for pancreatic cancer, resulted in activity comparable to doxorubicin, with an approximate 30% primary tumor volume reduction. Additionally, although no statistically significant inhibition of tumor spread was demonstrated by either INNO-206 or gemcitabine, due to large variability between individual animals in the control, a substantial trend was observed in the INNO-206 group with an approximate 10-fold decrease in tumor spread to the liver and stomach. The toxicity associated with drug treatment was comparable among the treated groups.

About Pancreatic Cancer

Pancreatic cancer, although a relatively rare form of cancer, is the fourth leading cause of cancer mortality in the U.S. with only a 25% one-year survival rate, according to the American Cancer Society. The American Cancer Society estimates that more than 43,000 new pancreatic cancer cases occurred and more than 36,000 deaths were due to this disease in the U.S. in 2010. One in 76 people is expected to develop pancreatic cancer sometime in their life.

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: bafetinib, tamibarotene and INNO-206. The Company is evaluating bafetinib in the ENABLE Phase 2 clinical trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL), a pharmacokinetic clinical trial in brain cancer and the PROACT Phase 2 clinical trial in advanced prostate cancer. With its tumor-targeting pro-drug candidate INNO-206, CytRx is conducting a safety trial with plans to initiate Phase 2 proof-of-concept clinical trials as a treatment for soft tissue sarcomas and to support third-party development for pancreatic cancer. CytRx's pipeline also includes tamibarotene, which it is testing 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 initiation, outcome, timing and results of clinical testing of INNO-206, including in patients with pancreatic cancers, 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 pancreatic cancer, 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.


Investor Relations
Legend Securities, Inc.
Thomas Wagner
800-385-5790 x152
718-233-2600 x152
[email protected]

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