FDA approves orphan drug designation for Cytrx’s INNO-206 to treat pancreatic cancer

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.