CytRx Corporation Overview

CytRx Corporation (NASDAQ: CYTR), located in Los Angeles, California, is a biopharmaceutical research and development company specializing in oncology. 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 has initiated an international Phase 2b clinical trial as a treatment for soft tissue sarcomas, is completing its ongoing Phase 1b/2 clinical trial and plans to initiate a Phase 2 trial for an undisclosed solid tumor indication in the first half of 2012. CytRx's pipeline also includes tamibarotene, which it is testing in a double-blind, placebo-controlled, international Phase 2b clinical trial in patients with non-small-cell lung cancer, and which is in a clinical trial as a treatment for acute promyelocytic leukemia (APL). The Company is evaluating 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. In 2011, CytRx completed its strategy of monetizing its non-core assets through the sale of its molecular chaperone technology to Denmark-based Orphazyme ApS in a transaction valued up to $120 million, the sale of its 19% interest in SynthRx to ADVENTRX Pharmaceuticals, and the disposition of its remaining shares of RXi Pharmaceuticals in a series of transactions that provided CytRx with approximately $17 million in non-dilutive financing.

The CytRx Drug Development Portfolio

INNO-206 (formerly DOXO-EMCH) is a novel, tumor-targeted conjugate of the commonly prescribed chemotherapeutic agent doxorubicin that binds covalently to albumin, the most abundant protein in blood plasma, and is circulated throughout the body. Doxorubicin is a standard chemotherapeutic treatment for a variety of cancers and is used either alone or in combination with other chemotherapy agents. INNO-206 is designed with a linker that releases doxorubicin in the low pH environment of tumors, concentrating the chemotherapeutic agent where it preferentially damages the tumor while minimizing the effect on healthy tissues. This conjugate formulation has the potential to safely deliver greater amounts of doxorubicin directly to the tumor compared with standard doxorubicin treatment, which could lead to improved efficacy. CytRx holds the exclusive worldwide rights to INNO-206.

In a Phase 1 clinical trial, INNO-206 was administered in doses at up to six times the standard dosing of doxorubicin without an increase in observed side effects over those historically seen with doxorubicin. Animal studies conducted by INNO-206 inventor Dr. Felix Kratz, Department of Medical Oncology, Clinical Research, at the Tumor Biology Center in Freiburg, Germany, demonstrated statistically significant results in breast, ovarian, pancreatic and small cell lung cancers. Results of these studies were published in the peer-reviewed journal Investigational New Drugs.

CytRx is conducting a Phase 1b/2 clinical trial with INNO-206 in patients with advanced solid tumors, and has initiated a Phase 2b international clinical trial in patients with soft tissue sarcomas. Of six patients who have completed four cycles with INNO-206 at the maximum tolerated dose in the Phase 1b/2 clinical trial, two patients have exhibited a partial tumor response (greater than 30% tumor shrinkage) and four patients have stable disease. Unexpectedly, a large, painful oral sarcoma that caused difficulty eating in one patient was greatly reduced following a single INNO-206 treatment. Common side effects reported to date from the Phase 1b/2 trial include low neutrophil (white blood cell) and platelet counts, minor mouth ulcers and mild nausea, which are expected side effects of doxorubicin.

In December 2011, CytRx initiated its international Phase 2b clinical trial to evaluate the preliminary efficacy and safety of INNO-206 as a first-line treatment in patients with soft tissue sarcoma who are ineligible for surgery. The Phase 2b clinical trial will provide the first direct clinical trial comparison of INNO-206 with native doxorubicin, which is dose-limited due to toxicity, as a first-line therapy.

INNO-206 has been granted orphan drug designation by the Office of Orphan Product Development of the U.S. Food and Drug Administration (FDA) for the treatment of patients with soft tissue sarcomas and pancreatic cancer.

Tamibarotene (formerly known as TM-411, TOS-80T, Am-80, and INNO-507) is an orally available, rationally designed, synthetic retinoid compound which was designed to potentially avoid toxic side effects by binding to its molecular target more selectively than all trans-retinoic acid (ATRA), the current first line treatment for APL. CytRx holds the North American and European rights to tamibarotene as a treatment for Non-small-cell lung cancer and APL, among other indications.

In December 2010, CytRx initiated an international Phase 2b clinical trial in patients with advanced non-small-cell lung cancer (NSCLC). In this randomized clinical trial, patients with advanced NSCLC are treated with paclitaxel plus carboplatin and either tamibarotene or placebo. The primary objective of this trial is to determine the objective response rate (complete and partial responses) and progression-free survival. Secondarily, the trial will evaluate overall survival, quality-of-life and examine the pharmacokinetics of tamibarotene in this population, among other measures.

A clinical trial conducted by Arrieta et al. and published in the peer-reviewed Journal of Clinical Oncology (June 17, 2010) compared ATRA added to a regimen of paclitaxel plus cisplatin to a regimen of paclitaxel plus cisplatin alone as a treatment for patients with advanced NSCLC. The group administered ATRA plus the chemotherapeutical agents showed improved response rates of 55.8% versus 25.4%, and increased progression-free survival of 8.9 months versus 6.0 months. Median overall survival was increased from 9.5 months to 23.5 months when ATRA was added to the above chemotherapy regimen, representing a 14-month median extension of life.

There is currently a Special Protocol Assessment (SPA) in place with the FDA for a Phase 2 registration clinical trial, known as STAR-1, which is evaluating the efficacy and safety of tamibarotene as a third-line treatment for APL. CytRx reported that, of the 11 patients enrolled in the STAR-1 trial to date, three (27%) achieved a hematologic complete response, and four (36%) a morphologic leukemia-free state.

The FDA has granted Orphan Drug Designation for APL and Fast Track Designation for the use of tamibarotene in patients with relapsed or refractory APL following treatment with all-trans retinoic acid (ATRA) and arsenic trioxide. In addition, tamibarotene has been granted orphan medicinal product status by the European Medicines Agency for the treatment of APL. The efficacy of orally-administered tamibarotene was demonstrated in two Phase 2 studies conducted in Japan in a total of 63 Japanese subjects with APL. The overall complete response rate in these subjects was 60%. In subjects experiencing their first relapse, the overall complete response rate was 81%.

Tamibarotene has also showed statistically significant anti-tumor activity in animal trial for multiple myeloma, an incurable malignant tumor of the plasma cells of bone marrow. CytRx retains an option to expand its licenses for the use of tamibarotene in other fields in oncology, including multiple myeloma, myelodysplastic syndrome and certain solid tumors in the U.S., and multiple myeloma, myelodysplastic syndromes and solid tumors other than hepatocellular carcinoma in Europe.

Bafetinib (formerly known as NS-187) is a potent, orally available, rationally designed, Bcr-Abl, Lyn and Fyn kinase inhibitor, which was being developed as a third-line treatment for patients with CML and certain forms of acute myeloid leukemia (AML) that are refractory or intolerant of other approved treatments.

The Company is evaluating bafetinib in the ENABLE Phase 2 clinical trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL), and recently announced that results from that clinical trial demonstrated bafetinib's clinical activity and preliminary safety in patients with relapsed or refractory B-CLL. CytRx plans to seek a partner for further development of bafetinib, and holds rights to bafetinib in all territories except Japan.