CytRx provides update on RNAi-based drug discovery programs - progress made toward developing...

CytRx Corporation (Los Angeles, CA) reported substantial progress in its drug discovery programs aimed at using its proprietary RNA interference (RNAi) technologies to develop novel molecular medicines to treat obesity and type 2 diabetes.

"During the past year, CytRx-sponsored research

programs have discovered and validated approximately 30 new type 2 diabetes and obesity drug targets through the use of our high throughput functional RNAi screening platform. This event supports our continued evolution as a leader in the field of RNAi-based drug discovery in identifying and developing potential treatments for these metabolic diseases," said Steven A. Kriegsman, President and CEO of CytRx.

CytRx reported the following developments in its RNAi-based drug discovery programs:

* Extension of its high throughput RNAi screening platform to include muscle and liver cells in addition to fat cells. CytRx is utilizing highly efficient, parallel RNAi-based gene silencing in these cells to identify and validate novel targets that function in insulin signaling and other metabolic pathways.

* Progress with its RIP140 RNAi therapeutics program. CytRx research has shown that suppression by gene deletion or small interfering RNA (siRNA) of RIP140, which is a nuclear hormone co-repressor that regulates fat accumulation, results in the acceleration of fat burning in animals and fat cells. Depletion of RIP 140 in vivo can improve responsiveness to insulin under circumstances such as a high fat diet, where normal animals develop insulin resistance and type 2 diabetes.

* Advancement of two proprietary small molecule leads against novel upstream AMP activated protein kinase (AMPK) targets into proof of concept animal experiments. AMPK is a well known, highly validated pathway of fatty acid oxidation. Increased recruitment of the AMPK signaling system, either due to exercise or pharmaceutical activators, may be effective in lowering fatty acids and correcting insulin resistance in patients with forms of impaired glucose tolerance.

* Submission of United States & European patent applications covering the use of regulators of its upstream AMPK targets to treat type 2 diabetes and obesity.

* Identification of multiple promising new chemical entities against targets that have never before been linked with type 2 diabetes

Mark A. Tepper, Ph.D., Senior Vice President of Drug Discovery and head of CytRx's laboratory in Worcester Massachusetts said, "We have continued to develop our RNAi screening platform by expanding its application to different metabolic tissues and implementing rapid in vivo RNAi target validation methods. Using this platform, we have shown that we can quickly identify and validate novel drug targets, screen them for small molecule inhibitors and show that these inhibitors are active in the original functional assay. With this platform, we plan to continue to build a pipeline of molecular medicines against multiple novel drug targets for treating obesity and type 2 diabetes."

"We are already in a Phase II clinical trial with our lead small molecule drug candidate arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and a Phase I trial with our HIV vaccine," added Mr. Kriegsman. "We look forward to entering the clinic with drug candidates derived from our RNAi-based drug discovery program, which represents an important step toward the development of next generation medicines that address the large, underserved obesity and type 2 diabetes markets."

Obesity has reached epidemic proportions. The World Health Organization (WHO) reports that worldwide more than 1 billion adults are overweight and at least 300 million of them are clinically obese. WHO cites overweight and obesity as major contributors to the growing incidence of chronic diet-related diseases and disabilities, including type 2 diabetes, cardiovascular disease, hypertension and stroke, and certain forms of cancer. According to the Journal of the American Medical Association, obesity-related deaths rose 33% to an estimated 400,000 between 1990 and 2000. A recent Rand study found that by 2020, approximately one in five healthcare dollars spent on people aged 50 to 70 will be due to obesity-related disabilities, if the current trend of overeating and inactivity continues.

CytRx is a biopharmaceutical research and development company engaged in the development of high value human therapeutics. The company owns three clinical-stage compounds based on its small molecule "molecular chaperone" co-induction technology (CCI), as well as a targeted library of 500 small molecule CCI analogs. CytRx has initiated a Phase II clinical trial with its lead CCI small molecule product candidate arimoclomol for the treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Arimoclomol has received Orphan Drug status and Fast Track designation from the United States Food and Drug Administration. CytRx has previously announced that a novel polyvalent HIV DNA + protein vaccine exclusively licensed to CytRx and developed by researchers at UMMS and Advanced BioScience Laboratories, and funded by the National Institutes of Health, demonstrated very promising interim Phase I clinical trial results that indicate its ability to produce potent antibody responses with neutralizing activity against multiple HIV viral strains. CytRx also has a broad-based strategic alliance with UMMS to develop novel compounds in the areas of ALS, obesity, type 2 diabetes and cytomegalovirus using RNAi technology. The company has a research program with Massachusetts General Hospital, Harvard University's teaching hospital, to use RNAi technology to develop a drug for the treatment of ALS. CytRx Drug Discovery division (Worcester, MA) focuses on the use of RNAi technologies to develop small molecule and RNAi therapeutics to treat obesity and type 2 diabetes.

CytRx Corporation

+1-310-826-5648

www.cytrx.com