CytRx Corporation has reported substantial progress in its drug discovery programs aimed at using its proprietary RNA interference technologies to develop 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," said Steven A. Kriegsman, President and CEO of CytRx.
"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."
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 parallel RNAi-based gene silencing in these cells to identify and validate 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 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.
- Advancement of two proprietary small molecule leads against novel upstream AMP activated protein kinase (AMPK) targets into proof of concept animal experiments.
- Submission of U.S. & 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."