CytRx Scientific Advisory Board Member Dr. Craig C. Mello Awarded Nobel Prize in Medicine for RNAi Discovery
Tuesday October 3, 8:51 am ET
LOS ANGELES--(BUSINESS WIRE)--CytRx Corporation (Nasdaq:CYTR - News) today applauded the announcement that its Scientific Advisory Board member Craig C. Mello, Ph.D., together with Andrew Fire, Ph.D., was awarded the Nobel Prize in medicine for discovery of the use of double-stranded RNA for gene silencing, a technology that has come to be known as RNA interference (RNAi). Drs. Mello and Fire acted as key inventors on the first approved RNAi patent, which is licensed to CytRx on a non-exclusive basis.
As a member of the CytRx Scientific Advisory Board, Dr. Mello acts in an advisory capacity to assist the Company to develop RNAi therapeutics for specific diseases.
Dr. Mello is the Blais University Chair and Distinguished Professor of Molecular Medicine and a Howard Hughes Medical Institute Investigator at the University of Massachusetts Medical School, in Worcester, Mass. In this capacity, Dr. Mello studies both the biochemical pathway responsible for RNAi and the regulation of gene expression. He has numerous publication credits including 16 in the prestigious journals Science, Nature, Cell, and the Proceedings of the National Academy of Sciences.
"We extend our sincerest congratulations to Drs. Mello and Fire for receiving this celebrated award and are delighted that the Nobel committee has recognized the potential of this highly novel approach that has implications over a broad range of catastrophic diseases," said President and CEO Steven A. Kriegsman. "RNAi could offer an entirely new paradigm in disease treatment by regulating the effect of specific diseases, making it a valuable tool in drug discovery, as well as a direct therapeutic. At CytRx, we are involved in drug discovery using RNAi targeting for major global health concerns that include obesity and type 2 diabetes."
CytRx uses RNAi to help screen and identify classical, orally-available small molecule drugs. In its obesity and type 2 diabetes programs, CytRx is using its proprietary high throughput RNAi-based screening technology to screen the more than 2,000 candidate genes that may be involved in diabetes and obesity in order to rapidly validate numerous drug targets in the most critical pathways which regulate metabolism. During the past year, CytRx-sponsored research programs have discovered and validated approximately 30 new type 2 diabetes and obesity drug targets. CytRx also is exploring the utility of RNAi for direct therapeutic applications when technically feasible.
"We strongly believe in the value of RNAi technology, which has prompted us to pursue a plan, subject to obtaining necessary funding, to transfer all of our RNAi therapeutics assets into a newly-formed subsidiary to accelerate the development and commercialization of drugs based on this platform," added Mr. Kriegsman. "We plan to continue using RNAi gene silencing technology as a drug discovery tool to facilitate our small molecule drug discovery program."
RNA is a constituent of all living cells and many viruses, consisting of a long, usually single-stranded chain of alternating phosphate and ribose units with the bases adenine, guanine, cytosine, and uracil bonded to the ribose. The structure and base sequence of RNA is used by the cell as a messenger (mRNA) to be used as an "instruction manual" for the synthesis of a specific protein. RNAi technology uses short double-stranded RNA, or dsRNA, molecules to silence targeted genes and, as a result, is commonly referred to as "gene silencing." RNAi has been shown to effectively silence targeted genes within living cells with great specificity and potency. The end result is the destruction of the specific mRNA, thus silencing that gene and the protein that the mRNA would normally be used to synthesize.
About CytRx Corporation
CytRx Corporation 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. In September 2006, CytRx reported reaching the primary endpoints of safety and tolerability from a Phase IIa trial with its lead small molecule product candidate arimoclomol for the treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Also in September 2006 CytRx announced receipt of $24.5 million in a non-dilutive agreement with the privately funded ALS Charitable Remainder Trust to fund continued arimoclomol development for the treatment for ALS in return for one percent royalty payment from potential worldwide sales of arimoclomol for the treatment of ALS to The Greater Los Angeles Chapter of The ALS Association. Arimoclomol has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration.
CytRx has previously announced that a novel polyvalent HIV DNA + protein vaccine exclusively licensed to CytRx, developed by researchers at the University of Massachusetts Medical School (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 (CMV) 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, located in Worcester, Mass., focuses on the use of RNAi technologies to develop small molecule and RNAi therapeutics to treat obesity and type 2 diabetes. For more information, visit CytRx's Web site at www.cytrx.com.
This press release may contain forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to the expected timing, scope and results of our clinical development and research programs, including the initiation of clinical trials, and statements regarding the potential benefits of our drug candidates and potential drug candidates. 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 early stage of CytRx's RNAi, diabetes, obesity, cytomegalovirus and ALS research, the need for future clinical testing of any RNAi-based product candidates and small molecules that may be developed by CytRx using RNAi screening methods, the significant time and expense that will be incurred in developing any of the potential commercial applications for CytRx's RNAi technology or small molecules, uncertainties related to regulatory approvals for clinical testing and the scope of the clinical testing that may be required by regulatory authorities for its molecular chaperone co-induction drug candidates, including arimoclomol, and other products, and the timing and outcomes of those tests, risks relating to the enforceability of any patents covering CytRx's products and to the possible infringement of third party patents by those products, and the impact of third party reimbursement policies on the use of and pricing for CytRx's products. Additional uncertainties and risks are described in CytRx's most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K filed since the date of the last Form 10-K. 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.