Healthcare Industry News: RNAi therapeutic
News Release - October 31, 2006
CytRx's Arimoclomol Granted Orphan Drug Designation in European Union for Treatment of ALSLOS ANGELES--(HSMN NewsFeed)--CytRx Corporation (Nasdaq:CYTR ), a biopharmaceutical company engaged in the development and commercialization of human therapeutics, today announced that its lead drug candidate arimoclomol has been granted orphan medicinal product status for the treatment of amyotrophic lateral sclerosis (ALS) by the European Commission. This orphan medicinal product status grants CytRx market exclusivity in the European Union (EU) for 10 years in the event that arimoclomol receives marketing approval, and provides for incentives such as reduced fees for protocol assistance and scientific advice.
"We appreciate the European Commission's recognition of arimoclomol as a potential drug for ALS," said CytRx President and CEO Steven A. Kriegsman. "This is a life-threatening and debilitating condition for which there is currently no effective treatment and we believe that arimoclomol is based on a novel mechanism of action that may hold a key in ALS therapy."
CytRx Senior Vice President of Drug Development Jack Barber, Ph.D., commented, "We intend ultimately to pursue product approval for arimoclomol in ALS in the EU, which will require us to follow a modified regulatory process from that in the U.S. The granting of orphan medicinal product status provides us the mechanism with which to meet with the EMEA and other regulatory officials in the EU in pursuit of EU approval going forward."
The European Commission grants orphan medicinal product status to promote the innovation of drugs that are developed to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. Arimoclomol received orphan drug status from the U.S. Food and Drug Administration for the treatment of ALS in May 2005. Orphan drug status in the U.S. holds numerous potential benefits, including opportunities for grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, seven years of U.S. market exclusivity should the FDA grant marketing approval for the drug and an added mechanism for more frequent communication with the FDA.
Based on the Company's September 2006 announcement of meeting the primary safety and tolerability endpoints in a Phase IIa trial with arimoclomol as an ALS treatment, CytRx plans to initiate a Phase IIb trial in the U.S. and Canada in mid-2007, subject to FDA approval. The Company believes that positive efficacy and safety results from the Phase IIb clinical trial could be sufficient for arimoclomol product registration in the U.S. for this indication.
Arimoclomol is one of CytRx's three orally-administered, small molecule compounds. This small molecule drug candidate is believed to function by stimulating a normal cellular protein repair pathway through the activation of "molecular chaperones." Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.
ALS is a progressive degeneration of the brain and spinal column nerve cells that control the muscles that allow movement. Over a period of months or years, ALS causes increasing muscle weakness, inability to control movement and problems with speaking, swallowing and breathing. According to the ALS Survival Guide, 50% of ALS patients die within 18 months of diagnosis and 80% die within five years. More than 120,000 people are living with ALS worldwide.
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 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 a one percent royalty from potential worldwide sales of arimoclomol for the treatment of ALS. The Greater Los Angeles Chapter of The ALS Association is the charitable beneficiary of the ALS Charitable Remainder Trust.
CytRx has a broad-based strategic alliance with the University of Massachusetts Medical School 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's Drug Discovery division, located in Worcester, Massachusetts, 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 contains forward-looking statements, including those related to the results and achievements of CytRx's clinical Phase IIa trial for arimoclomol, which involve known and unknown risks and uncertainties that may cause actual future results and achievements of CytRx to be materially different from those expressed or implied by these forward-looking statements. In particular, the results and achievements described may not be supported by further analysis of the Phase IIa trial data or by the results of any subsequent clinical trials. These risks and uncertainties also include risks and uncertainties regarding CytRx's ability to obtain regulatory approvals for further clinical testing of arimoclomol, the scope of clinical testing that may be required by regulatory authorities and the timing and outcome of further clinical trials. Additional uncertainties and risks regarding regulatory approval of CytRx's drug candidates, financing needs, reliance upon strategic relationships, intellectual property protections and other relevant matters are described in CytRx's reports filed with the Securities and Exchange Commission, including 10-K, 10-Q and 8-K reports. All forward-looking statements in this press release are based upon information available to CytRx as of the date of this press release. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
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