Clinical Trial for ALS
Monday July 24, 8:30 am ET
Plans to Announce Final Results in Early 4th Quarter
LOS ANGELES--(BUSINESS WIRE)--July 24, 2006--CytRx Corporation (Nasdaq:CYTR - News), a biopharmaceutical company engaged in the development and commercialization of human therapeutics, today announced that the final patients have completed dosing and follow-up with arimoclomol in the Company's Phase IIa clinical trial for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Phase IIa patients were evaluated while receiving either placebo (a capsule without drug) or one of three dose levels of CytRx's lead product candidate arimoclomol three times daily for 12 weeks and during a 4-week follow-up period without drug. "This is an important milestone in arimoclomol's clinical development and moves us a step closer to our goal of commercializing drugs aimed at reducing human suffering," said Steven A. Kriegsman, President and CEO of CytRx. "Now that we have successfully completed the clinical aspect of the trial we can begin the process of data analysis."
The data will now be evaluated and analyzed under strict quality-controlled guidelines. CytRx expects to complete data analysis of the Phase IIa trial and announce final results early in the fourth quarter of this year.
Ten clinical centers across the U.S. participated in CytRx's double-blind, placebo-controlled Phase IIa trial. The primary endpoints of the Phase IIa trial were safety and tolerability. Secondary endpoints included a preliminary evaluation of efficacy using two widely accepted surrogate markers, the revised ALS Functional Rating Scale (ALSFRS-R), which is used to determine a patient's capacity and independence in 13 functional activities, and Vital Capacity (VC), an assessment of lung capacity. The Phase IIa trial was powered to detect only extreme responses in these two categories.
In January 2006, CytRx initiated an open-label extension of this Phase IIa trial allowing patients who complete the trial to continue treatment with arimoclomol at the highest dose level three times daily for up to an additional six months. The open-label extension is designed to provide additional safety and tolerability data in combination with the current Phase IIa trial. Approximately 99% of the patients who completed the Phase IIa trial elected to enroll in the open-label extension trial. It is anticipated that the open-label extension trial will be completed in January 2007.
Subject to approval from the U.S. Food and Drug Administration (FDA), CytRx intends to initiate a Phase IIb efficacy trial in the first half of 2007. This efficacy trial will be powered to detect more subtle efficacy responses and is expected to include a minimum of 390 ALS patients recruited from 30 clinical sites and is anticipated to take approximately 18 months to complete after enrollment begins. Given the severity of ALS and the lack of therapeutic treatment options, CytRx believes that positive efficacy and safety results from the Phase IIb clinical trial could be sufficient for arimoclomol product registration for this indication.
In March 2006, arimoclomol was cited as one of the "100 Great Investigational Drugs in Development" by the pharmaceutical industry magazine R&D Directions in its fifth annual report. According to the publication, the report "looks at compounds distinguishing themselves as innovative, first-in-class or clearly advanced compared with those on the market. Many of the compounds address unmet medical needs and were selected for inclusion in the list from the thousands being developed at large and small companies throughout the world."
The FDA has granted Fast Track designation and Orphan Drug status to arimoclomol for the treatment of ALS. Fast Track is designed to facilitate the development and expedite the review of a new drug that demonstrates the potential to address a significant unmet medical need for the treatment of a serious or a life-threatening condition. Orphan Drug status holds numerous potential benefits, including opportunities for grant funding towards 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.
About Arimoclomol
Arimoclomol 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.
About ALS
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. In the U.S., an estimated 30,000 people are living with ALS and
nearly 6,000 new cases are diagnosed annually, according to the ALS Association.
There are more than 120,000 people 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 (CCI), as well as a targeted library of 500 small molecule CCI analogs. CytRx has completed patient dosing in a Phase IIa clinical trial with its lead CCI small molecule product candidate arimoclomol for the treatment of ALS. Arimoclomol has received Orphan Drug status and Fast Track designation from the FDA. CytRx has previously announced that a novel polyvalent HIV DNA + protein vaccine exclusively licensed to CytRx and 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 .
Forward-Looking Statements 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 outcome and timing of CytRx's Phase II clinical trial for arimoclomol, including the open-label extension of that trial, uncertainties regarding regulatory approvals for future clinical testing of arimoclomol and the scope of the clinical testing that may be required by regulatory authorities for arimoclomol, uncertainties related to the early stage of CytRx's diabetes, obesity, cytomegalovirus and ALS research, the significant time and expense that will be incurred in developing any of the potential commercial applications for arimoclomol and the need for additional capital to fund the development of arimoclomol, 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.
Contact:CytRx Corporation
Ed Umali, 310-826-5648 ext. 309
eumali@cytrx.com
or
CEOcast, Inc.
Kevin Theiss, 212-732-4300
ktheiss@ceocast.com
Cormac Glynn, 212-732-4300
cglynn@ceocast.com