CytRx Provides Arimoclomol and Iroxanadine Clinical Trial Updates
"We are delighted to report that we continue on track with our plans for clinical testing in three indications that represent large market opportunities," said CytRx's President and CEO Steven A. Kriegsman.
Arimoclomol in ALS
CytRx is on course to initiate a Phase IIb efficacy trial with arimoclomol for the treatment of ALS later this year. The Phase IIb study is designed as a double-blind, placebo-controlled trial and is expected to include 390 ALS patients enrolled at 30 to 40 clinical sites in the U.S. and Canada. Two-thirds of enrolled patients will receive 400 mg of arimoclomol in capsule form three times daily and the remaining patients will receive placebo administered three times daily. The Phase IIb trial's primary endpoint will be efficacy at nine months following enrollment as determined by the Revised ALS Functional Rating Scale (ALSFRS-R), which measures overall functional capacity. The Company expects to report primary endpoint data approximately 18 months following the initiation of patient enrollment. In order to maximize the amount of long term efficacy data obtained from these volunteers CytRx has recently decided to continue the trial for an additional 9 months after collection of primary endpoint data. Secondary Phase IIb efficacy endpoints will include survival at 18 months, as well as pulmonary function, muscle strength and quality of life, which will be measured at nine and 18 months following enrollment.
The decision to dose at 400 mg three times daily, well above the highest arimoclomol dose of 100 mg three times daily administered in the Phase IIa ALS study, was determined in part by encouraging safety data obtained from a multiple dose clinical trial in healthy volunteers announced in June 2007. A follow-up clinical trial in healthy volunteers, also announced in June and designed to provide longer-term safety and tolerability data at the 400 mg dose, is nearing completion. This double-blind, placebo-controlled study includes 16 healthy volunteers: four receiving a placebo capsules without drug and 12 receiving 400 mg of arimoclomol three times daily for 28 days. To date 14 of the 16 volunteers have completed the study. The results of this safety study will be reported early in the fourth quarter of this year, prior to the planned initiation of the Phase IIb efficacy trial.
Based on consultation with the U.S. Food and Drug Administration (FDA), CytRx is planning a second efficacy trial for ALS, possibly to be executed in parallel with the Phase IIb trial, to provide additional data to support an NDA submission for arimoclomol in this indication. The second efficacy trial will be powered to measure survival at 18 months, as well as to include supporting secondary efficacy endpoints, including ALSFRS-R data. CytRx expects to provide additional detail on the second efficacy trial following initiation of the Phase IIb trial.
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, resulting in the inability to control movement and problems with speaking, swallowing and breathing. According to the National Institute of Neurological Disorders and Stroke, most ALS patients die within 3 to 5 years after the onset of symptoms
Arimoclomol for Stroke Recovery
CytRx continues on plan to initiate a Phase II clinical trial with arimoclomol in recovering stroke volunteers in the first half of 2008, subject to FDA clearance. The decision to pursue this clinical program was supported in part by favorable results announced in April 2007 from a stroke functional recovery study that suggested that arimoclomol in a rat model accelerated the repair of neurological damage caused by stroke even when treatment was delayed up to 48 hours following stroke inducement. Most other stroke drug candidates do not repair the damage caused by stroke, but instead attempt to limit the initial damage caused by stroke, and are generally required to be administered no later than three hours after stroke in order to show significant therapeutic benefit in rats. The flexibility to administer arimoclomol for delayed intervention is considered by CytRx to be a substantial advantage compared with currently-marketed drugs for the treatment of stroke. If proven efficacious, arimoclomol could substantially penetrate the $58 billion stroke market.
Iroxanadine in Diabetic Foot Ulcers
CytRx also continues on track with its previously announced plan to move into a Phase II clinical trial with its next clinical pipeline candidate iroxanadine for the treatment of diabetic foot ulcers in the first half of 2008, subject to FDA clearance. CytRx announced in May 2007 that iroxanadine dramatically and reproducibly accelerated the healing of skin wounds in diabetic animals.
According to the American Diabetes Association, 20.8 million Americans, or 7% of the population, have diabetes. The National Institutes of Health (NIH) estimates that 15% of these patients will experience a foot ulcer in their lifetime. Costs for the care of ulcerated wounds in the United States have been estimated at $5 billion.
About Molecular Chaperone Co-induction Technology
Arimoclomol and iroxanadine are two of CytRx's three orally-administered, small molecule compounds. These small molecule drug candidates are believed to function by stimulating a normal cellular protein repair pathway through the activation of "molecular chaperones." Since damaged toxic 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 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 that arimoclomol was shown to be safe and well tolerated at all three doses tested in its Phase IIa clinical trial in patients with ALS. The Company plans to enter a Phase IIb clinical trial with arimoclomol in ALS in 2007, subject to FDA clearance. The FDA has granted Fast Track designation and Orphan Drug status to arimoclomol for the treatment of ALS, which has also been granted orphan medicinal product status for the treatment of ALS by the European Commission. The Company has announced plans to commence a Phase II clinical trial for arimoclomol in stroke recovery in the first half of 2008, subject to FDA clearance. The Company has also announced plans to commence a Phase II clinical trial with its next drug candidate, iroxanadine, for diabetic foot ulcers in the first half of 2008, subject to FDA clearance. CytRx has recently opened a research and development facility in San Diego. For more information on the Company, visit www.cytrx.com.
About RXi Pharmaceuticals Corporation
Worcester, Massachusetts-based RXi Pharmaceuticals Corporation, a majority-owned subsidiary of CytRx, is a biopharmaceutical research and development company that focuses on developing RNAi-based therapeutics for the treatment of human disease. RXi's initial focus is on neurodegenerative diseases, oncology, type 2 diabetes and obesity. RXi has licenses to a diverse series of early patents and patent applications that were filed from 1998 to 2006 in the areas of RNAi target sequences, RNAi chemistry and RNAi delivery. The Company was founded by CytRx and RNAi pioneers Craig Mello, Ph.D., 2006 Nobel Laureate for discovering RNAi and inventing RNAi therapeutics; Tariq M. Rana, Ph.D., inventor of fundamental technology for stabilizing RNAi and of RNAi nanotransporters; Greg Hannon, Ph.D., discoverer of RNAi mechanism (RISC) and short hairpin RNAi (shRNAi); and Michael Czech, Ph.D., a leader in the application of RNAi to diabetes and obesity. RXi's CEO, Tod Woolf, Ph.D., previously co-invented and commercialized STEALTH(TM) RNAi, one of the most widely used second-generation RNAi research products. For more information on RXi, please visit www.rxipharma.com.
This press release may contain forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. 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 regarding regulatory approvals for future clinical testing of arimoclomol and iroxanadine, including CytRx's planned Phase IIb clinical trial for arimoclomol in ALS, and the scope of the clinical testing that may be required by regulatory authorities for arimoclomol and iroxanadine, uncertainties regarding the timing and amount of revenues, if any, that will be realized by CytRx from the commercialization of arimoclomol or iroxanadine, the significant time and expense that will be incurred in developing any of the potential commercial applications for arimoclomol or iroxanadine and the potential need for additional capital to fund the development of arimoclomol and iroxanadine, as well as other risks or uncertainties described in CytRx's most recently filed SEC documents, such as its most recent annual report on Form 10-K 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.
Dan Schustack, 212-732-4300
Posted: September 2007