CytRx Initiates Phase IIb Efficacy Clinical Trial with Arimoclomol for Treatment of ALS
- Arimoclomol dose designed to optimize clinical benefit -
LOS ANGELES--(BUSINESS WIRE)-- CytRx Corporation (NASDAQ:CYTR) today announced that it has initiated screening volunteers in its Phase IIb clinical trial with arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease). The double-blind, placebo-controlled trial is expected to require nine months to complete enrollment of approximately 390 ALS volunteers at 30 to 40 U.S. and Canadian clinical sites, and will evaluate the safety and efficacy of 400 mg arimoclomol administered orally three times daily for a nine-month period. In order to maximize the amount of long-term safety and efficacy data from this trial, CytRx will continue double-blind arimoclomol administration for an additional nine months after collection of primary endpoint data to collect additional data on secondary endpoints.
"We have taken multiple steps to maximize the probability of measuring efficacy in the Phase IIb clinical trial, including administering arimoclomol at a significantly higher dose than that used in our Phase IIa trial," said Jack Barber, Ph.D., CytRx's Chief Scientific Officer. "Our decision to move forward with this higher dose was based on a large body of preclinical results and encouraging safety data obtained from two clinical studies recently completed in healthy volunteers."
"We are pleased to continue clinical testing of arimoclomol for the treatment of ALS, a debilitating disease that currently lacks effective therapeutic treatment," said CytRx's President and CEO Steven A. Kriegsman. "The timely initiation of this trial keeps us on track to report primary efficacy results in approximately 18 months," added Mr. Kriegsman.
About the Phase IIb Trial in ALS
In the randomized Phase IIb clinical trial in ALS, two-thirds of the 390 enrolled ALS volunteers will receive 400 mg of arimoclomol in capsule form three-times daily and the remaining volunteers will receive placebo administered three-times daily. The Phase IIb trial's primary endpoint will be efficacy after nine months of dosing as determined by the Revised ALS Functional Rating Scale (ALSFRS-R), which is used to determine a patient's capacity and independence in 13 functional activities. Secondary Phase IIb efficacy endpoints include ALSFRS-R at 18 months and survival at 18 months, as well as pulmonary function, muscle strength, Motor Unit Number Estimate (MUNE) (a non-invasive test that estimates the number of functioning motor units), and quality of life, all of which will be measured at nine and 18 months.
The trial is designed in two tiers, both of which will receive the same dose for the same length of time. The first tier will enroll 24 ALS volunteers and will have a four-week safety lead-in phase designed with weekly clinical monitoring to assure that the safety previously observed in healthy volunteers is also observed in ALS volunteers. After the initial four weeks, dosing will continue uninterrupted and clinical monitoring will be reduced for the remainder of the study. Safety data from the first four-week period of Tier I will be reviewed by an independent data monitoring committee (IDMC) and, if no serious safety issues are identified, the remainder of the ALS volunteers will be enrolled without this safety lead-in phase (Tier II). With the exception of the first four weeks of Tier I, all 390 ALS volunteers enrolled in the Phase IIb trial will be followed every four weeks for the first nine months and every eight weeks for the remaining nine month trial period.
The Clinical Investigators
CytRx has assembled a team of renowned clinicians at clinical sites throughout the U.S. and Canada to conduct the Phase IIb clinical trial. Additional information concerning the trial, its clinical investigators and clinical sites is available by searching for “arimoclomol” on the website www.clinicaltrials.gov.
Recent Clinical Trial Data on Arimoclomol for ALS
In June 2007, CytRx reported promising data from the six-month, open-label extension of its Phase IIa clinical trial with arimoclomol in ALS volunteers. Arimoclomol at the 100 mg dose level administered orally three times daily appeared to be safe and well-tolerated throughout the extended period. Further, the rate of decline of the ALSFRS-R and vital capacity in the arimoclomol-treated group compared favorably to a matched historical control. While difficult to draw definitive conclusions without a concurrent placebo control group, CytRx observed a decrease in the rate of decline of 21% for ALSFRS-R, 8.0% for vital capacity, 23% for total body weight and 20% for body mass index when compared with an historical control.
Also in June 2007, CytRx announced clinical data from a short-term, rising multiple dose study in 40 healthy volunteers indicating that arimoclomol was safe and well-tolerated at doses as high as 600 mg administered three times daily over a seven-day period. Last month, the Company reported results from a double-blind, placebo-controlled clinical trial with 16 healthy volunteers indicating that arimoclomol was safe and well-tolerated at the 400 mg dose administered orally three times daily throughout a 28-day trial period.
About Arimoclomol
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.
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Orphan Drug status to arimoclomol for the treatment of ALS. Arimoclomol has also been granted orphan medicinal product status for the treatment of ALS from the European Medicines Agency.
About ALS
ALS is a progressive degeneration of the brain and spinal column nerve cells that control the muscles that allow movement. ALS is one of the most common neuromuscular diseases worldwide, affecting an estimated 120,000 people of all races and ethnic backgrounds worldwide. 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 Association more than 5,600 people in the U.S. are diagnosed with ALS annually and an estimated 30,000 Americans have ALS at any given time. According to the National Institute of Neurological Disorders and Stroke, most ALS patients die within three to five years after the onset of symptoms.
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 volunteers with ALS. In December 2007, the Company initiated its Phase IIb clinical trial with arimoclomol in ALS. The Company 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™ RNAi, one of the most widely used second-generation RNAi research products.
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. 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 IIb clinical trial for arimoclomol, uncertainties regarding regulatory approvals for current and future clinical testing of arimoclomol and the scope of the clinical testing that may eventually be required by regulatory authorities for arimoclomol, uncertainties related to the comparison of clinical data to historical controls in the absence of concurrent placebo controls, the significant time and expense that will be incurred in developing any of the potential commercial applications for arimoclomol and the potential need for additional capital to fund the development of arimoclomol, 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.
CytRx Corporation CEOcast, Inc. Dan Schustack, 212-732-4300 dschustack@ceocast.com
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