CytRx's Arimoclomol Demonstrates Statistically Significant Neurorestorative Results in a Preclinical Embolic Stroke Trial
Data Supports Previous Preclinical Results Indicating Improvement in Stroke Recovery Following Oral Arimoclomol Administration
LOS ANGELES--(BUSINESS WIRE)--Mar 16, 2009 - <!-- cpurl -->CytRx<!-- /cpurl --> Corporation (NASDAQ:CYTR), a biopharmaceutical company engaged in the development and commercialization of human therapeutics, today announced data from an animal stroke trial indicating that treatment with its molecular chaperone amplifier drug candidate, <!-- ppurl -->arimoclomol<!-- /ppurl -->, initiated at either six, 10, 24 or 48 hours post-stroke, demonstrated neurorestorative ability based on multiple behavioral tests measuring motor and sensory recovery.
“We believe the data from this trial confirm and extend previous highly favorable preclinical results indicating that arimoclomol has the potential to restore brain function following stroke, even when administered well beyond the three-hour therapeutic window of t-PA, the currently approved treatment for stroke,” said CytRx President and CEO Steven A. Kriegsman. “If efficacious in stroke patients, we believe arimoclomol would represent a major breakthrough in improving their quality of life. Due to the compelling nature of this opportunity we have had pre-IND discussions with the U.S. Food and Drug Administration (FDA) concerning a possible IND application necessary to initiate a Phase 2 human stroke trial with arimoclomol. We are also working with the FDA to potentially resume Phase 2 testing with arimoclomol as a therapeutic treatment for ALS, and we do not believe that the current clinical hold on that trial will impact the ability to initiate human testing in stroke recovery. We continue to compile a very compelling data set with arimoclomol in stroke recovery as we actively seek a partner to sponsor future clinical development of this promising drug candidate.”
Stroke is caused by a lack of blood flow, most usually caused by an embolism (blood clot), and results in a lack of oxygen to regions of the brain. In the United States alone, stroke affects about 700,000 persons each year. On average, every 40 seconds someone in the United States has a stroke, and stroke is the leading cause of serious, long-term disability, according to the American Heart Association. Currently, t-PA is the only FDA-approved treatment and must be administered within three hours of the initiation of stroke.
CytRx scientists used the embolic animal model in the stroke trial as it closely mimics part of the typical human stroke pathology, particularly the clot formation in cerebral vessels that stop or slow blood flow to effect damage. Over time these clots in the embolic animal model, as in humans, can spontaneously dissolve, resulting in restoration of partial blood flow to the damaged regions. This rapid “reperfusion” of oxygen-rich blood is known to contribute to some of the long-term damage that occurs during stroke.
In the study, stroke was induced in 10 rats in each group through the introduction of blood clots into the middle cerebral artery, blocking blood flow to parts of the brain and causing cerebral oxygen deprivation. These stroke-induced rats then received an oral dose of arimoclomol or a control substance daily for 28 days after stroke with the initial dose being withheld until 6, 10, 24, or 48 hours after stroke. Sensorimotor impairment was evaluated by three different tests given before stroke and again at intervals after stroke.
In one functional test, a statistically significant improvement in stroke recovery was observed for all arimoclomol-treated groups regardless of when treatment was initiated. In the two other tests, a statistically significant improvement was observed in the groups treated at six, 10 and 24 hours after stroke was induced. The assessment of the remaining group, treated at 48 hours after stroke, showed a trend for improvement that did not reach statistical significance in these latter two tests.
“t-PA works as a fibrolytic ('clot buster'), to restore blood flow as quickly as possible,” stated Jack Barber, Ph.D., CytRx's Chief Scientific Officer. “Arimoclomol instead apparently acts by restoring (repairing) the proper functioning of the nerve cells previously damaged by oxygen deprivation, allowing it to be therapeutic even when administered at later times after stroke initiation. The observation that arimoclomol showed therapeutic benefit even after 48 hours in this embolic model of stroke gives us even more confidence in the drug's potential in this indication,” added Dr. Barber.
CytRx previously reported positive results from animal permanent stroke studies indicating arimoclomol significantly accelerated the recovery of sensory and motor function in an experimental rat model of stroke, even when treatment was withheld as long as 48 hours after stroke was induced.
Arimoclomol, a molecular chaperone regulator drug candidate, is being considered as a treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and stroke recovery. Arimoclomol has been studied in seven Phase 1 and two Phase 2 clinical trials without any significant adverse events. CytRx's Phase 2b clinical trial with arimoclomol as a treatment for ALS was placed on clinical hold by the FDA in January 2008, unrelated to any data generated by human studies, and additional preclinical toxicology studies are underway to resolve this issue. CytRx expects early in the second quarter to submit animal toxicology data to the FDA relating to the arimoclomol clinical hold. Given this timeline, the Company anticipates a response from the FDA in the third quarter.
About Molecular Chaperone Regulation
CytRx is a leader in molecular chaperone regulation technology. The Company currently has two orally administered, clinical-stage, drug candidates and recently discovered a series of additional compounds that may provide a pipeline for additional drug candidates. The Company's drug candidates are believed to function by regulating a normal cellular protein repair pathway through the activation or inhibition of "molecular chaperones." Because damaged proteins are thought to play a role in many diseases, activation of molecular chaperones that help to reduce the accumulation of misfolded proteins may have therapeutic efficacy in a broad range of disease states. Similarly, CytRx believes that the inhibition of molecular chaperones that normally help protect cancer cells from toxic misfolded proteins may result in the selective destruction of cancer cells.
About CytRx Corporation
CytRx Corporation is a biopharmaceutical research and development company engaged in the development of high-value human therapeutics. The CytRx drug development pipeline includes programs in clinical development for cancer indications, including <!-- ppurl -->tamibarotene<!-- /ppurl --> in a registration study for the treatment of acute promyelocytic leukemia (APL). CytRx is developing two drug candidates based on its industry-leading molecular chaperone technology, which aims to repair or degrade misfolded proteins associated with disease. The Company owns and operates a research and development facility in San Diego. CytRx also maintains a 45% equity interest in publicly traded <!-- cpurl -->RXi Pharmaceuticals<!-- /cpurl -->, Inc. (NASDAQ: RXII). For more information on the Company, visit www.cytrx.com.
This press release contains 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 relating to the predictive power of this or any other animal model of stroke recovery to humans, the outcome or results of any future pre-clinical or clinical testing of arimoclomol for stroke recovery, uncertainties related to the impact of the FDA's clinical hold on our clinical program for arimoclomol for the treatment of amyotrophic lateral sclerosis on the timing and ability to initiate further clinical development for stroke recovery, the risk that any requirements imposed on our planned clinical trial design for stroke recovery by the FDA as a result of the concerns expressed in their clinical hold of our ALS program might adversely affect our ability to demonstrate that arimoclomol is efficacious in treating stroke patients, risks related to CytRx's ability to enter into partnerships to advance the clinical development of arimoclomol, risks related to CytRx's need for additional capital or strategic partnerships to fund its ongoing working capital needs and development efforts, risks related to the future market value of CytRx's investment in RXi and the liquidity of that investment, and the risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. 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: Legend Securities, Inc.
Posted: March 2009