2005 - CytRx Corporation (Nasdaq: CYTR), a biopharmaceutical company engaged in the development and commercialization of human therapeutics primarily in the area of small molecules and ribonucleic acid interference (RNAi), today announced that after receiving notification from the U.S. Food and Drug Administration (FDA) yesterday,
it has initiated a Phase II clinical trial with its lead small molecule product candidate, arimoclomol, for the treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The Company reports that patient identification has begun at several clinical trial sites, and that patient enrollment will begin immediately.
"The start of our Phase II trial is a major event for CytRx and the many thousands who suffer daily from this deadly disease of the nervous system," said Steven A. Kriegsman, President and CEO of CytRx. "We are moving aggressively in our fight against ALS and we expect to rapidly enroll sufficient numbers of patients for this trial from our carefully selected clinical sites. Additionally, we have already received numerous inquiries about this trial from physicians and ALS patients."
"We plan to complete this portion of the Phase II program in the first half 2006," added Mr. Kriegsman.
Robert H. Brown, Jr, M.D., Professor of Neurology at Harvard Medical School, founder and director of the Cecil B. Day Laboratory for Neuromuscular Research at Massachusetts General Hospital (MGH), noted world authority in ALS, and co-principal investigator of the clinical trial, said,
"Arimoclomol is one of the most promising new neuroprotective drug candidates that I am aware of. It is thought to work through a novel mechanism that has produced very exciting results in several animal models. We are all delighted that we can now move forward with a trial of arimoclomol in ALS."
Louis Ignarro, Ph.D., Nobel Laureate in Medicine and member of CytRx's Board of Directors and CytRx's Scientific Advisory Board, said,
"If arimoclomol acts similarly in humans as it did in animals, it may inhibit the progression of ALS and potentially other neurodegenerative diseases such as Parkinson's and Huntington's." Dr. Ignarro also serves as the Jerome J. Bezler Distinguished Professor of Pharmacology in the Department of Molecular and Medical Pharmacology at the UCLA School of Medicine.
Clinical Development Plan
The current Phase II clinical trial (Phase IIa) is a multicenter, double-blind, placebo-controlled study of patients with ALS. 80 ALS patients at eight to ten centers across the U.S. will be included in the clinical trial. Patients will receive either placebo (a capsule without drug), or one of three dose levels of arimoclomol capsules three times daily, for a period of 12 weeks.
The primary endpoints of this Phase II trial are safety and tolerability. Secondary endpoints include a preliminary evaluation of efficacy using two widely accepted surrogate markers, the revised ALS Functional Rating Scale (ALSFRS-R), which is used to determine patient's capacity and independence in 13 functional activities, and Vital Capacity (VC), an assessment of lung capacity. The trial is powered to monitor only extreme responses in these two categories.
The subsequent Phase II trial (Phase IIb), which is planned to begin soon after completion of the present Phase II study subject to FDA approval, will be powered to detect more subtle efficacy responses. Although this second trial is still in the planning stages, it is expected to include 300 ALS patients recruited from 25 clinical sites and will take approximately 18 months to complete.
According to Mr. Kriegsman, "Given the devastating health consequences of ALS, and the lack of an effective therapeutic treatment, we believe that successfully demonstrating safety and efficacy in this latter Phase II clinical trial could be sufficient to support product registration with the FDA."
The Principal Investigators
CytRx has assembled a team of renowned clinicians to conduct the Phase II clinical trial with Dr. Brown, Merit Cudkowicz, M.D., and Jeremy Shefner, M.D. acting as co-principal investigators for the study. Dr. Cudkowicz is Associate Professor of Neurology at Harvard Medical School, co-chair of the Northeastern ALS Clinical Trials Consortium, co-director of the MGH Clinical Trials unit, and is an experienced principal investigator of ALS clinical trials. Dr. Shefner is Professor of Neurology at SUNY Upstate Medical University, co-chair of the Northeastern ALS Clinical Trials Consortium, and is also an experienced principal investigator of ALS trials.
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.
Arimoclomol, which was well absorbed and well tolerated in two Phase I clinical trials in healthy volunteers, was granted orphan drug status designation by the FDA for the treatment of ALS in May 2005. Receipt of orphan designation holds numerous potential benefits, including opportunities for grant funding toward clinical trial costs, tax advantages, FDA use-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 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 products, primarily in the area of small molecules and ribonucleic acid interference (RNAi), in a variety of therapeutic categories. The Company owns three clinical-stage compounds based on its small molecule "molecular chaperone" co-induction technology, as well as a targeted library of 500 small molecule drug candidates that may be used to screen for new drug candidates. CytRx has previously announced that a novel HIV DNA + protein vaccine exclusively licensed to CytRx and developed by researchers at 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. For more information, visit CytRx's Web site at www.cytrx.com.
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