Press Release

Anti-Cancer Activity Drug IRX4204 Shows Promise as a Potential Disease-Modifying Agent for Alzheimer’s Disease

Giulio Pasinetti, MD, PhD, presented research at 6th Neurodegenerative Conditions Research and Development Conference.

New York
(September 13, 2012)

New research presented today at the 6th Neurodegenerative Conditions Research and Development Conference in San Francisco, California demonstrates the role of the investigational compound IRX4204 in alleviating cognitive decline in animal models of Alzheimer's disease. The presentation entitled "Investigation of the RXR-specific agonist IRX4204 as a Disease Modifying Agent of Alzheimer's Disease Neuropathology and Cognitive Impairment" was made by lead researcher Guilio Maria Pasinetti, MD, PhD, of the Mount Sinai School of Medicine in New York City.

"The data presented include studies demonstrating attenuation of cognitive deterioration in IRX4204-treated mouse model of Alzheimer's disease through promotion of electophysiological features in the brain associated with retention of memory function," said Dr. Pasinetti, the Saunders Family Chair and Professor in Neurology, and Director, Center of Excellence for Novel Approaches to Neurotherapeutics, in the Department of Neurology of Mount Sinai School of Medicine.

The study included data from preclinical studies of the Retinoid X Receptor (RXR) agonist compound IRX4204, a compound produced by Io Therapeutics, Inc. The company also announced the establishment of a sponsored research agreement with Mount Sinai School of Medicine to conduct additional studies of this compound in Alzheimer's disease.

"IRX4204 is a highly specific and very potent agonist of the RXR nuclear receptor pathways, which has at least a 1000-fold difference in the concentrations that activate RXR versus concentrations that activate RAR nuclear receptors," said Rosh Chandraratna, PhD, Chief Scientific Officer of Io Therapeutics. "This specificity and potency distinguishes IRX4204 from bexarotene, an RXR agonist compound, which also has been reported to be active in animal models of Alzheimer's disease."

Dr. Chandraratna added, "IRX4204 has previously completed a phase I/II clinical trial in patients with a variety of solid tumors, in which it was well tolerated for up to 20 months without observation of typical RAR-related toxicities, and in which findings supporting potential clinical activity in a variety of types of cancer were observed."

The company plans to file an additional IND for evaluation of IRX4204 in patients with Alzheimer's disease, with clinical studies in Alzheimer's patients anticipated to start in 2013. IRX4204 is currently being evaluated in a phase II clinical trial in men with advanced prostate cancer.

"The treatment of Alzheimer's Disease remains a serious unmet medical need which IRX4204 may be able to address," said Martin Sanders, MD, Chairman and Chief Executive Officer of Io Therapeutics. "Dr. Pasinetti's studies, combined with data from other laboratories investigating the less specific RXR agonist bexarotene, support that RXR agonists have potential for slowing, and possibly reversing pathology and cognitive deficits in Alzheimer's disease patients."

Alzheimer's disease currently afflicts more than 5 million Americans and may triple in prevalence to more than 16 million Americans by 2050, according to data from The Alzheimer's Association

About The Mount Sinai Medical Center

The Mount Sinai Medical Center encompasses both The Mount Sinai Hospital and Mount Sinai School of Medicine. Established in 1968, Mount Sinai School of Medicine is one of the leading medical schools in the United States. The Medical School is noted for innovation in education, biomedical research, clinical care delivery, and local and global community service. It has more than 3,400 faculty in 32 departments and 14 research institutes, and ranks among the top 20 medical schools both in National Institutes of Health (NIH) funding and by U.S. News and World Report.

The Mount Sinai Hospital, founded in 1852, is a 1,171-bed tertiary- and quaternary-care teaching facility and one of the nation's oldest, largest and most-respected voluntary hospitals. In 2012, U.S. News and World Report ranked The Mount Sinai Hospital 14th on its elite Honor Roll of the nation's top hospitals based on reputation, safety, and other patient-care factors. Mount Sinai is one of 12 integrated academic medical centers whose medical school ranks among the top 20 in NIH funding and by U.S. News and World Report and whose hospital is on the U.S. News and World Report Honor Roll. Nearly 60,000 people were treated at Mount Sinai as inpatients last year, and approximately 560,000 outpatient visits took place.

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Tags: Icahn School of Medicine at Mount Sinai, Mount Sinai Health System, Neurology, Psychiatry, Research

About the Mount Sinai Health System

Mount Sinai Health System is one of the nation’s leading integrated academic health systems and one of the largest in the New York metropolitan area. Its comprehensive system includes seven hospitals, more than 400 outpatient practices, over 600 research and clinical laboratories, the Icahn School of Medicine at Mount Sinai, the Graduate School of Biomedical Sciences, and the Mount Sinai Phillips School of Nursing. Together, the Health System comprises approximately 48,000 employees, more than 9,000 physicians, and 8,600 nurses.

As a leading learning health system, Mount Sinai combines clinical expertise with scientific discovery to improve patient care while training the next generation of health care and biomedical leaders. The Health System provides care across every stage of life, from prenatal care through geriatrics, while advancing personalized medicine through artificial intelligence, data science, and biomedical research.

Mount Sinai is consistently recognized among the nation’s leading academic health systems for patient care, research, and education. The Mount Sinai Hospital is ranked No. 1 in New York by Newsweek and No. 5 on the magazine’s World’s Best Hospitals list. The Icahn School of Medicine at Mount Sinai ranks No. 11 among U.S. medical schools and No. 1 among freestanding medical schools for National Institutes of Health funding, reflecting the strength of its scientific enterprise and leadership in biomedical research.