IRX4204 as a Potential Disease-Modifying Treatment for Alzheimer’s Disease

San Francisco, California – September 13, 2012 – Io Therapeutics, Inc. announced today the presentation of data from preclinical studies of its Retinoid X Receptor (RXR) agonist compound IRX4204 in animal models of Alzheimer’s disease, at the 6th Neurodegenerative Conditions Research and Development conference, currently being held in San Francisco, CA. The presentation titled “Investigation of the RXR-specific agonist IRX4204 as a Disease-Modifying Agent of Alzheimer’s Disease Neuropathology and Cognitive Impairment” was presented by Dr. Giulio Maria Pasinetti.

The studies were directed by Dr. Pasinetti, M.D., Ph.D., 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 in New York City. Dr. Pasinetti said, “The data presented today 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.”

Io Therapeutics, Inc. 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.

Rosh Chandraratna, Ph.D., Chief Scientific Officer of Io Therapeutics, said “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. 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.”

Martin Sanders, M.D., Chairman and Chief Executive Officer of Io Therapeutics, said, “IRX4204 is currently being evaluated in a phase II clinical trial in men with advanced prostate cancer. 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.” Dr. Sanders continued, “The treatment of Alzheimer’s disease remains a serious unmet medical need which IRX4204 may be able to address. Recent failures of phase III clinical trials of two different monoclonal antibodies specific for beta amyloid have reinforced the need for development of additional types of interventions for this devastating disease, which currently afflicts more than 5 million Americans and may afflict 16 million Americans by 2050 according data from the Alzheimer’s Association. 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.”

About Io Therapeutics

Io Therapeutics, Inc. is a privately held company headquartered in Santa Ana, California, focused on the development of treatments for Alzheimer’s disease, cancer and autoimmune diseases. For more information on Io-Therapeutics and its product pipeline see:

Forward-looking statements

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