Press Release: Exonhit Identifies a Panel of Genes to Diagnose, From Blood, Alzheimer's Disease

Exonhit Identifies a Panel of Genes to Diagnose, From Blood, Alzheimer's Disease PARIS, France, March 15 -- ExonHit Therapeutics, a drug and diagnostic discovery company, today announces that it has reached an important milestone in the differentiation, from blood, of patients with Alzheimer's disease from patients with no cognitive impairments. Scientific data, which are presented at the 8th International Conference on Alzheimer and Parkinson diseases in Salzburg, Austria, are expected to lead to the development of novel diagnostic tests that will enable the detection of Alzheimer's disease from blood. Such tests should also facilitate recruitment of patients for the evaluation of therapeutic entities in the context of clinical trials. The scientific data shows that a panel of 60 biomarkers can clearly distinguish individuals, males or females, presenting the symptoms of Alzheimer's disease as judged by a MMSE (Mini Mental State Examination) score < 20 and a GDS (Global Deterioration Scale) score greater than or equal to 4, from individuals with no cognitive losses. From a total of 85 individuals, 51 patients with Alzheimer's disease (AD) and 34 without any symptoms of disease (non AD), this panel correctly classified 78% of the AD and 82% of the non AD, with an accuracy of 76%. The same panel properly classified as non AD 81% of the individuals (n=27) presenting neurological disorders such as depression, schizophrenia or bipolar disorders. A prospective clinical study will further define the specificity and selectivity of the markers identified. This is an important development as there is currently no diagnostic test that can accurately identify patients with Alzheimer's disease. The diagnosis of Alzheimer's disease is determined by default, thus with no direct evidence, following an in-depth medical examination that finds no evidence of other forms of dementia. Currently, the only way to confirm Alzheimer's disease is by performing a post-mortem analysis of the brain. This method used to diagnose the disease is far from satisfactory, especially for the identification of patients suffering from early stage disease. "Biomarkers from blood represent a major step as compared to current methodology aimed at detecting biomarkers from cerebrospinal fluid obtained through a lumbar puncture" declares Gustavo Roman, Professor of Medicine and Neurology at the University of Texas in San Antonio, and a member of ExonHit's Scientific Board. "Molecular diagnostics represent a large market potential and are intimately linked to the future of our company. We already have a partner for the marketing of our cancer diagnostics, and it is our intention to bring the non-cancer diagnostics to registration" commented Bruno Tocque, President of the Management Board of ExonHit. About Alzheimer's disease Initially thought of as mainly neuronal, Alzheimer's disease is now better understood and is thought to be caused by alterations in the three main biological systems which ensure the body's homeostasis: the nervous, endocrine and immune system. Hence, alterations of certain neurons lead to modifications in endocrine regulation. This in turn, impacts the activity and viability of other neurons and the function of the immune system. Alteration in the immune response also plays a key role in the development of Alzheimer's disease. As a result of this better understanding of the disease and ExonHit's expertise, it is now potentially possible to measure the various stages of Alzheimer's disease by examining cells circulating in the blood. Study results details ExonHit has applied its gene profiling technologies, DATAS(TM) (Differential Analysis of Transcripts with Alternative Splicing) and SpliceArray(TM), to the blood samples of patients with or without Alzheimer's clinical symptoms. This has led to the identification and isolation of a set of 60 biomarkers including spliced variants using a "nearest neighbour" (KNN) algorithm on a train group. The application of these 60 biomarkers to a test group allowed to classify properly 78% of the patients with AD (sensitivity) and 82% of the non AD (specificity) with an accuracy of 76%. This set of biomarkers will now be further used using ExonHit's SpliceArray(TM) technology applied to Affymetrix GeneChip(R) microarray platform. ExonHit Therapeutics is the world's leader in the analysis of alternative RNA splicing, a process which when deregulated plays a key role in the onset of various diseases. ExonHit has a multi-component commercial strategy to capture the maximum value from its leadership in alternative splicing. The Company is already generating revenues from a new generation of microarrays, SpliceArrayTM family of products, which enable life science researchers to detect crucial disease-associated information. These products are marketed worldwide in conjunction with Agilent and Affymetrix. In the field of diagnostics, ExonHit has a major collaboration with bioMerieux to develop completely novel predictive blood-based cancer diagnostics, which could play a key role in improving the treatment of breast cancer and other major cancers. ExonHit is also developing its own internal diagnostic projects for the detection of other chronic diseases such as Alzheimer's disease and atherosclerosis. In parallel, ExonHit is building its own therapeutic pipeline in the fields of neurodegenerative disease and cancer. The Company has advanced drug candidates into clinical trials and is evaluating several promising pre-clinical compounds. ExonHit also has a strategic partnership with Allergan, to discover and develop new therapeutics in the areas of pain, neurological diseases and ophthalmology. This collaboration provides on-going research funding to ExonHit. Founded in 1997, ExonHit is headquartered in Paris, France and has a U.S. facility in Gaithersburg, Maryland. The Company has been listed on Alternext of Euronext Paris (ticker: ALEHT; ISIN: FR0004054427) since November 17, 2005. In addition, ExonHit Therapeutics, its shareholders, and its affiliates, directors, officers, advisors and employees have not verified the accuracy of, and make no representations or warranties in relation to, statistical data or predictions contained in this press release that were taken or derived from third party sources or industry publications, and such statistical data and predictions are used in this press release for information purposes only. Finally, this press release may be drafted in the French and English languages. In an event of differences between the texts, the French language version shall prevail.

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