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ExonHit Reports Proof-of-Concept in Breast Cancer Diagnostic Using its SpliceArray(TM) Platform

PARIS, March 16 /PRNewswire-FirstCall/ -- ExonHit Therapeutics (Alternext: ALEHT) today reported the publication in Lancet Oncology of a study conducted by Institut Gustave Roussy, which describes the identification of a deregulated cell function in breast cancer through the analysis of alternative RNA splicing [1]. Study data demonstrate that exons are differently expressed in malignant and benign lesions, and alternative transcripts determine the molecular characteristics of breast malignancy.

"The splice variants that we have identified could be used as targets for development of targeted therapies and also for a more precise diagnostic test. A molecular diagnosis for breast cancer could improve the performance of current diagnosis methods by increasing accuracy of cytological investigations and decreasing the use of core-needle biopsy or exploratory surgery," said Fabrice Andre, M.D. Breast Cancer Unit, Department of Medical Oncology, Institut Gustave Roussy, Villejuif, France. He added: "Patients could greatly benefit from a more precise diagnosis."

"We are very happy to report that this independent study demonstrates the clinical usefulness of our SpliceArray(TM) platform. It constitutes another example of its many possible applications," stated Dr. Loic Maurel, President of the Management Board of ExonHit Therapeutics. "We are using the same technology platform for all our blood based diagnostic projects; our internally funded diagnostic test for Alzheimer's disease (EHT Dx21) and the breast, colon and prostate cancer diagnostics developed with bioMerieux. We look forward to seeing other applications of our technology and launching the first product developed with this technology on the market."

SpliceArray(TM) biochips are ExonHit's proprietary research tools [2]. They quantify mRNA expression at the exon level. They also provide a comprehensive RNA splicing analysis across the entire human genome and enable the identification of known splice variants as well as the detection of variants which have not yet been discovered or catalogued. They represent a significant improvement over DNA arrays which only quantify expression at the gene level.

The study conducted by Institut Gustave Roussy and leading to this signature identification is the first study based on a large dataset of 165 breast tumor samples that compares gene expression of malignant versus benign breast tumors. The signature was tested on 71 tumor samples, 68 of which were properly classified; sensitivity and specificity were respectively 96% and 95% [1].

About ExonHit's SpliceArray(TM)

ExonHit's SpliceArray(TM) platform represents a novel generation of microarrays that incorporates a specific probe configuration, enabling an exhaustive monitoring of RNA splice variants [2]. The Human Genome Wide SpliceArray(TM) biochips which profile close to 21,000 human genes associated to 140,000 RNA splice events have been used in the Company's biomarker discovery process.

RNA splicing represents a key regulatory mechanism of gene expression. One single gene can be transcribed into several splice variants encoding proteins exhibiting different biological functions. Current predictions suggest that as many as 80% of human genes exhibit some form of alternative splicing. Given the large contribution of splice variants to the human transcriptome and the high density of disease-causing mutations that occur in splicing-related sequences, the monitoring of alterations in alternatively spliced transcripts represents not only a source of potential drug targets, but also a biomarkers discovery engine.

About Institute Gustave Roussy

The Institut Gustave Roussy (IGR, Villejuif, France), European leading Comprehensive Cancer Center, is a non profit academic entity belonging to the French public hospital service and that is authorized to receive donations and legacies. IGR is a center wholly patient-oriented with a global expertise and the mission to fight cancer. Located South of Paris on an unique site, IGR employs 2,000 professionals working in care, research and teaching. Some IGR figures: 368 beds and places for day-cases, 159 certified physicians, 751 professional caregivers, 161,000 consultations and 43,000 patients cared per year, 14 basic research units, 300 research scientists, and 260 students, researchers and physicians trained per year. For more information, please visit

About ExonHit Therapeutics

ExonHit Therapeutics (Alternext: ALEHT) is a fast emerging healthcare player active in both therapeutics and diagnostics. The Company is applying its proprietary technology, based on the analysis of alternative RNA splicing, to develop diagnostic tests and innovative treatments for neurodegenerative diseases, especially Alzheimer's disease and cancers. ExonHit has a balanced investment strategy with internal development programs and strategic collaborations, in particular with bioMerieux and Allergan.

ExonHit is headquartered in Paris, France and has a U.S. facility in Gaithersburg, Maryland. The Company is listed on Alternext of NYSE Euronext Paris. For more information, please visit


This press release contains elements that are not historical facts including, without limitation, certain statements on future expectations and other forward-looking statements. Such statements are based on management's current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those anticipated.

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.


    [1] Andre F, Michields S, Dessen P, Scott V, Suciu V, Uzan C,
        Lazar V, Lacroix L, Vassal G, Spielmann M, Vielh P, Delaloge S.
        Exonic expression profiling of breast cancer and benign lesions: a
        retrospective analysis. The Lancet Oncology, Early Online
        Publication, 26 February 2009


SOURCE ExonHit Therapeutics SA
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