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ExonHit Presents New Data on its Alzheimer's Lead Candidate at the International Geneva/Springfield Symposium

PARIS--(BUSINESS WIRE)--Mar 29, 2010 - Regulatory News:


  • EHT 0202 responders have a specific gene expression profile
  • Companion diagnostic could give early indication of responders

ExonHit Therapeutics (Paris:ALEHT) (Alternext: ALEHT) announced today that promising new data on EHT 0202, its lead candidate in Alzheimer's disease (AD), were presented at the 11th International Geneva/Springfield Symposium on Advances in Alzheimer Therapy from March 24th to March 27th in Geneva, Switzerland.

The Geneva/Springfield Symposium focuses on the pharmacological therapy of Alzheimer's disease with particular emphasis on the discovery of new drugs.

“Identifying patients that will respond to a treatment before its initiation is an approach that will be increasingly used in the coming years. ExonHit has the technology necessary to developing biomarkers that will be the cornerstone of tomorrow's personalized medicine,” said Loïc Maurel MD, President of the Management Board of ExonHit Therapeutics.

The EHT 0202 poster presentation reports preliminary results from a study designed to determine whether patients that responded best to EHT 0202, during the recently completed Phase IIa study, have a distinct blood-based gene expression profile. These data show that ExonHit's Genome-Wide SpliceArray™ expression profiling technology is able to clearly separate clinical patient subpopulations. Patients whose condition improved while on EHT 0202 have a different gene expression profile from those whose condition declined, and these gene expression profile differences are specific to EHT 0202. These data raise the possibility of identifying expression profiles that could allow the stratification of patients who will benefit from EHT 0202 therapy from those who won't prior to study initiation (1).

Applying this approach to other clinical development programs could significantly increase the likelihood of successfully achieving primary endpoints and could help identify the most relevant patient populations for a given therapeutic product.

A poster on AclarusDx™, ExonHit's first research-use-only molecular test for AD launched in December 2009, entitled “Identification of patients with Alzheimer's disease using molecular signatures derived from splice variant expression profiles from peripheral blood” was also presented (2).

About the International Geneva/Springfield Symposium

The bi-annual International Geneva/Springfield Symposium on Advances in Alzheimer Therapy focuses entirely on the pharmacological therapy of Alzheimer Disease with particular emphasis on the discovery of new drugs. The themes covered during that symposium include drug development at different preclinical and clinical stages, results of clinical trials and pharmaco-economics implications. The meeting is organized jointly by an American (Southern Illinois University, School of Medicine in Springfield Illinois, USA) and a European (University of Geneva, Medical School Dept. of Rehabilitation and Geriatrics) university. For more information, please visit

About EHT 0202

EHT 0202 has a novel mechanism of action when compared to existing Alzheimer's disease therapeutics: it stimulates the α-secretase pathway, thus enhancing the production of the procognitive and neuroprotective sAPPα fragment of APP (Amyloid Precursor Protein). Since the stimulation of the α-secretase pathway is to the detriment of Aβ amyloid peptide production, EHT 0202 potentially reduces toxic Aβ plaque formation (3). Phase I studies demonstrated good tolerability of EHT 0202 in both young and elderly healthy volunteers. Preclinical studies have shown that EHT 0202 protects cortical neurons against Aβ42-induced toxicity and that this neuroprotection is associated with sAPPα induction. EHT 0202 has also demonstrated procognitive properties in several animal models: age-related memory impairment and scopolamine-induced amnesia (4). Procognitive properties of EHT 0202 were recently published in an aged rat model (5).

About Alzheimer's disease

Alzheimer's disease is a progressive neurodegenerative condition that is the most frequent cause of dementia in the elderly. Worldwide, an estimated 26.6 million people had Alzheimer's disease in 2006. This number is set to quadruple by 2050 to more than 100 million; 1 in 85 people worldwide will be living with the disease (6). In France alone, 800,000 people, or 18% of the population over-75, have Alzheimer's disease (7).

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 innovative molecular diagnostic tests and therapeutics for neurodegenerative and cancer indications. ExonHit has a balanced investment strategy with in-house development programs and strategic collaborations, in particular with bioMérieux and Allergan).

ExonHit is headquartered in Paris, France and has U.S. offices 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.

Lastly, 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) Beurdeley P, Sol O, Zhou W, Carrière J, Einstein R, Haddad R, Désiré L, Pando M. “Identification of blood transcriptomic signatures in AD patients related to EHT 0202 treatment response”. Poster presented at the 11th International Geneva/Springfield Symposium on Advances in Alzheimer Therapy; 24-27 March, 2010; Geneva, Switzerland.

(2) Fehlbaum-Beurdeley P, Zhou W, Jarrige A-C, Calciano M, Gill P, Sol O, Dallares D, Jordan H, Wu D, Lei L, Einstein R, Vellas B. “Identification of patients with Alzheimer's disease using molecular signatures derived from splice variant expression profiles from peripheral blood”. Poster presented at the 11th International Geneva/Springfield Symposium on Advances in Alzheimer Therapy; 24-27 March, 2010; Geneva, Switzerland.

(3) Marcade M, Bourdin J, Loiseau N, Peillon H, Rayer A, Drouin D, Schweighoffer F, Desire L. Etazolate, a neuroprotective drug linking GABAA receptor pharmacology to amyloid precursor protein processing. Journal of Neurochemistry. 2008; 106: 392-404

(4) Pando M, Marcade M, Peillon H, Rayer A, Drouin D, Desire L. “An alpha-secretase stimulator drug for cognitive disorders associated with neurodégénération”. Poster presented at the 12th congress of the European Federation of Neurological Societies; 23-26 August, 2008; Madrid, Spain

(5) Drott J, Desire L, Drouin D, Pando M, Haun F. “”Etazolate improves performance in a foraging and homing task in aged rats”. Eur J Pharmacol. 2010 March 16. [Epub ahead of print]. PubMed: 20223232

(6) Brookmeyer R, Johnson E, Ziegler-Graham K, MH Arrighi (July 2007). "Forecasting the global burden of Alzheimer's disease". Alzheimer's and Dementia 3 (3): 186–91

(7) Plan Maladie d'Alzheimer 2004-2007- Ministère des solidarités, de la santé et de la famille

Contact: ExonHit Therapeutics
Corinne Hoff
+33 1 58 05 47 04
Alize RP
Caroline Carmagnol
+ 33 6 64 18 99 59



Posted: March 2010