New Paper Highlights ExonHit’s Novel Approach for the Treatment of Alzheimer’s Disease
Exonhit Therapeutics (FRANCE)

November 24, 2005 - Paris, France.
ExonHit Therapeutics, a drug and diagnostic discovery company, today announced that EHT 0206, one of its drug candidates, has shown promising new data in the treatment of Alzheimer’s disease. Preclinical studies of EHT 0206, published in the US peer reviewed magazine Journal of Biological Chemistry (J Biol Chem. 2005 Nov 11;280(45):37516-25), have shown that EHT 0206 has the potential not only to slow down the progression of the Alzheimer’s disease but can also change the course of the underlying disease.
The newly published data shows that EHT 0206 may decrease the formation of senile plaques in animal models. This is a key finding, as these plaques are thought to be responsible for the neuronal loss and consequent cognition impairment seen in Alzheimer’s patients. EHT 0206 is a small molecule that can be administered both orally and systemically which was synthesised in-house by ExonHit. These preliminary preclinical results also show that EHT0206 crosses the hemato-encephalic barrier to reach the brain and is well tolerated. “We are excited by the initial data that we have generated with EHT 0206.
The results that have been published today suggest that we have found a therapeutic approach to Alzheimer’s disease that major pharmaceutical companies have been looking for many years, a compound that is capable of reducing the formation of the senile plaques which are central to this devastating illness,” commented Bruno Tocqué, President of the Management Board of ExonHit. “Our new molecular series has the potential to significantly alter the course of this disease and we intend to evaluate it in human trials as quickly as possible. This will be our second clinical project with EHT0202 in this indication,” he added. By applying its unique DATAS technology on the brain tissue of patients with Alzheimer’s disease, ExonHit has been able to identify alterations in the Rac protein, which pinpointed out the possible essential role of the Rac protein in the development of the disease. It has been shown that alterations of Rac and its functionality in the brain increases production of beta-amyloid peptide (Aß, a key step in the formation of senile plaques. EHT 0206 blocks the activity of the Rac protein and the subsequent accumulation of the Aß which forms the senile plaques. ExonHit has filed patents for all the original chemical series showing activity on the Rac protein as well as their uses for various appropriate therapeutic indications, in particular Alzheimer’s disease.