ExonHit Therapeutics Reports Advancements in the Clinical Development of EHT 0202, its Phase II Drug for Alzheimer’s Disease

February 26, 2009

PARIS, February 26 /PRNewswire-FirstCall/ — ExonHit Therapeutics
(Alternext: ALEHT) is pleased to announce that clinical testing of EHT 0202,
its lead compound in Alzheimer’s disease, is progressing well. Patient
enrolment for the Phase IIa trial assessing EHT 0202 in patients with
Alzheimer’s disease is completed.

“The successful completion of patient enrolment for the Phase IIa
represents an important milestone in the development of EHT 0202, and
provides a clear timeline for the release of results,” stated Dr. Loic
, President of the Management Board of ExonHit Therapeutics. “Our
strategy is to bring EHT 0202 up to the end of the ongoing study and then to
look for a partner to move EHT 0202 through further clinical development. We
have already met with several pharmaceutical companies that are looking
forward to the Phase IIa results.”

The trial is conducted under the supervision of Professor Bruno Vellas,
Head of Alzheimer’s Disease Clinical Research Center and Gerontopole,
Toulouse University Hospital, France.

“EHT 0202 has an original mechanism of action: it stimulates the
alpha-secretase pathway. If the neuroprotective and symptomatic effects of
EHT 0202 demonstrated in animal models are confirmed in humans, it could
change the treatment paradigm for Alzheimer’s disease,” stated Professor
Vellas. “Current available drugs are symptomatic only and their clinical
efficacy is limited over time, after which the evolution of the degenerative
process starts progressing again. EHT 0202 is an interesting approach to
potentially slow the evolution of the disease.”

The Phase IIa trial is a multicenter, randomized, double-blind,
placebo-controlled study primarily investigating the safety and tolerability
of EHT 0202 in approximately 150 patients with Alzheimer’s disease. The
effect of two different doses of EHT 0202 as adjunctive therapy to an
acetylcholinesterase inhibitor will be evaluated in comparison to placebo.
Ambulatory patients suffering from mild to moderate Alzheimer’s disease are
randomized and receive oral treatment, twice a day, of either 40 or 80 mg of
EHT 0202, or placebo over a three-month period. The study design will also
allow for the collection of preliminary data related to many clinical
efficacy parameters of EHT 0202, notably including a battery of cognitive
assessments (ADAS-Cog, NTB, MMSE) but also assessment of patients’ daily
living activities, global assessment and behaviour.

Study results will be available in Q4 2009.

About EHT 0202

EHT 0202 has a novel mechanism of action when compared to existing
Alzheimer’s disease therapeutics: it stimulates the alpha-secretase pathway,
thus enhancing the production of the procognitive and neuroprotective
sAPPalpha fragment of APP (Amyloid Precursor Protein). The stimulation of the
alpha-secretase pathway being to the detriment of Abeta amyloid peptide
production, EHT 0202 potentially reduces toxic Abeta plaque formation [1].

Phase I studies demonstrated good tolerability of EHT 0202 in both young
and aged healthy volunteers; importantly, no sedation or emesis were observed

Preclinical studies have shown that EHT 0202 protects cortical neurons
against Abeta42-induced stress and that this neuroprotection is associated
with sAPPalpha induction. EHT 0202 has also demonstrated pro-cognitive
properties in several animal models: age-related memory impairment and
scopolamine-induced amnesia [2].

About Alzheimer’s disease

Alzheimer’s disease is the most frequent cause of dementia in the aging
population. The World Health Organization estimated in 2001 that 18 million
people around the world were suffering from Alzheimer’s disease and that this
figure could nearly double by 2025 to 34 million [3].

About ExonHit Therapeutics

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 Therapeutics 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,
SpliceArray(TM) family of products that 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 Therapeutics 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.

In parallel, ExonHit Therapeutics is developing its own therapeutic
pipeline in the field of neurodegenerative diseases and cancer. The Company
has advanced drug candidates into clinical trials and is evaluating several
promising preclinical compounds. ExonHit Therapeutics 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 ongoing research funding to ExonHit.

Founded in 1997, ExonHit is headquartered in Paris, France and has a U.S.
facility in Gaithersburg, Maryland. The Company is listed on Alternext of
Euronext Paris (ISIN: FR0004054427; ticker: ALEHT) since November 17, 2005.
For more information, please visit http://www.exonhit.com.


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] 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

[2] Pando M, Marcade M, Peillon H, Rayer A, Drouin D, Desire L. An alpha-secretase stimulator drug for cognitive disorders associated with
neurodegeneration. Presented at the 12th congress of the European Federation
of Neurological Societies; 23-26 August, 2008; Madrid, Spain

[3] WHO 2001. Azheimer’s disease: The Brain Killer.

WHO website:
066.htm (Due to the length of this URL, it may be necessary to copy and paste
this hyperlink into your Internet browser’s URL address field. Remove the
space if one exists.)

SOURCE ExonHit Therapeutics SA

Source: newswire

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