ExonHit Therapeutics announced that it plans to start a Phase IIa proof-of-concept study in
Alzheimer's disease with EHT 0202, its most advanced compound in development in the early part of 2005. The company has already demonstrated that EHT 0202 improves attention and cognition
and has neuroprotective properties in animal models.
Based on these data it is likely that
EHT0202 may prove suitable for symptom relief and as a disease modifying agent in patients
with Alzheimer's disease. Preclinical studies with EHT0202 have revealed protection from
cell death following induction of different stresses (
ischemia or toxic treatment) as well
as improved learning and cognitive performance in aged rats (water-maze and Barnes tests)
and mild anxiolytic properties at higher dosages (10mg/kg). Toxicological studies have
also demonstrated that EHT 0202 is well tolerated and well absorbed when given orally.
A Phase I single dose study by oral route has just been completed and confirmed that EHT
0202 is also well tolerated (Maximum Tolerated Dose of 120 mg) and well absorbed via oral
administration in healthy volunteers.
Based on these data, a Medical Advisory Board confirmed its interest in initiating a Phase
IIa proof-of-concept study with EHT 0202. The study is designed as a randomized, double-blind,
placebo-controlled, parallel group, multicentre study evaluating the effects of three doses
of EHT 0202 on the cognitive functions and psychomotor performance of patients suffering
from probable, mild to moderate Alzheimer's disease. It will enroll 40 patients that will
be treated for 28 consecutive days followed by a 14 days observation period without study
drug. The study is anticipated to start in early 2005.
"Our goal is to evaluate whether EHT0202 improves the performance of patients suffering
from cognitive disorders on a selected number of tests focusing on attention and learning
capabilities", commented Philippe Guillet, VP Clinical Development at ExonHit. "Current
treatments are clearly not yet satisfactory and there is a large medical need for drugs
with new mechanisms of action that can be associated with acetylcholine esterase inhibitors",
he added.
"ExonHit has developed a powerful technology based on RNA splicing analysis, which pinpoints
specific targets for therapeutic intervention. This technology combined with our internal
medicinal chemistry capabilities will allow us to bring to the clinic additional drugs
either with our own resources or through partnerships. If this proof-of-concept study is
positive", declared Bruno Tocqué, CEO ExonHit Therapeutics, "we intend to partner out EHT0202
for Alzheimer's disease and other types of dementia".