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Phase 3 Alzhemed
Study
Alzhemed™ is an
investigational oral product candidate for mild-to- moderate AD that has
been specifically designed to modify the course of the disease through its
anti-amyloid activity. Phase III trials on the product candidate were
launched in 70 clinical centers in North America last month.
Cognitive Function
The study followed 19 mild-to-moderate AD patients who received study
medication for 20 months. The mild-to-moderate AD patients (n (equal sign)
19)(5) showed an average ADAS-cog score of +6.2 points, as opposed to +11.9
points on average in comparable historical controls(6) with AD. A subset of
mild AD patients (n (equal sign) 10) responded best and showed a change from
baseline in their average ADAS-cog score of +2.4 points. This result
compares favorably with a score of +8.6 points on average in comparable
historical controls.
Last April, the Company issued data on the first 18 patients who had
completed 16 months. Neurochem is now reporting on all patients with
mild-to- moderate AD who received study medication for 16 months. The
mild-to-moderate AD patients (n (equal sign) 26) showed an average ADAS-cog
score of +5.3 points, as opposed to +9.6 points on average in comparable
historical controls with AD. A subset of mild AD patients (n (equal sign)
15) responded well and showed a change from baseline in their average ADAS-cog
score of +1.7 points. This result compares favorably with an average score
of +7.6 points in comparable historical controls. Overall, nine out of the
15 mild AD patients were stabilized or improved at 16-months.
Global Measure of Performance
The average CDR-SB score in the mild-to-moderate AD patients after 20 months
on study medication showed +2.7 points on average. These results compare
favorably with the already reported(7) 12-month mean change in the CDR-SB
score of +2.2 points change in comparable mild-to-moderate AD patients.
Safety and Tolerability
Alzhemed™ continues to be safe and well tolerated after up to 20 months of
follow-up. Nausea and vomiting occurred primarily at the beginning of the
treatment and decreased over time. By 16 months, no nausea and vomiting were
reported. Overall, five patients withdrew prematurely from the beginning of
the Phase III clinical trial over the 20-month period due to adverse events:
three due to nausea and vomiting, one because of weakness and weight loss
and another for increased agitation and delusion.
"These results in Alzheimer's patients are promising because typically,
patients decline significantly over a prolonged period," said Dr. Aisen,
who also is principal investigator in the United States for the Phase III
study on Alzhemed™. "Alzhemed™ has the potential to modify the
progression of AD because it acts directly on its core pathology. Because
existing therapies can only treat disease symptoms, Alzhemed™ could become
the new paradigm for AD treatment if it is approved by regulatory
authorities."
"Given the very long treatment period with Alzhemed™ in our on-going
open-label Phase II extension study, and even though the study was not
designed to be statistically significant, we are encouraged by the
persisting stabilization of the majority of mild AD patients receiving this
product candidate. This interim data strengthens our belief that Alzhemed™
is a potential breakthrough to stop the cause of this devastating
disease," said Francesco Bellini, Ph. D., Chairman and CEO of Neurochem.
Neurochem's Phase III clinical trial on Alzhemed™ in North America will
run for a period of 18 months and be conducted in 50 U.S. and 20 Canadian
clinical centers. The Company anticipates launching its Phase III trial in
Europe early in 2005.
About Alzhemed™
Alzhemed™ is an orally administered, small organic molecule that has been
specifically designed to modify the course of AD through its anti-amyloid
activity. As part of a "disease modifying" novel class of product
candidates, Alzhemed™ is expected to act at many levels: in binding to
soluble amyloid beta (A(B)) protein, to prevent and stop the formation and
the deposition of amyloid fibrils in the brain, and to reduce the amyloid-induced
toxicity on neuronal and brain inflammatory cells associated with amyloid
build-up in AD.
References 1 - Mini
Mental State Exam (MMSE): 19-25 2 - Mini Mental State Exam (MMSE): 13-18 3 -
Alzheimer's Disease Assessment Scale, cognitive subpart (ADAS-cog). The ADAS-cog
is a 70-point scale designed to measure, with the use of questionnaires, the
progression and the severity of cognitive decline as seen in AD. The ADAS-cog
scale quantifies the number of wrong answers. Consequently, a high score on
the scale indicates a more severe case of cognitive decline. When analysing
results, a negative score indicates the improvement of cognitive function
and a positive score the deterioration of such function. The ADAS-cog has
been validated by the regulatory authorities as the gold standard scale for
the monitoring of cognitive function in AD patients. This scale is a
compulsory parameter of efficacy when submitting for market approval of an
AD drug to the authorities such as the Food and Drug Administration. 4 -
Clinical Dementia Rating - sum of boxes rating scale (CDR-SB), a measure of
global performance. 5 - n: number of patients per group. 6 - Stern, R.G., et
al. Am.J.Psychiatry 151:3, March 1994. 7 - Aisen, P.S., et al. JAMA 289:
2819, June 4, 2004.
Source: Neurochem Inc.
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