LAVAL, QC, June 22 /CNW Telbec/ - Neurochem Inc. (NASDAQ: NRMX; TSX: NRM)
has issued an update on the progress made since announcing in April 2007 that
adjustments would be required to arrive at a reliable statistical model for
the North American Phase III clinical trial for tramiprosate (ALZHEMED(TM))
for the treatment of Alzheimer's disease (AD). Neurochem has been advised by
its external team of statisticians that they have made progress adjusting the
statistical model, reaching an acceptable level of validity for the disease
modification endpoint, as measured by magnetic resonance imaging (MRI).
However, further work is required to reach a reliable model particularly for
the co-primary clinical efficacy endpoint composed of ADAS-cog(1) and
CDR-SB(2). Therefore, the Company expects the results to be available later in
Since April, Neurochem has provided regular updates on the program,
including preliminary data and reports on the model. Certain of the
confounding factors identified by the statisticians and included so far in
adjustments to the model are the use of concomitant drugs, including change in
memantine use and dose, change in vitamin E use and dose, change in
anti-depressant use, and change in acetylcholinesterase inhibitor dose.
To date, Neurochem has invested significant human and financial resources
in this program. The Company is committed to continuing its efforts to adjust
the model in order to evaluate the significance of the data and remains
hopeful that the results will prove valuable in the development of the first
potential disease-modifying treatment for this terrible disease. Given the
magnitude and duration of this trial, the size of the patient population, and
the challenge of testing a first-in-class product candidate, which is
hypothesized to target the underlying pathology of AD, Neurochem will need
more time to work with experts in the field, including the trial's
investigators and the regulatory authorities.
The Company has been also advised by its statistical team that refinement
of the statistical model needs further adjustments. In view of the continuing
complexities of the trial, which involves the first attempt to employ these
particular primary outcome measures across multiple clinical sites for a
potential disease modification effect over 18 months, the next steps are
expected to require the guidance of the U.S. Food and Drug Administration
(FDA). Consequently, the Company has contacted the agency to request a meeting
with the objective of seeking its advice and explore alternative approaches
acceptable to the agency.
At the meeting, the Company expects to discuss some of the alternatives
it is considering to further adjust the model. These include suggestions for a
more detailed review of individual patient files to try to identify other
About the Two Phase III Clinical Trials in North America and Europe
The North American study was a multicenter, randomized, double-blind,
placebo-controlled, three-armed and parallel-designed, 18-month Phase III
clinical trial. The study included 1,052 patients with mild-to-moderate AD
recruited across 67 sites in Canada and the United States randomized to
receive either placebo or one of two doses (100mg or 150mg twice daily) of
tramiprosate (ALZHEMED(TM)). All patients were required to be treated with
conventional symptomatic AD therapies during the clinical trial and were
required to be on a stable dose of such therapies for at least four months
prior to the initial screening visit of the trial. At study entry, patients in
this trial had been administered standard therapies for an average of
approximately 20 months.
All patients who completed the clinical trial were eligible to receive
tramiprosate (ALZHEMED(TM)) in an open-label extension study initiated in May
2006. Approximately 90% of the patients who completed the Phase III
double-blind study for tramiprosate (ALZHEMED(TM)) elected to enroll in the
ongoing extension study and receive 150mg twice daily of tramiprosate
Neurochem is also currently conducting a European Phase III clinical
trial for tramiprosate (ALZHEMED(TM)) for the treatment of AD. The European
study is of a similar design to the North American double-blind trial and over
950 mild-to-moderate AD patients are already enrolled at about 70 clinical
centers across 10 European countries. To date, the European Data Safety
Monitoring Board has met three times and recommended on each occasion that the
study should continue as planned. The Company expects to continue patient
enrolment and is presently considering modifications that would need to be
made to the design of the European trial to take best advantage of the
experience gained from the North American Phase III clinical trial.
About Tramiprosate (ALZHEMED(TM))
Tramiprosate (ALZHEMED(TM)) is a small, orally-administered molecule
known as an amyloid Beta antagonist. Tramiprosate (ALZHEMED(TM)) crosses the
blood-brain-barrier, binds to soluble ABeta peptide and interferes with the
amyloid cascade that is associated with amyloid deposition and the toxic
effects of ABeta peptide in the brain. The presence of amyloid in the brain is
one of the major histopathological characteristics of AD. The amyloid cascade
hypothesis proposes that certain forms of the ABeta peptide are toxic and
causally related to the severity of AD. The ABeta peptide is one of the most
promising targets for the development of AD therapies.
About Alzheimer's Disease
AD is a leading cause of death in older people. The disease is
characterized by the progressive death of nerve cells in the brain, making it
difficult for the brain's signals to be transmitted properly. A person with AD
experiences problems with memory, judgment, thinking, and eventually with
motor functions, all of which make it difficult for the person to participate
in daily activities.
According to the U.S. Alzheimer Association (2007), there are now more
than five million people in the United States living with Alzheimer's disease.
This number includes 4.9 million people aged 65 and older. It also includes at
least 200,000 individuals younger than 65 with early-onset Alzheimer's
disease. It is estimated that by 2010, there will be 454,000 new cases of AD a
year, 615,000 new cases by 2030 and 959,000 new cases by 2050. In the United
States, the direct and indirect costs of AD and other dementias amount to more
than US$148 billion annually.
Neurochem Inc. is focused on the development and commercialization of
innovative therapeutics to address critical unmet medical needs. Eprodisate
(KIACTA(TM)) is currently being developed for the treatment of Amyloid A (AA)
amyloidosis, and is under regulatory review for marketing approval by the U.S.
Food and Drug Administration, the European Medicines Agency and Swissmedic.
Tramiprosate (ALZHEMED(TM)), for the treatment of Alzheimer's disease, has
completed a Phase III clinical trial in North America and is currently in a
Phase III clinical trial in Europe, while tramiprosate (CEREBRIL(TM)), for the
prevention of Hemorrhagic Stroke caused by Cerebral Amyloid Angiopathy, has
completed a Phase IIa clinical trial.
To Contact Neurochem
For additional information on Neurochem and its drug development
programs, please call the North American toll-free number 1 (877) 680-4500 or
visit our Web site at www.neurochem.com.
(1) 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.
(2) Dementia Rating - sum of boxes rating scale (CDR-SB), a measure of
This news release contains forward-looking statements regarding
tramiprosate (ALZHEMED(TM)) as well as regarding continuing and further
development efforts. These statements are based on the current analysis and
expectations of management. Drug development necessarily involves numerous
risks and uncertainties, which could cause actual results to differ materially
from this current analysis and these expectations. Analysis regarding the
results of clinical trials may not provide definitive results regarding
safety, tolerability or therapeutic benefits. Even if all the endpoints sought
in the clinical trials were met (which is not certain), there is no certainty
that regulators would ultimately approve tramiprosate (ALZHEMED(TM)) for sale
to the public. Risks and uncertainties may include: failure to demonstrate the
safety, tolerability and efficacy of our product, that actual results may vary
once the final and quality-controlled verification of data and analyses has
been completed, the expense and uncertainty of obtaining regulatory approval,
including from the FDA, and the possibility of having to conduct additional
clinical trials. Further, even if regulatory approval is obtained, therapeutic
products are generally subject to: stringent on-going governmental regulation,
challenges in gaining market acceptance, and competition. Neurochem does not
undertake any obligation to publicly update its forward-looking statements,
whether as a result of new information, future events, or otherwise. Please
see the Annual Information Form for further risk factors that might affect the
Company and its business.
For further information:
For further information: Dr. Lise Hébert, Vice President, Corporate
Communications, (450) 680-4572, email@example.com