|
July 18, 2004
Neurochem's on-going open-label Phase II extension study for Alzhemed™ shows continued
positive interim results in Alzheimer's Disease patients after 20 months.
Conference Call and live webcast on July 19, at 10:00 A.M.
- Neurochem Inc. (NASDAQ: NRMX; TSX: NRM) announced today that interim results of an on-going
open-label Phase II extension study of the effects of Alzhemed™ on cognitive function and
global measure of performance in patients with mild1-to-moderate2 Alzheimer's
Disease (AD) were presented at the 9th International Conference on Alzheimer's Disease and Related
Disorders (ICAD) in Philadelphia. The study showed that overall, approximately 70% of the mild AD
patients had stabilized or improved cognitive function tests even after 20 months of enrollment
in the Alzhemed™ open-label Phase II extension 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.
Paul Aisen, M.D., Professor of Neurology and Medicine and Director, Memory Disorders Program at
Georgetown University Medical Center in Washington, D.C., presented data from the on-going open-label
Phase II extension study for Alzhemed™. The data are based on the evaluation of cognitive and
global performance as measured by the ADAS-cog3 and CDR-SB4 tests,
respectively.
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 = 19) 5 showed an average ADAS-cog score of +6.2 points, as
opposed to +11.9 points on average in comparable historical controls6 with AD. A subset
of mild AD patients (n = 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 = 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 = 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 reported7
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 II 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 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.
The 9th International Conference on Alzheimer's Disease and Related Disorders, sponsored by the
Alzheimer's Association, is the largest gathering in history of Alzheimer's Disease researchers. More
than 4,500 scientists from around the world are present at the conference.
Live Web Conference
Neurochem will hold a conference call on Monday, July 19, 2004, at 10:00 A.M., EDT, to discuss the
latest interim clinical results of the Phase II open-label extension study on Alzhemed™. The
conference call will be webcast simultaneously.
During the call, Dr. Francesco Bellini, Chairman and Chief Executive Officer of Neurochem, and Dr.
Denis Garceau, Neurochem's Vice President, Drug Development, will summarize and comment on the new
clinical results. Francine Gervais, Ph.D., Neurochem's Vice President, Research and Development, will
also be available following the presentations for questions from callers.
To participate in the conference call and webcast, please dial 1 877-888-4210 approximately 10 minutes
prior to the start of the call AND access Neurochem's website at www.neurochem.com. The dial-in number
will allow participants to listen and ask questions, while the webcast will provide a visual
presentation.
A replay of the web cast will be available on Neurochem's website at 12:00 P.M., EDT. This webcast
will include the conference call and the visual presentation. For those wanting to access the audio
portion only of the conference call, a replay will be available from 12:00 P.M. EDT. Interested parties
can dial 1-866 816-8948 or 416 695-9675, access code: 8490#. The replay will be available for
72 hours.
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 (Aß) 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.
About Alzheimer's Disease
Alzheimer's Disease (AD) is a brain disorder in which nerve cells in the brain die, making it
difficult for the brain's signals to be transmitted properly. A person with AD has problems with
memory, judgment, thinking, and eventually with motor functions, making it difficult for the person
to work or continue to take part in day-to-day life.
According to the National Institute on Aging's "Progress Report on Alzheimer's Disease, 2000,"
AD is the most common cause of dementia among people aged 65 and older. It presents a major health
problem because of its enormous impact on individuals, families, the health care system, and society
as a whole. Scientists estimate that up to four and a half million people in the United States alone
currently suffer with the disease and the prevalence (the number of people with the disease at any
one time) doubles every five years beyond age 65. It is also estimated that approximately 360,000 new
cases (incidence) will occur each year in the United States and that this number will increase as the
population ages.
In a 2000 report, the Biotechnology Industry Organization (BIO) estimated that AD is becoming the most
widespread and costly age-related disorder in the United States; the total cost of the disease has been
estimated at US$100 billion per year.
About Neurochem
Neurochem is focused on the development and commercialization of innovative therapeutics for
neurological disorders. The Company's pipeline of proprietary, disease-modifying, oral products
addresses critical unmet medical needs. Fibrillex™, designated as an orphan drug and as a Fast
Track Product candidate, is also part of a Continuous Marketing Applications Pilot 2 program and is
currently in a Phase II/III clinical trial for the treatment of AA Amyloidosis. Alzhemed™, for
the treatment of Alzheimer's Disease, is in a Phase III clinical trial and Cerebril™, for the
prevention of Hemorrhagic Stroke caused by Cerebral Amyloid Angiopathy, has completed a Phase II
clinical trial.
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 website at: www.neurochem.com.
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, 2003.
This news release contains forward-looking statements regarding the potential for Alzhemed™ and
further development efforts. These statements are based on the current expectations of management. Drug
development involves numerous risks and uncertainties, which could cause actual results to differ
materially. Promising results and successes in early stage clinical trials do not ensure that later
stage or larger scale clinical trials will be successful. For instance, Alzhemed™ may not show the
same benefits, results or safety profile in later stage or larger scale clinical trials. Unexpected
concerns may arise during clinical trials or in the course of developing Alzhemed™ which would
delay the start of later stage or larger scale clinical trials. Additional risks and uncertainties
include: the impact of general economic conditions, general conditions in the pharmaceutical
industry, changes in the regulatory environment in the jurisdictions in which Neurochem does
business, stock market volatility, fluctuations in costs, and changes to the competitive environment
due to consolidation or otherwise. Neurochem does not undertake any obligation to publicly update
its forward-looking statements, whether as a result of new information, future events, or
otherwise.
For further Information, please contact:
Dr. Lise Hébert
Vice President, Corporate Communications
lhebert@neurochem.com
275 Armand-Frappier
Laval (Quebec)
H7V 4A7
Tel: (450) 680-4500
Fax: (450) 680-4501
|
