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November 4, 2003
Interim results of Alzhemed™ open-label phase 2 extension study demonstrates cognitive
benefits in patients with Alzheimer's disease.
- Neurochem, Inc. (NASDAQ: NRMX, TSX: NRM) - announced today positive results on cognitive
function as measured by ADAS-cog1 of patients suffering from
mild-to-moderate Alzheimer's Disease (AD) in an open-label Phase II extension study2,3. Neurochem is reporting
on the first 10 patients out of the 33 enrolled in the ongoing study who have now completed 9
months of treatment on the highest dose (300 mg daily) of the investigational product candidate,
Alzhemed™.
The data has revealed that patients with mild AD4 (n=6) showed
the greatest benefits from Alzhemed™ as compared to patients with moderate AD
patients5 (n=4). Indeed, all mild AD patients in the study
showed improvement in their cognitive function (-3.5 points on average) after 9 months of therapy
with Alzhemed™. This result compares favorably with that of historical control data from a
longitudinal study with mild AD patients who showed an average decline in their cognitive function
of approximately +4.6 points over the same period of time6.
Overall, the cognitive function in the combined cohort of mild-to-moderate patients on
Alzhemed™ was improved by 59% when compared to the decline exhibited by historical, untreated
control patient groups after 9 months of follow-up.
"While this interim data was obtained in a small number of patients, and while the open-label
Phase II extension study with Alzhemed™ was neither powered nor designed to detect
statistically significant clinical benefits in psychometric tests, we are extremely encouraged by
the results," said Denis Garceau, Ph.D., Vice President, Drug Development at Neurochem.
"The promising results released today are consistent with the data presented in July 2003
after 6 months of treatment. Furthermore, the good safety profile of Alzhemed™ combined with
the high rate of response among patients, indicates potential for a better treatment regimen for
AD patients. We expect to complete the first year of the open-label Phase II extension study in
the first quarter of 2004, and to initiate our Phase III trial in the spring of
2004."
Neurochem expects to release additional interim data as the results from other groups of patients
become available and a full report of the study in the first quarter of 2004.
"Alzheimer's Disease is one of the scourges of our age," said Dr. Paul S. Aisen,
Professor of Neurology and Medicine, Georgetown University Medical Center. "Alzhemed™,
by targeting the amyloid protein, has shown promising results in clinical trials as a drug that
may favourably influence the disease process. Literally millions of patients would stand to gain
if the results of Neurochem's Phase III trials are positive.™
About Alzhemed™
Alzhemed™ is an orally administered, small synthetic molecule that has been specifically
designed to modify the course of AD by interfering with the association between glycosaminoglycans
(GAGs) and Aß protein. As part of a "disease modifying" class of product candidates,
Alzhemed™ is expected to act at two levels: in preventing and stopping the formation and
deposit of amyloid fibrils in the brain and in binding to soluble Aß protein to inhibit the
inflammatory response associated with amyloid build-up in AD. Its anti-amyloid potential has been
demonstrated in an aggressive transgenic mouse model of brain amyloid with a significant (61%)
reduction in Aß plasma levels and a 30% reduction in Aßß brain soluble and
insoluble fractions after only eight weeks of treatment.
About Neurochem
Neurochem is focused on the development and commercialization of innovative therapeutics for
neurological disorders. The Company's staged pipeline of proprietary, disease-modifying, oral
products addresses critical unmet medical needs. Fibrillex™, designated an orphan drug, is in
a Phase II/III clinical trial for AA amyloidosis. Alzhemed™ has completed a Phase II clinical
trial. Cerebril™ is in a Phase II trial for the prevention of hemorrhagic stroke caused by
cerebral amyloid angiopathy. Neurochem is also developing a product for the potential treatment of
epileptic seizures induced by traumatic brain injury. For additional information on Neurochem,
please visit our website 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. 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.
2 - In an open-label extension study, in contrast to a blind study, all patients are treated with
an investigational drug candidate, and no patients receive a placebo.
3 - In June 2003, Neurochem announced the results of its Phase II clinical trial for
Alzhemed™ in 58 patients with mild-to-moderate AD who had been treated with doses of either
100, 200 or 300 mg of Alzhemed™. The study revealed promising results for both the primary
and secondary objectives of the trial. There were no safety findings of concern in patients
treated with Alzhemed™. The pharmacokinetic profile of Alzhemed™ was well characterized
and it was detected in the cerebrospinal fluid (CSF) of the patients, suggesting an ability to
cross the blood brain barrier. Patients with the greatest decrease in amyloid protein in the CSF
were all on Alzhemed™.
4 - Mini Mental State Exam (MMSE): 19-25
5 - Mini Mental State Exam (MMSE): 13-18
6 - Stern, R.G., et al. Am.J.Psychiatry 151:3, March 1994.
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
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