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January 14, 2004
Neurochem's Data Safety Monitoring Board issues third recommendation to continue pivotal
phase 2/3 Clinical Trial for Fibrillex. About Fibrillex
The pivotal Phase II/III clinical trial for Fibrillex is a two-year, international, multicenter,
randomized, double-blind, placebo-controlled, and parallel-designed trial to evaluate the safety
and efficacy of Fibrillex in patients suffering from AA Amyloidosis. Neurochem has successfully
completed the enrollment of more than the 150 patients required for this pivotal Phase II/III trial,
which is being conducted at 27 sites located across North America, Europe and Israel. This trial is
planned to end in January 2005. Fibrillex has received the orphan drug designation in the United States
and Europe, which normally entails market exclusivity on this product candidate for seven years and
ten years respectively, once it is approved. About AA Amyloidosis
AA Amyloidosis is associated with chronic inflammatory diseases (e.g., rheumatoid arthritis,
ankylosing spondylitis, inflammatory bowel diseases), chronic infections (e.g., osteomyelitis,
tuberculosis) and Familial Mediterranean Fever. The most common clinical presentation of AA
amyloidosis is kidney malfunction. Involvement of the gastrointestinal system is also frequent
and is usually manifested as chronic diarrhea, gastrointestinal bleeding, abdominal pain and
malabsorption. Enlargement of the liver and the spleen may also occur in some patients.
Patients suffering from AA Amyloidosis have a poor prognosis with five- to 15-year survival
rates of 50% and 25%, respectively. End-stage renal failure is the cause of death in 40% to
60% of cases. There is currently no therapy for the treatment of AA Amyloidosis. 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 for
the treatment of Alzheimer's Disease. Cerebril is in a Phase II trial for the prevention of
hemorrhagic stroke caused by cerebral amyloid angiopathy. For additional information on Neurochem,
please visit our website at: (www.neurochem.com).
All of the statements contained in this news release, other than statements of fact that are independently verifiable at the date hereof, are forward-looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown. Some examples of known risks are: 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. Consequently, actual future results may differ materially from the anticipated results expressed in the forward-looking statements.
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