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December 7, 2004
Neurochem announces completion of Phase II/III clinical trial for Fibrillex. About the "FAST" Trial for Fibrillex
The Phase II/III clinical trial for Fibrillex was a two-year, international, multi-center, randomized,
double-blind, placebo-controlled, and parallel-designed trial to evaluate the efficacy and safety of the investigational
product candidate in patients suffering from AA Amyloidosis confirmed by biopsy and renal involvement. Neurochem
successfully completed the enrollment of 183 patients for the Phase II/III trial, which was conducted at 27 sites
located across North America, Europe, North Africa and Israel. The most frequent underlying diseases in patients
during the trial were rheumatoid arthritis and Familial Mediterranean Fever (49% and 19%, respectively)1.
The mean time from onset of underlying disease until AA Amyloidosis was 14.6 ± 10.6 years and nephrotic syndrome was
present in 32% of the participating patients. About Fibrillex
Fibrillex is an oral product candidate for the treatment of AA Amyloidosis through the prevention of amyloid
fibril formation. Fibrillex belongs to the class of glycosaminoglycan (GAG) mimetics, better known as amyloid
disease-modifiers. By mimicking GAGs, Fibrillex prevents the AA fibril formation and the deposition of AA
fibrils in organs, which, when left untreated, leads to organ failure. About AA Amyloidosis
AA Amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with chronic inflammatory
diseases, including rheumatoid arthritis, ankylosing spondylitis, juvenile rheumatoid arthritis, and Crohn's disease. The
disease also occurs in patients suffering from many other conditions ranging from chronic infections to inherited
inflammatory diseases such as Familial Mediterranean Fever. The most common clinical presentation of AA Amyloidosis
is renal dysfunction. Involvement of the gastrointestinal system is also frequent and is usually manifested as chronic
diarrhea, abdominal pain and malabsorption. Enlargement of the liver and the spleen may also occur in some patients. 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. 1,3PDS;
Fibrillex designated as an orphan drug and a Fast Track Product candidate, while having completed a Phase II/III
clinical trial for the treatment of AA Amyloidosis with data expected to be released in the second quarter of 2005, is
also part of an FDA Continuous Marketing Applications Pilot 2 program. 3-amino-1-propanesulfonic acid (3APS;
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. 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 - As recently presented at the American Society of Nephrology's Renal Week 2004 Conference.
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