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July 17, 2001
Neurochem Provides Update On Clinical Treatment for Secondary Amyloidosis
-- Safety data from Company's Fibrillex™ program reported at the IXth International
Symposium on Amyloidosis in Budapest --
Neurochem Inc. (TSX : NRM) today announced positive clinical Phase I safety data for Fibrillex™
, its therapeutic in development for the treatment of Secondary Amyloidosis. Secondary Amyloidosis
is a potentially fatal disease associated with chronic infection or chronic inflammatory disease
in which amyloid fibrils deposit in tissues and organs, imparing their function. Animal models
have demonstrated Fibrillex™ ability to prevent the formation of amyloid fibrils associated
with Secondary Amyloidosis. Safety data presented today shows that in both healthy and renal-impaired
human volunteers, Fibrillex™ was well tolerated, with a side effect profile comparable
to that of placebo. Fibrillex™ has advanced to late-stage clinical trials, with a
pivotal Phase II/III study currently underway.
Dr. Denis Garceau, Vice-President, Drug Development, reported the data at the IXth International
Symposium on Amyloidosis in Budapest Hungary, an event of international stature with world-renowned
speakers and participants in the expanding field of amyloid-related research and its clinical aspects.
Supported in part by Neurochem Inc., a Canadian based industry leader in biopharmaceuticals, this
symposium is devoted to all aspects of amyloid and amyloidosis and related disciplines. This event
is being held at the Hotel Inter-Continental Budapest, Hungary from July 15-21 2001. The Company will
be presenting a total of 7 original abstracts including 2 oral presentations on their innovative
amyloid-based research at the conference in Budapest this week.
The results of the four clinical Phase I studies reported today exposed both a total of 80 healthy
volunteers and 16 renal impaired human subjects to varying dosage levels of the small drug candidate.
The side effect profile for Fibrillex™ was similar to that of the placebo, with no apparent
dose-related trends in the occurrence and the severity of side effects. No serious side effects were reported
and no subjects were withdrawn prematurely due to side effects in any of the studies. This positive
safety profile, in conjunction with pre-clinical data demonstrating Fibrillex™ ability
to prevent the formation of amyloid fibrils associated with Secondary Amyloidsis in mice, has advanced
the product to late-stage clinical trials.
Amyloid is a class of human proteins with normal function in their soluble forms. Amyloidosis
is a diverse group processes characterized by extracelluar tissue deposits of amyloid fibrils
including Alzheimer's Disease, amyloid-associated Diabetes, Hemorrhagic Stroke due to Cerebral Amyloid
Angiopathy (CAA), Systemic Amyloidosis, and a number of other common and rare disorders.
Secondary Amyloidosis is associated with chronic inflammatory disease (e.g. rheumatoid arthritis,
ankylosing spondylitis, inflammatory bowel disease) and chronic infections (e.g. osteomyelitis,
tuberculosis) and familial Mediterranean fever. The most common clinical presentation of Secondary
Amyloidosis is nephrotic-range proteinuria with or without renal insufficiency. Gastrointestinal
involvement 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. Without control of the acute phase response, Secondary Amyloidosis has a poor prognosis
with 5- 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 no known specific effective therapy for Secondary Amyloidosis
at the present time.
Fibrillex™ received Orphan Drug Designation in the U.S. in 1999, and is currently being tested
for the treatment of Secondary Amyloidosis in a pivotal Phase II/III clinical trial. For more information
about this clinical trial, please call the Secondary AA Amyloidosis Clinical Trial Toll-Free Information Line
(North America) at 1-866-234-AMYL (2695) or visit www.neurochem.com.
About Neurochem
Neurochem is a leader in the development of novel, proprietary compounds that inhibit the
formation, deposition and toxic effects of amyloid fibrils within the body. Neurochem's innovative
core technology consists of the design and synthesis of compounds that mimic the properties of
sulphated glycosaminoglycans or GAGs, complex carbohydrates that promote the amyloid fibril formation
that is characteristic of Secondary Amyloidosis, Alzheimer's Disease and other indications. Neurochem's
synthetic compounds compete with the naturally occurring GAGs by interfering with the formation and
deposition of amyloid aggregates. This biochemical "deception" has been proven successful in studies
showing the compounds disrupting the formation of indicators of Secondary Amyloidosis, and many other
detrimental amyloid-based compounds. Neurochem has an extensive pipline of amyloid-based therapeutics.
At present, the Company employs over 73 people and is located in Saint-Laurent, Canada
(www.neurochem.com).
All of the statements contained in this news release, other than statements of fact which
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.
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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