Newsroom 2005
Ecublens (Switzerland) - Neurochem (International) Limited (Neurochem), a wholly-owned subsidiary of Neurochem Inc.
(NASDAQ: NRMX; TSX: NRM), today announced that the U.S. Food and Drug Administration (FDA) has agreed to file and
review a New Drug Application (NDA) for Fibrillex™ that would include the efficacy and safety data from Neurochem's
single Phase II/III trial. Fibrillex(TM) is Neurochem's investigational product candidate for the treatment of Amyloid A
(AA) Amyloidosis. This announcement comes as a result of a recent meeting with the Cardio-Renal Division of the FDA to
discuss the results of Neurochem's Phase II/III clinical trial. The Agency encouraged Neurochem to look at and provide
additional follow-up data collected from the open-label extension study as part of its NDA submission.
"AA Amyloidosis is a serious medical condition that frequently leads to organ dysfunction, subsequent death and for
which no specific treatment exists," said Denis Garceau, PhD, Neurochem's Senior Vice President, Drug
Development. "The filing of our first NDA is a significant event for Neurochem. We are therefore very pleased that the
FDA has agreed to file and review a NDA for Fibrillex™, recognizing its Orphan Drug Status. We look forward to working with
the regulatory agencies and hope to make this potential treatment available to over 40,000 patients in North America and
Europe presently diagnosed with the disease."
As previously announced in December 2004, Neurochem shares an exclusive collaboration and distribution agreement for
Fibrillex™ with Centocor, Inc., of Malvern, PA (USA).
About Fibrillex™
Fibrillex™ is an oral investigational product candidate for the treatment of AA Amyloidosis through the prevention
of amyloid fibril formation. It has received Orphan Drug Status designation in the United States and Orphan Medicinal
Product designation in Europe.
Fibrillex™ also has been accorded "Fast Track Product" designation by the FDA and has been selected by the
Cardio-Renal Drug Product Division of the FDA to be part of the Continuous Marketing Applications Pilot 2 program aimed
at further accelerating the development of this product candidate. Under this Pilot 2 program, each FDA division is permitted
to select only one product candidate.
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, gastrointestinal
bleeding, abdominal pain and malabsorption. Enlargement of the liver and the spleen may also occur in some patients.
End-stage renal failure is the main cause of death in 40-60% of cases. The median survival time from diagnosis varies from
2 to 10 years depending on the stage of the disease at the time of diagnosis.
No specific treatment is currently available for this orphan disease. The goal of existing therapies is limited to the
control of the underlying chronic inflammatory disease.
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.
1,3-propanedisulfonate (1,3PDS; Fibrillex™) is designated as an orphan drug and a Fast Track Product candidate
and is also part of an FDA Continuous Marketing Applications Pilot 2 program. The Phase II/III clinical trial of
Fibrillex™ for the treatment of AA Amyloidosis was recently concluded and preliminary results have been issued
in April 2005. 3-amino-1-propanesulfonic acid (3APS; Alzhemed™), for the treatment of Alzheimer's Disease, is in
a Phase III clinical trial and 3APS (Cerebril™), for the prevention of Hemorrhagic Stroke caused by Cerebral
Amyloid Angiopathy, has completed a Phase IIa 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.
This news release contains forward-looking statements regarding Fibrillex™, as well as continuing and further
development efforts. These statements are based on the current analysis and expectations of management. Drug development
necessarily involves numerous risks and uncertainties, which could cause actual results to differ materially from this
current analysis and these expectations. Analysis regarding the results of clinical trials may not provide definitive results
regarding safety, tolerability or therapeutic benefits. There can be no assurance that a NDA for Fibrillex™ would be granted
or that any regulator would ultimately approve it for sale to the public. Risks and uncertainties may include: failure to
demonstrate the safety, tolerability and efficacy of our product, the expense and uncertainty of obtaining regulatory approval,
including from the FDA, and the possibility of having to conduct additional clinical trials. Additionally, even if
regulatory approval is obtained, therapeutic products are generally subject to: stringent on-going governmental regulation,
challenges in gaining market acceptance, and competition. Neurochem does not undertake any obligation to publicly update its
forward-looking statements, whether as a result of new information, future events, or otherwise. Please see the Annual Information
Form for further risk factors that might affect the Company and its business.
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
