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July 7, 2004
Neurochem's Fibrillex™ selected for new FDA Pilot 2 program.
Fibrillex™ Already Granted Fast-Track Product Designation.
- Neurochem Inc. (NASDAQ: NRMX; TSX: NRM) announced today that Fibrillex™, the Company's
investigational product candidate for the treatment of Amyloid A (AA) Amyloidosis, has been selected
by the Cardio-Renal Drug Product Division of the US Food and Drug Administration (FDA) to be part of
the Continuous Marketing Applications Pilot 2 program aimed at further accelerating the development
and eventual marketing of this product candidate. Under this Pilot 2 program, each FDA division is
permitted to select only one product candidate.
The selection of Fibrillex™ by the FDA is based in part on the fact that it has been designated
as a Fast Track Product due to the life-threatening nature of AA Amyloidosis, an unmet medical need
which often results in end-stage renal disease. The decision is based on other criteria as well. For
example, the FDA looked at the potential value of enhanced interaction with Neurochem and emphasized
the potential public health benefit from the development of the product and the likelihood that
concentrated scientific dialogue could facilitate the availability of Fibrillex™ as a promising
novel therapy. Fibrillex™ is presently in an on-going Phase II/III clinical trial which Neurochem
expects to complete by January 2005.
"We are very pleased that the FDA's Cardio-Renal Division has accepted Fibrillex™ to be
part of this new pilot project under which Neurochem and the FDA will engage in frequent scientific
feedback and interaction during the development of this product candidate," said Francesco
Bellini, Ph. D., Chairman and CEO of Neurochem. "This frequent communication based on a
prospectively defined agreement between the FDA and Neurochem should allow us to agree on information
to be presented in our NDA submission. These on-going interactions could further expedite the FDA's
review of Fibrillex™."
Fibrillex™ has already received orphan drug status designation in the United States and Orphan
Medicinal Product designation in Europe, which normally provide a drug seven and ten years of
market exclusivity, respectively, upon market entry.
About Fibrillex™
The Phase II/III clinical trial for Fibrillex™ is a two-year, international, multi-center,
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 183 patients for the Phase II/III trial, which is being conducted at 27
sites located across North America, Europe and Israel. Neurochem anticipates completing the trial by
January 2005. To date, approximately 42 patients have completed the two-year Phase II/III trial for
Fibrillex™. A two-year open-label extension study for the product candidate is on-going.
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 disease-modifiers. By mimicking GAGs, Fibrillex™ competitively binds to the AA
protein, preventing natural GAGs from binding to the same AA protein. Fibrillex™ is expected to
prevent AA fibril formation and the deposition of AA fibrils in organs.
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 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.
This disease has a poor prognosis, with a five-year survival rate of approximately 50%.1
Left untreated, patients gradually progress to end-stage renal failure, which is the cause of death
in at least 35% of the cases.2, 3
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. Fibrillex™, designated as an orphan drug and a Fast
Track Product candidate, is also part of a Continuous Marketing Applications Pilot 2 program and is
currently in a Phase II/III clinical trial for the treatment of AA Amyloidosis. 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 website at: www.neurochem.com.
1 - Pepys, M., et al. Amyloidosis. Oxford Textbook of Medicine (4th ed.) Oxford:
Oxford University Press 2003; 162-73.
2 - Gertz, M.A., et al. Medicine (Baltimore) 1991 Jul: 70(4); 246-56.
3 - Joss, N., et al. QJMed. 2000: 93; 535-42.
This news release contains forward-looking statements regarding the potential for Fibrillex™ 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 from expectations. Promising results and successes in early stage clinical trials
do not ensure that later stage or larger scale clinical trials will be successful. 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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