AA Amyloidosis Drug, Neurochem's Fibrillex, Selected for New FDA Pilot 2 Program

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Neurochem's Fibrillex Selected for New FDA Pilot 2 Program

Fibrillex Already Granted Fast-Track Product Designation

Source: Neurochem Inc. http://biz./prnews/040707/to109_1.html

MONTREAL, July 7 /PRNewswire-FirstCall/ - Neurochem Inc. 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)

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.