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Independent Review Board Recommends Phase II/III Study To Continue of Fibrillex in Amyloid A Amyloidosis
"I am very pleased with this third positive recommendation from our Data Safety Monitoring Board," stated Dr. Francesco Bellini, Chairman and CEO of Neurochem. "At the same time, I regret the market volatility we are seeing on Neurochem shares at the present time. I want to reassure the financial community and our shareholders that all of our programs are on schedule and that there are no company events nor any pending news to justify the current stock volatility. I am very pleased with the progression of all of the Company's programs."
Dr. Denis Garceau, Neurochem's Vice President, Drug Development, added: "The continued positive and unanimous recommendation of our independent Data Safety Monitoring Board for the continuation of this Phase II/III clinical study is encouraging. AA Amyloidosis is a fatal disease with no treatment available and we are on target for the commercialization of this product late next year. People who suffer from uncontrolled chronic inflammation such as those with Rheumatoid Arthritis who do not respond to their therapy, are at high risk to develop this disease," he stated.
The DSMB is comprised of independent medical experts and was established by Neurochem as part of the Company's compliance with good clinical practices guidelines. The DSMB is responsible for monitoring and reviewing the safety of patients participating in the Phase II/III clinical trial.
The pivotal Phase II/III clinical trial for Fibrillex is a two-year, international, multicenter, 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 more than the 150 patients required for this pivotal Phase II/III trial, which is being conducted at 27 sites located across North America, Europe and Israel. This trial is planned to end in January 2005. Fibrillex has received the orphan drug designation in the United States and Europe, which normally entails market exclusivity on this product candidate for seven years and ten years respectively, once it is approved.
Fibrillex is an oral drug candidate for the treatment of AA amyloidosis through the prevention of amyloid fibril formation. Fibrillex belongs to the class of glycosaminoglycan (GAG) mimetics which competes with natural GAGs for the same binding sites on the AA protein. By preventing natural GAGs from binding to the AA protein, Fibrillex is expected to prevent AA fibril formation and the deposition of AA fibrils in organs.
About AA Amyloidosis
AA Amyloidosis is associated with chronic inflammatory diseases (e.g., rheumatoid arthritis, ankylosing spondylitis, inflammatory bowel diseases), chronic infections (e.g., osteomyelitis, tuberculosis) and 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. Patients suffering from AA Amyloidosis have a poor prognosis with five- 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 currently no therapy for the treatment of AA Amyloidosis.
Source: Neurochem Inc