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Our ultimate goal: to answer critical unmet medical needs

Over the last years, Neurochem has made significant advances on its lead product candidates, which are at various stages of development.

These product candidates share one purpose: they aim to address vital unmet medical needs for diseases to which no cure exists, especially in the field of neurology.

Regarding research and development programs, Neurochem is at the forefront of research on neurological diseases.

AA amyloidosis

AA amyloidosis Brochure

Eprodisate (KIACTA™) is under regulatory review for marketing approval by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMEA) and Swissmedic for the treatment of Amyloid A (AA) amyloidosis, a disease associated with chronic inflammatory diseases, including rheumatoid arthritis and Crohn's disease.

United States Regulatory Path and Milestones

December 2007	Committee for Medicinal Products for Human Use (CHMP) issues negative opinion recommending refusal of the marketing authorization application for eprodisate (KIACTA™). The Company is currently reviewing its options, including the option of requesting a re-examination of the opinion.
July 2007	FDA issues second approvable letter to which the Company submitted a response in September 2007. Letter stated: -FDA indicated that the Phase II/III clinical trial provided some evidence of the effectiveness of eprodisate (KIACTA™) in the treatment of the renal manifestations of amyloidosis. -FDA also indicated that an additional efficacy trial with a target p-value of 0.05 will be necessary before the FDA could approve the investigational product candidate. -Additional submissions, filed by Neurochem as part of its complete response to this approvable letter, may address issues raised in this letter. The FDA has indicated that such additional submissions could persuade the agency to eliminate the requirement for an additional trial. -FDA also asked for additional information, including further pharmacokinetic studies, and again acknowledged that a QT clinical study should be submitted as part of a Phase IV (post-approval) commitment.
April 2007	Neurochem advised that new PDUFA date extended to July 16, 2007; original PDUFA date was April 16, 2007.
November 2006	Neurochem advised that amendment submitted in October 2006 was a complete response; answers all questions raised in approvable letter of August 2006. Class II review (PDUFA date: April 16, 2007).
August 2006	Neurochem receives first approvable letter for eprodisate (KIACTA™) for the treatment of AA amyloidosis. Letter stated: -FDA requested additional efficacy information, as well as a safety update. -FDA stated that this efficacy information would probably need to be addressed by one or more additional clinical trials. -As an alternative, the FDA also stated that significant findings obtained from a complete follow-up of patients in the existing study could be persuasive. -FDA asked for further manufacturing and pharmacokinetic information, and acknowledged that a QT clinical study should be submitted as part of a Phase IV (post approval) commitment.
April 2006	Eprodisate (KIACTA™) NDA granted Priority review by FDA.
February 2006	Neurochem submitted final modules of ‘rolling’ NDA.

Neurochem is also seeking marketing approval for eprodisate (KIACTA™) in the European Union and Switzerland. With regards to Europe, the Company was advised by the EMEA in September 2006 that its Marketing Authorization Application is valid and that the regulatory review has started. A decision is expected from Switzerland in 2008.

The Phase II/III clinical trial for eprodisate (KIACTA™) was a two-year, international, multicenter, randomized, double-blind, placebo-controlled, and parallel-designed trial to evaluate the efficacy and safety of the investigational product candidate in patients suffering from AA amyloidosis confirmed by biopsy and renal involvement. Neurochem successfully completed the enrolment of 183 patients for the Phase II/III trial, which was conducted at 27 sites located across North America, Europe, North Africa and Israel.

All patients who completed the trial have been invited to join the ongoing open-label Phase II/III extension study.

Eprodisate (KIACTA™) has received orphan drug status in the United States, the European Union and Switzerland.

Other significant milestones achieved for eprodisate (KIACTA™)

• Phase II/III clinical trial completed in 2005.

  
  

• Eprodisate (KIACTA™) was 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 and marketing of this product candidate. Under this Pilot 2 program, each FDA division is permitted to select only one product candidate.

  
  

• Neurochem received four recommendations from its independent Data Safety Monitoring Board (DSMB) to continue the Phase II/III clinical trial. The role of the DSMB is to monitor the safety of patients participating in this study and review the safety data throughout the duration of the study.

  
  

• The FDA designated eprodisate (KIACTA™) as Fast Track Product.

  
  

• Neurochem received a prestigious $1.4 Cdn million grant from the FDA for the Phase II/III study of eprodisate (KIACTA™).

  
  

• In the four human clinical Phase I studies, the product candidate underwent rigorous toxicity and pharmacokinetic testing.

  
  

Why target AA amyloidosis?

AA amyloidosis is a fatal condition for which there is no effective therapy. This condition is a consequence of chronic inflammatory diseases such as rheumatoid arthritis, ankylosing spondylitis, inflammatory gastrointestinal diseases, and chronic infections such as osteomyelitis and tuberculosis. The hereditary condition known as Familial Mediterranean Fever can also lead to AA amyloidosis. Patients with rheumatoid arthritis are reported to represent the majority - approximately 50% - of the thousands of patients suffering from AA amyloidosis. With the impact of shifting demographics to an aging population in the next decade, the number of people with chronic inflammatory diseases such as rheumatoid arthritis will dramatically increase.

Because the amyloid fibrils in AA amyloidosis mostly deposit in the liver, spleen and kidneys, the most common clinical presentation of this lethal disorder is kidney failure. Gastrointestinal complications are also frequent and are 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. Left untreated, patients gradually progress to end-stage renal failure, which is the cause of death in at least 35% of the cases.

Alzheimer's disease

Alzheimer’s disease brochure

Leveraging the numerous years of accumulated knowledge and the experience it has gained in developing the pharmaceutical product candidate, tramiprosate, for AD, Neurochem is prioritizing and accelerating the development of its next generation lead compound developed as a new chemical entity, or NCE, and a prodrug of tramiprosate for the treatment of AD. A prodrug is a pharmaceutical substance which is administered in an inactive form and once absorbed is metabolized in vivo into its active form.

The Company is committed to analyzing and understanding the wealth of data generated by the tramiprosate North American Phase III clinical trial completed in April 2007. As announced previously, descriptive data from the North American Phase III clinical trial shows numerical differences in favor of tramiprosate on the primary clinical endpoints. The descriptive data also shows by magnetic resonance imaging differences between groups on the primary disease modification endpoint of change in the volume of the hippocampus, a structure of the brain that is considered to be important in memory function.

Additional findings obtained from a preliminary post-hoc analysis performed by the Neurochem's external team of statisticians that allowed adjustment for potential confounding factors showed a dose-dependent reduction in hippocampal atrophy in patients treated with tramiprosate. When compared to placebo, patients treated with 200 mg of tramiprosate per day experienced significantly less atrophy (relative difference of 65%; P = 0.036) and patients treated with tramiprosate with 300 mg of tramiprosate per day presented no atrophy (P = 0.003).

The Company is pursuing several post-hoc analyses which are revealing promising preliminary results and is currently working to submit results for publication in a peer-reviewed journal.

In view of these results, coupled to the large number of physicians and families requesting access to the compound, and given that tramiprosate occurs naturally in certain foods, Neurochem is planning to provide commercial access to tramiprosate as a branded nutraceutical product, potentially as early as 2008, via the creation of a new self-sustaining company. This decision was taken in view of the fact that AD is a devastating disease and remains an unmet medical need.

In November 2007, Neurochem announced the early termination of the European Phase III clinical trial and that it will complete the ongoing North American open-label extension study.

Alzheimer's disease: Some facts

AD is a leading cause of death in the elderly. The disease is characterized by the progressive death of nerve cells in the brain, making it difficult for the brain's signals to be transmitted properly. A person with AD experiences problems with memory, judgment, thinking, and eventually with motor functions, all of which make it difficult for the person to participate in daily activities.

According to the U.S. Alzheimer Association (2007), there are now more than five million people in the United States living with Alzheimer's disease. This number includes 4.9 million people aged 65 and older. It also includes at least 200,000 individuals younger than 65 with early-onset Alzheimer's disease. It is estimated that there will be 454,000 new cases of AD a year by 2010, 615,000 new cases by 2030 and 959,000 new cases by 2050. In the United States, the direct and indirect costs of AD and other dementias amount to more than $148 US billion annually.

Diabetes Type II/Features of Metabolic Syndrome

As announced in May 2007, eprodisate has shown beneficial effects in preclinical in vivo models of Diabetes Type II/features of metabolic syndrome. Preliminary results have shown that eprodisate protects the kidney function in obese diabetic rats. As well, eprodisate has shown an impact on metabolic changes associated with diabetes and obesity, including a significant decrease in triglyceride levels and cholesterol, a significant decrease in glycemia and an increase in insulin plasma levels. The Company is on track to initiate a Phase II clinical trial in diabetic patients in Canada in early 2008.

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