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Ongoing Phase 2 Study of Alzhemed Shows Positive Interim Results in Alzheimer’s Disease Patients
PHILADELPHIA, PA, July 19 /PRNewswire-FirstCall/ -- Neurochem Inc. (NASDAQ: NRMX; TSX: NRM) announced today that interim results of an on-going open-label Phase II extension study of the effects of Alzhemed™ on cognitive function and global measure of performance in patients with mild(1)-to-moderate(2) Alzheimer's Disease (AD) were presented at the 9th International Conference on Alzheimer's Disease and Related Disorders (ICAD) in Philadelphia. The study showed that overall, approximately 70% of the mild AD patients had stabilized or improved cognitive function tests even after 20 months of enrollment in the Alzhemed™ open-label Phase II extension study.
Alzhemed™ is an investigational oral product candidate for mild-to- moderate AD that has been specifically designed to modify the course of the disease through its anti-amyloid activity. Phase III trials on the product candidate were launched in 70 clinical centers in North America last month.
Paul Aisen, M.D., Professor of Neurology and Medicine and Director, Memory Disorders Program at Georgetown University Medical Center in Washington, D.C., presented data from the on-going open-label Phase II extension study for Alzhemed™. The data are based on the evaluation of cognitive and global performance as measured by the ADAS-cog(3) and CDR-SB(4) tests, respectively.
The study followed 19 mild-to-moderate AD patients who received study medication for 20 months. The mild-to-moderate AD patients (n (equal sign) 19)(5) showed an average ADAS-cog score of +6.2 points, as opposed to +11.9 points on average in comparable historical controls(6) with AD. A subset of mild AD patients (n (equal sign) 10) responded best and showed a change from baseline in their average ADAS-cog score of +2.4 points. This result compares favorably with a score of +8.6 points on average in comparable historical controls.
Last April, the Company issued data on the first 18 patients who had completed 16 months. Neurochem is now reporting on all patients with mild-to- moderate AD who received study medication for 16 months. The mild-to-moderate AD patients (n (equal sign) 26) showed an average ADAS-cog score of +5.3 points, as opposed to +9.6 points on average in comparable historical controls with AD. A subset of mild AD patients (n (equal sign) 15) responded well and showed a change from baseline in their average ADAS-cog score of +1.7 points. This result compares favorably with an average score of +7.6 points in comparable historical controls. Overall, nine out of the 15 mild AD patients were stabilized or improved at 16-months.
Global Measure of Performance
The average CDR-SB score in the mild-to-moderate AD patients after 20 months on study medication showed +2.7 points on average. These results compare favorably with the already reported(7) 12-month mean change in the CDR-SB score of +2.2 points change in comparable mild-to-moderate AD patients.
Safety and Tolerability
Alzhemed™ continues to be safe and well tolerated after up to 20 months of follow-up. Nausea and vomiting occurred primarily at the beginning of the treatment and decreased over time. By 16 months, no nausea and vomiting were reported. Overall, five patients withdrew prematurely from the beginning of the Phase II clinical trial over the 20-month period due to adverse events: three due to nausea and vomiting, one because of weakness and weight loss and another for increased agitation and delusion.
"These results in Alzheimer's patients are promising because typically, patients decline significantly over a prolonged period," said Dr. Aisen, who also is principal investigator in the United States for the Phase III study on Alzhemed™. "Alzhemed™ has the potential to modify the progression of AD because it acts directly on its core pathology. Because existing therapies can only treat disease symptoms, Alzhemed™ could become the new paradigm for AD treatment if it is approved by regulatory authorities."
"Given the very long treatment period with Alzhemed™ in our on-going open-label Phase II extension study, and even though the study was not designed to be statistically significant, we are encouraged by the persisting stabilization of the majority of mild AD patients receiving this product candidate. This interim data strengthens our belief that Alzhemed™ is a potential breakthrough to stop the cause of this devastating disease," said Francesco Bellini, Ph. D., Chairman and CEO of Neurochem.
Neurochem's Phase III clinical trial on Alzhemed™ in North America will run for a period of 18 months and be conducted in 50 U.S. and 20 Canadian clinical centers. The Company anticipates launching its Phase III trial in Europe early in 2005.
The 9th International Conference on Alzheimer's Disease and Related Disorders, sponsored by the Alzheimer's Association, is the largest gathering in history of Alzheimer's Disease researchers. More than 4,500 scientists from around the world are present at the conference.
Alzhemed™ is an orally administered, small organic molecule that has been specifically designed to modify the course of AD through its anti-amyloid activity. As part of a "disease modifying" novel class of product candidates, Alzhemed™ is expected to act at many levels: in binding to soluble amyloid beta (A(B)) protein, to prevent and stop the formation and the deposition of amyloid fibrils in the brain, and to reduce the amyloid-induced toxicity on neuronal and brain inflammatory cells associated with amyloid build-up in AD.
About Alzheimer's Disease
Alzheimer's Disease (AD) is a brain disorder in which nerve cells in the brain die, making it difficult for the brain's signals to be transmitted properly. A person with AD has problems with memory, judgment, thinking, and eventually with motor functions, making it difficult for the person to work or continue to take part in day-to-day life.
According to the National Institute on Aging's "Progress Report on Alzheimer's Disease, 2000," AD is the most common cause of dementia among people aged 65 and older. It presents a major health problem because of its enormous impact on individuals, families, the health care system, and society as a whole. Scientists estimate that up to four and a half million people in the United States alone currently suffer with the disease and the prevalence (the number of people with the disease at any one time) doubles every five years beyond age 65. It is also estimated that approximately 360,000 new cases (incidence) will occur each year in the United States and that this number will increase as the population ages.
In a 2000 report, the Biotechnology Industry Organization (BIO) estimated that AD is becoming the most widespread and costly age-related disorder in the United States; the total cost of the disease has been estimated at US$100 billion per year.
1 - Mini Mental State Exam (MMSE): 19-25 2 - Mini Mental State Exam (MMSE): 13-18 3 - Alzheimer's Disease Assessment Scale, cognitive subpart (ADAS-cog). The ADAS-cog is a 70-point scale designed to measure, with the use of questionnaires, the progression and the severity of cognitive decline as seen in AD. The ADAS-cog scale quantifies the number of wrong answers. Consequently, a high score on the scale indicates a more severe case of cognitive decline. When analysing results, a negative score indicates the improvement of cognitive function and a positive score the deterioration of such function. The ADAS-cog has been validated by the regulatory authorities as the gold standard scale for the monitoring of cognitive function in AD patients. This scale is a compulsory parameter of efficacy when submitting for market approval of an AD drug to the authorities such as the Food and Drug Administration. 4 - Clinical Dementia Rating - sum of boxes rating scale (CDR-SB), a measure of global performance. 5 - n: number of patients per group. 6 - Stern, R.G., et al. Am.J.Psychiatry 151:3, March 1994. 7 - Aisen, P.S., et al. JAMA 289: 2819, June 4, 2003.
Source: Neurochem Inc.