Accera, Inc., a biotechnology company delivering therapies in central nervous system diseases, today announced further evidence for genetic interactions impacting the efficacy of the ketogenic compound AC-1202 (Axona(TM)) in Alzheimer’s disease.
New data from the company’s previously completed double-blind, placebo-controlled trial in patients with mild-to-moderate Alzheimer’s disease demonstrates an interaction between two genetic markers that strongly influence the therapeutic response in patients. Dr. Samuel Henderson, Executive Director of Research, will present these results at the 2009 International Conference on Alzheimer’s Disease (ICAD) sponsored by the Alzheimer’s Association.
During this study, patients received daily administration of either AC-1202 or placebo for 90 days, with efficacy assessments performed at Baseline (Day 0), Day 45, Day 90 and after a two week washout from their assigned product on Day 104. In addition, analyses of a number of genotypic markers judged to be relevant to the physiological background of Alzheimer’s disease were also performed.
Previous analysis of the study revealed that patients administered AC-1202 who lacked the epsilon 4 variant of the APOE gene (E4(-)), demonstrated significant improvement from baseline values in the Alzheimer’s Disease Assessment Scale-Cognitive (ADAS-Cog) and improvement compared to placebo of 4.77 at Day 45 and 3.36 at Day 90 (p 0.05).
ADAS-Cog, a neuropsychometric battery of tests that measures short-term memory and cognition, is probably the most widely used cognitive instrument used in clinical trials of anti-dementia drugs within the United States and Europe. Numerous clinical studies have demonstrated that modest improvements in ADAS-cog scores - on the order of 2 to 3 points over the course of a year - have been associated with significant cost reductions in overall managed care expenditures.
To further investigate pharmacogenomic responses of AC-1202 in AD, the effects of APOE4 carriage status and a polymorphism (IDE_7) in the insulin degrading enzyme gene (IDE) on ADAS-Cog scores were evaluated over the study course. In addition to degrading insulin, the Ide protein also degrades amyloid beta peptide and has been implicated in playing a role in Alzheimer’s disease.
In the population of patients who were both APOE4(-) and lacked the C/C polymorphism in IDE 7, more pronounced improvements in ADAS-cog scores than those previously reported were observed at each assessment time point (Day 45, 90 and 104). At Day 45 the improvement in ADAS-cog score was 4.18 (p=0.0004), while at Day 90 the difference was 4.73 (p=0.001). Of interest, a significant difference in cognitive test scores of 3.27 was observed two weeks after termination of AC-1202 treatment (p=0.034). This finding suggests that daily administration of AC-1202 may produce lasting effects in those patients with this combination of genotypic markers.
The combination of the E4(-); IDE_7(C/C)(-) genotype is prevalent in approximately 40% of the AD population, so the number of potential responder patients is substantial.
This pharmacogenomic finding provides both insight into the mechanism of ketone-based therapies for Alzheimer’s disease, and also allows for the identification of patients who may respond best to therapy, said Dr. Samuel Henderson, Research Director at Accera. This is the first scientific report of the role of IDE and its interaction with APOE on therapeutic efficacy in Alzheimer’s disease patients.
The study results will be presented on Wednesday, July 15 under the title, Evidence of an Interaction Between APOE and IDE in Ketone Body Therapies in Mild to Moderate Alzheimer’s Disease. The conference is being held in Vienna, Austria at the Messe Wien Exhibition and Congress Center.
About Alzheimer’s Disease
Alzheimer’s disease, the most common form of dementia, is a progressive and fatal disease for which there is no cure. In the United States, 5.2 million people are living with AD, and it has become the sixth leading cause of death. The disease attacks the brain’s cells, resulting in loss of memory, executive function and language skills.
AD significantly impacts millions of family members and other caregivers - mentally, physically and financially. The national Family Caregiver Alliance estimates that approximately 80 percent of caregivers provide unpaid assistance seven days a week. With the lack of innovative new medications for AD, both patients and caregivers are seeking alternatives to improve quality of life.
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