Closely-held US biotech firm Alzheon has announced the publication of data that shows for the first time a ‘gene-dose’ effect for a drug targeting Alzheimer’s disease to treat a subpopulation of up to 65% of Alzheimer’s patients with a genetic risk factor.

Based on these results, Alzheon is advancing its drug candidate tramiprosate into Phase III clinical studies to seek approval of a new drug for Alzheimer’s disease.

Back in 2007, Canadian drugmaker Neurochem abandoned development of its Alzheimer’s disease drug candidate Alzhemed (tramiprosate), on the basis that data from the program was unlikely to be sufficient for regulatory approval. Alzheon was launched in October 2013, when is gained rights to ALZ-801, an oral prodrug of tramiprosate.

Alzheon has lead authorship on a paper co-authored by leading Alzheimer’s experts that was just published in The Journal of the Prevention of Alzheimer’s Disease (JPAD), detailing the first report from a large Phase III clinical study analysis to associate the efficacy of an amyloid-targeted drug with the APOE4 genetic status in Alzheimer’s patients.

APOE4 is a major genetic risk factor is present in up to 65% of Alzheimer’s disease (AD) patients. The presence of APOE4 alleles is associated with earlier onset of AD, more severe symptoms and faster progression.

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