Generation 2+ antisense drug
IONIS-MAPTRx, also known as BIIB080, is an antisense oligonucleotide (ASO) drug designed to selectively reduce production of the tau protein in the brain. Microtubule-associated protein tau (MAPT) or tau, is thought to be a contributor or cause of certain neurodegenerative diseases, known as tauopathies, that are characterized by the deposition of abnormal tau protein in neurons and glia in the brain. These disorders include Alzheimer’s disease (AD), Progressive Supranuclear Palsy (PSP), and some forms of frontotemporal degeneration (FTD).
About Alzheimer’s Disease (AD)
It is estimated that in the U.S., 10% of people age 65 and older is living with dementia. Alzheimer’s disease (AD) is the most common form of dementia, accounting for an estimated 70% of cases. AD is a neurodegenerative disorder characterized by cognitive decline and behavioral disturbances that eventually result in a person’s inability to perform daily activities. AD relentlessly progresses to death over 5-20 years. Unlike amyloid deposits that appear diffusely throughout the brain for up to 20 years before the onset of AD, tau deposits are temporally and spatially closely-related to where brain atrophy occurs and cognitive deficits originate.
About Frontotemporal Degeneration
Frontotemporal degeneration (FTD) is a rare form of dementia resulting from neuronal damage in parts of the brain called the frontal and temporal lobes. As neurons die in these regions, these lobes atrophy. FTD typically occurs between the ages of 45 and 64 years old, with patients surviving approximately 9 years from diagnosis. In the early stages of the disease, patients experience substantial personality and social behavior changes, including apathy, loss of empathy, emotional bluntness, impulsiveness, poor judgment, communication impairment, among others. Death is typically the result of disease progression leading to pneumonia, failure to thrive, or cardiopulmonary failure.