information: Initiation of development program
Announcement: initiation of development program
Company: AC Immune (Switzerland)
Product: Tau Morphomers
Disease: Alzheimer's disease
area: Neurodegenerative diseases
- • On April 5, 2018, AC Immune demonstrated target-specific reduction of pathological Tau and cognitive and functional improvement in proof-of-concept studies in Alzheimer's disease. In addition to the Tau inhibition, a significant parallel reduction of microglia activation and neuroinflammation has been observed; another key factor in Alzheimer's and other neurodegenerative diseases.
- Tau Morphomers are the first candidates derived from AC Immune's proprietary Morphomer™ platform generating therapeutic CNS small molecules with high selectivity for misfolded proteins in multiple proteinopathies.
The specifically designed Tau Morphomers have a unique mode of action, inhibit intracellular Tau pathology the source of Tau spreading and reduce neuroinflammation.
- Several chemical series of small molecules (Morphomers™) have been identified which selectively and potently reduce toxic intracellular misfolded and aggregated Tau. Targeting intracellular misfolded and aggregated Tau is widely recognized as an important and attractive approach for interfering with the spread of Tau pathology throughout the brain. The activity of Tau may act as a seed that induces native endogenous Tau forms to misfold and aggregate into toxic species. In proof-of-concept Tauopathy models, reduction of Tau pathology was also accompanied by a reduction of associated neuroinflammatory markers - another key pathologic feature of Alzheimer's disease (AD).
- Lead compounds have been identified which display excellent ADME (absorption, distribution, metabolism and elimination or excretion) and pharmacokinetics properties suitable for targeting the central nervous system. Two candidates in clinical studies are currently undergoing further preclinical safety assessment, with the goal to initiate a clinical Phase°1 study by end 2018.