Our pipeline is composed of innovative small-molecule therapeutics aiming to prevent disease progression in proteinopathies and reverse cognitive impairment in dementia.
Due to their nature, our molecules readily enter the brain and are orally bioavailable, meaning that they are suitable for dosage as pills.
For each program, we implement a clinical biomarker. The clinical biomarker is expected to demonstrate that the drug reaches the intended target in humans and will support dose selection for the seminal proof-of-concept trials in patients.
Proteinopathies (Tau / α-Synuclein)
Proteinopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. This group of neurodegenerative disorders is characterized by the intracellular formation of insoluble and toxic protein aggregates in the brain that are closely linked to disease progression.
In Alzheimer’s disease and in rare tauopathies, such as progressive supranuclear palsy (PSP) and frontotemporal dementia (FTD), aggregation of the microtubule-associated tau protein leads to the formation of toxic neurofibrillary tangles (NFT). NFT formation is generally believed to cause neurodegeneration in these diseases.
In Parkinson’s disease (PD) and other α-synucleinopathies, such as dementia with Lewy bodies (DLB), intracellular Lewy bodies containing aggregates of α-synuclein constitute the pathological hallmark.
Cognitive improvement in dementia
Dementia is the umbrella term for the memory impairment and cognitive decline which is major clinical symptom in a large number of neurological diseases such as Alzheimer’s disease (AD), Parkinson’s disease (PD) and Lewy body dementia (LBD). Current pro-cognitive treatment options provide limited benefits, supporting the urgent need for more effective and better tolerated medicines that offer symptomatic relief as well.
Asceneuron OGA inhibitors offer multimodal mechanism of action with potential for multiple approaches to clinical development in Alzheimer’s and Parkinson’s disease