Asceneuron strives to address high unmet medical needs in neurodegenerative diseases such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases. Our pipeline is composed of innovative small-molecule therapeutics aiming for disease modulation of tauopathies and cognitive impairment. 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)
Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as neurofibrillary tangles and other types of aggregate in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major driver of neurodegeneration, and mutations in the tau gene are causative of certain tauopathies such as Progressive Supranuclear Palsy (PSP) and Frontotemporal dementia (FTD).
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