Novel modulators of Abeta42 synthesis - The route towards primary prevention in Alzheimer´s Disease

Important results from the project

Alzheimer’s disease is a slowly progressive neurodegenerative disease with a long pre-symptomatic phase, during which Abeta pathology develops/spreads in the brain. Recent studies strongly suggest that anti-amyloidogenic treatments should be initiated early in the disease, i.e. as preventive treatments, in order to be most effective. The objective of this project is to develop a gamma-secretase modulator as a new disease modifying therapy. This therapeutic mechanism directly targets Abeta production, making it well-suited as a preventive treatment of Alzheimer’s disease.

Expected long term effects

We have identified a promising gamma-secretase modulator that we are currently pursuing in preclinical toxicology studies. The near-future objective is to test the compound in phase 1 clinical studies and then to further develop the compound in clinical phase 2 proof of concept studies. Gamma-secretase modulators are a class of anti-amyloidogenic compounds with a great potential for preventive treatment of Alzheimer’s disease. Our aim is to develop a first-in-class treatment for Alzheimer’s disease that will provide the solution to a huge unmet medical need.

Approach and implementation

We have, based on a project that stems from AstraZeneca, developed a potent gamma-secretase modulator. The identified compound has been progressed into pre-clinical development stage through the development of novel synthetic methods, large scale synthesis as well as being further evaluated in ongoing preclinical toxicological studies. The successful progress of the project towards clinical development has been possible due to the multi-disciplinary work of the project team.