APG has identified a new way of reducing aggregation. The drug candidate is a small molecule, which may be applied orally. Clinically modifying the function of the identified drug target has the potential to modulate HD pathology, improve symptoms, and slow down cognitive decline, behavioral changes and movement abnormalities. We are currently testing analogs of our hits with a binding assay to identify small molecules with improved affinity to the drug target. We have so far a lead candidate with excellent in vitro blood brain barrier penetrability and ADMET properties.
Current status of the drug discovery program
We are optimizing our lead compound.
Mutated huntingtin form intraneuronal inclusions are located in the striatum, cerebral cortex, cerebellum, and the spinal cord. The inclusion bodies are a pathological hallmark of HD. Inclusion bodies are found in neurons before the manifestation of behavioral symptoms and significant neuronal death.
There are no disease modifying therapies for HD available! In Europe and North America there are 90,000 patients who suffer from HD. It is estimated that there are 600,000 gene carriers who will inescapably develop symptoms over time.