Huntington's disease Drug Discovery Program


APG has identified a new way of preventing aggregation, oligomer formation and seeding of mutated huntingtin. 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 early leads with excellent in vitro blood brain barrier penetrability and high solubility.



Transcription Factor Inhibitor

We have identified in silico hits which might inhibit a new drug target that drives cancer proliferation, invasiveness and dissemination in various cancers. One of the hits was verified in cell experiments. APG-C-20 has good solubility, metabolic stability and no detectable toxicity in three different cell lines.


We are currently setting up analog screening assays and we are planning to test the compound in animal studies beginning end of 2018. As the first indication for market approval, we plan to do a study with melanoma patients. A second and very attractive indication for the new transcription factor inhibitor is glioblastoma multiforme.


Research Cooperations



Avergen is pursuing collaboration with university researchers and other pharmaceutical companies. Working with external partners allows Avergen to access different pools of knowledge and save R&D costs. Thus guaranteeing successful innovation. Partnerships will be maintained throughout the whole life cycle of our development programme and products.


Avergen is cooperating with the University of Erlangen to conduct animal studies in Huntington's disease.


In 2016 we have started a research collaboration with the University of California, Irvine.


Avergen is conducting drug screening programs in collaboration with research laboratories at the IZB in Martinsried, Germany and at the Stevenage Bioscience Catalyst, United Kindom.