Chemotargets Announces First AI-Designed Drug for Huntington’s Disease to Enter Clinical Trials

Chemotargets Announces First AI-Designed Drug for Huntington’s Disease to Enter Clinical Trials - Main Image

In less than 12 months, the collaboration between Chemotargets and Galyan Bio yields a scaffold for first-in-class clinical candidates in difficult area of neurodegenerative disease. Chemotargets employed its proprietary AI-driven de novo drug design methods in collaboration with Gaylan Bio to rapidly design novel molecules which would bind to the previously ’undruggable’ Huntington’s Disease target.

Barcelona, 22 April 2021 - Huntington’s Disease (HD) is a neurodegenerative disorder that affects thousands of patients worldwide. There is currently no cure or disease modifying treatment for HD. This is why US-based company Galyan Bio and Barcelona-based Chemotargets joined efforts to design a first in class drug for HD. The result has been the generation of a first-in-class drug candidate for HD. Galyan Bio plans to start clinical trials in HD in 2022. Galyan Bio’s new treatment is intended to slow down the disease in symptomatic patients and delay manifestation of HD in symptom-free gene carriers.

The development of new molecules through medicinal chemistry has always been a laborious and costly process. We used Chemotargets drug discovery platforms to speed up the process considerably. Based on Chemotargets’ AI-driven de novo designs, we selected and tested 49 compounds. Our expectations were exceeded as we identified 6 compounds which could actually bind to our drug target. The optimization of one of those hits led to a preclinical drug candidate for HD” said Dr. Marius Galyan, CEO of Galyan Bio.

Chemotargets’ proprietary ProSurfScan AI-driven de novo drug design platform uses novel methods of agnostically scanning the target protein to perform a virtual fragment-based screening of all possible drug binding areas. Fragments are then automatically linked to design effective binding molecules. The AI-driven methods employed by Chemotargets are particularly suited to novel, difficult, previously unaddressable targets as well as drug targets for which high isoform selectivity is required. ProSurfScan is part of a complete suite of AI-driven drug discovery tools which are in routine use within Chemotargets that include CLARITY®, the industry-leading software platform for anticipating the safety of new drug molecules. CLARITY® is currently in use in many of the top pharmaceutical companies worldwide and at the US Food and Drug Administration.

We are absolutely delighted to have effectively contributed to delivering a first-in-class drug candidate for HD. Having a tangible proof-of-principle that our de novo designs lead to progressible bioactive small molecules in a prospective drug discovery project means a world to us. This means that our in-silico platform is ready to address other novel, difficult, protein targets for which no therapeutic modalities exist.” said Dr. Jordi Mestres, CSO of Chemotargets.

First and foremost, we are grateful for the opportunity to contribute to a possible therapeutic alternative for Huntington’s Disease patients. I am, quite frankly, not surprised by the experimental activity results of the molecules arising from the de novo design. ProSurfScan has repeatedly demonstrated its ability to design novel, selective molecules which bind to novel and difficult targets in our internal programs and will no doubt continue to do so. Although artificial intelligence is necessary in nearly everything we do at Chemotargets, we are a company focused on helping patients. By using AI to aid decisionmaking in the design and development of novel therapeutics, our mission will always be to facilitate the development of safer, more effective therapeutics in less time and at a lower cost that was possible in the past. We are pleased that, for Huntington’s Disease patients, we have been able to demonstrate that potential.” said Dr. Scott Boyer, CEO of Chemotargets.

Galyan Bio, Inc. is a startup company located in Berkeley, California (USA). Galyan is developing first-in-class orally available small molecules for neurodegenerative diseases, cancer and aging. It was founded in 2019 by Dr. Marius Galyan.

Chemotargets is a biotech company focused on the development of state-of-the-art predictive analytics platforms for drug discovery and the design of novel bioactive small molecules for challenging difficult-to-address targets. It was founded in March 2006 by Dr. Jordi Mestres as a spin-off from the IMIM Hospital del Mar Medical Research Institute, a leading health research institute based in Barcelona, Catalonia (Spain). Chemotargets has its headquarters at the Parc Científic de Barcelona.

Chemotargets CLARITY® (Seal of Excellence project from the European Commission) uses artificial intelligence on carefully-curated data to connect small molecules, protein targets, safety events, and therapeutic indications with the help of state-of-the-art analytics tools. The platform can be used to identify the probable primary and secondary targets of molecules and their predicted metabolites by rapidly profiling through consensus ligandbased models of 4,799 proteins built from affinity data for almost 3 million molecules. In addition, machine learning models of 1,278 safety endpoints were constructed from data carefully extracted from over 39 million spontaneous reports, including hundreds of safetyrelated mechanisms annotated with preclinical toxicity and clinical safety issues. Beyond predictive pharmacology and safety, the CLARITY Suite of products can be applied to drug repurposing, deconvolution of phenotypic screens, translational safety and pharmacovigilance.

ProSurfScan is a novel AI-based method developed at Chemotargets to scan protein surfaces in search for amino acid environments likely to interact with chemical fragments. It uses convolutional neural networks derived from all known protein structures to perform a virtual fragment screening of a complete protein surface. The approach is especially suited for addressing novel, difficult to address targets, including protein-protein interactions, and a variety of modes of action, such as allosteric interactions.


Media Contacts:

Gaylan Bio: Dr. Marius Galyan, CEO (

Chemotargets: Dr. Scott Boyer, CEO (