X-Chem achieves milestone in drug discovery collaboration with Alexion Pharmaceuticals

X-Chem met this milestone under a partnership established with Alexion in December 2014. Under the terms of the agreement, Alexion obtains exclusive worldwide rights to develop and commercialize novel molecules arising from the collaboration.

X-Chem would receive additional payments upon the achievement of specified research, development and regulatory milestones. In addition, X-Chem would receive royalty payments on the sale of products resulting from the collaboration.

The two companies are focused on the discovery and development of candidate molecules for the treatment of rare, life-threatening diseases, leveraging X-Chem’s DEX discovery engine, powered by a unique high-diversity and high-content DNA-encoded small molecule library, to identify novel small molecule protein modulators for targets for rare-disease applications identified by Alexion.

X-Chem CEO Rick Wagner said: "Reaching this milestone with Alexion is a great achievement for X-Chem and another demonstration of the wide range of targets and diseases that can be addressed with the DEX discovery engine.

"Alexion has been an innovative collaborator, and we look forward to similar success in the other programs."

About the DNA-Encoded X-Chem (DEX) Library and Platform

Due to the size and diversity of the DEX library, X-Chem can discover multiple series of novel, potent and selective lead compounds at an unprecedented rate of success against a wide range of targets, including some that previously failed using conventional screening methods.

A number of proprietary innovations in library design, screening methodology and bioinformatics underlie the exceptional performance of the DEX platform. In particular, X-Chem’s approach to library construction allows for additional chemical reactions to become useable in DNA-encoded library synthesis.

Together, these developments result in a much greater repertoire of diversity for small molecules, which cover a range of categories including fragment molecules, small molecular weight heterocyclic compounds, and macrocyclic structures. This diverse library, combined with a heightened ability to detect active molecules, has yielded a robust process that has been highly successful against targets categorized as difficult or intractable.
About DNA-Encoding

The X-Chem drug discovery engine is based on a library generated by iterative combinatorial synthesis of small molecules tethered to DNA tags that record the synthetic history of the small molecule. Every small molecule in the library has a unique DNA barcode attached it. The library is screened as a mixture using affinity-based binding to a target of interest. Certain rare molecules in the library that bind to the target can be "fished out," while the rest of the molecules wash away.

DNA sequencing methods are then used to detect molecules that are enriched when bound to the target. The diverse nature of the library produces multiple families or clusters of related molecules that bind to the target, forming a basis for emergent structure-activity relationships.

Structure-activity relationships are typically used by medicinal chemists to guide iterative chemical maturation of a molecule into a drug. Based on the synthetic history encoded in the DNA sequence information, molecules are then made without the DNA tag attached, and tested for activity in conventional assays.