X-Chem, Vertex to Jointly Develop Small Molecules for CF, Other Severe Genetic Diseases

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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Vertex X-Chem

X-Chem Pharmaceuticals and Vertex Pharmaceuticals will jointly develop small molecules targeting cystic fibrosis (CF) and other severe genetic diseases.

Under the collaboration announced May 16, X-Chem will apply its proprietary DEX libraries — which holds more than 120 billion small compounds — to discover new drug candidates against targets involved in CF and other diseases. Vertex may license lead-like hits discovered under the partnership, and will develop and commercialize the resulting drug programs.

X-Chem will receive an undisclosed amount upfront, as well as potential research, development and regulatory milestones and licensing fees. In addition, the company — based in Waltham, Massachusetts — may receive royalties on revenues from drugs discovered under the collaboration.

“We look forward to working closely with Vertex, a recognized innovator in discovery of first-in-class treatments for severe, life-threatening diseases,” Rick Wagner, CEO of X-Chem, said in a press release. “The combination of X-Chem’s DEX platform and Vertex’s expertise has great potential to generate promising leads that could lead to medicines for the treatment of serious, specialty diseases with high unmet needs.”

X-Chem created its DEX library through the use of chemical synthetic methods based on small molecules tied to DNA tags. Every small molecule in the library has a unique DNA barcode attached to it. This DNA-encoded library includes rare molecules that can be used to target the genetic alterations seen in CF, an inherited disorder that severely damages the lungs, digestive system and other organs.

Thanks to its diversity and size, the DEX library can lead to the discovery of several novel and potent therapeutic compounds against a broad range of targets involved in several medical conditions.

“As Vertex continues to grow and diversify its R&D pipeline, we look to complement our productive internal efforts with other organizations who share our passion to treat diseases for which there is no existing treatment,” said Mark Bunnage, Vertex’s senior vice-president and Boston research site head. “With a cutting-edge small molecule lead discovery platform, X-Chem is ideally suited to help Vertex tackle challenging rare-disease targets.”

Vertex currently has on its drug portfolio several therapies for CF, including the approved therapies Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor). It is also testing several treatment combinations.