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Jul 18, 2013

Ultra-Rare Disorders Targeted in Ensemble, Alexion Collab

  • Ensemble Therapeutics is now in a drug discovery collaboration with a subsidiary of Alexion Pharmaceuticals, a biopharmaceutical company focused on developing and commercializing therapies for patients with life-threatening, ultra-rare disorders. The goal is to deploy Ensemble’s drug discovery platforms for several undisclosed drug targets identified by Alexion to create small molecule therapeutic candidates.

    Ensemble will screen its Ensemblin™ collection of more than 10 million macrocycles against several disease drug targets specified by Alexion, with the aim of using Ensemble’s medicinal chemistry capabilities to discover new small molecule clinical candidates. Ensemblins are a class of small molecule therapeutics that Ensemble says has the potential to modulate targets typically addressed by biologics. Ensemble is also in a research collaboration with Boehringer Ingelheim—one that was said to be worth up to $186 million when announced in November 2012—involving Ensemblins.

    Alexion will have the exclusive worldwide rights to develop and commercialize candidates arising from the collaboration. Ensemble will receive an upfront payment and research support, and could potentially earn additional payments upon the achievement of certain development and commercial milestones. Other terms were not disclosed.

    "This new collaboration with a leading biopharmaceutical innovator focused on rare diseases speaks to the breadth of utility of Ensemble’s discovery platforms to address clinically important yet challenging drug targets," said Ensemble CEO Michael D. Taylor, Ph.D.

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