Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a clinical-stage pharmaceutical company focused on developing small molecule inhibitors of factor D in the complement alternative pathway (AP), today announced that the Company had begun dosing healthy volunteer subjects in a first-in-human phase 1 trial of ACH-5548, an oral small-molecule complement factor D inhibitor. ACH-5548 is the third clinical compound discovered and developed by Achillion from its complement factor D platform.
ACH-5548, like the second compound from Achillions factor D portfolio, ACH-5228, is structurally designed to achieve significant improvements in potency and pharmacokinetic properties over first-generation ACH-4471. In pre-clinical studies, both ACH-5228 and ACH-5548 demonstrate multiple-fold increased potency with pharmacokinetic profiles that offer potential for less frequent dosing than ACH-4471.
The advancement of ACH-5548 into the clinic is an important milestone for Achillion as we continue to broaden our clinical stage factor D portfolio. Clinical data generated to date demonstrate that factor D inhibition represents a novel, first-in-class approach to developing potential treatments for patients suffering from alternative pathway-mediated diseases, commented Joe Truitt, President and Chief Executive Officer of Achillion. As we continue our transition to a late-stage clinical and commercial company in our global phase 2 clinical programs with ACH-4471 in both PNH and C3G, we also continue to advance next generation compounds in an effort to bring new treatments to patients. We believe this expanded portfolio of three clinical stage factor D inhibitors will provide important strategic optionality for Achillion.
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