Achillion Pharmaceuticals, Inc., a biopharmaceutical company, engages in discovering and developing small molecule drug therapies for infectious diseases and immune system disorders. The company focuses on its complement inhibitor platform, directed at advancing small molecule compounds that have the potential to be used in the treatment of immune-related diseases associated with the complement system. It is advancing novel small molecules from this platform, which would initially target complement factor D, an essential protein within the amplification loop of the alternative pathway. The company has initiated a first-in-human phase I clinical trial for its first complement factor D inhibitor, ACH-4471, to assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD). It plans to advance this compound to potentially treat patients with PNH and possibly, one additional systemic ultra-rare disease. Programs ACH-4471: ACH-4471 is a potent and specific inhibitor of factor D, which has demonstrated complete suppression of the complement alternative pathway with a single oral dose in non-human primates. Further, the compound has demonstrated dose-proportional in vitro suppression of red blood cell destruction, or hemolysis, in plasma samples from PNH patients and similar suppression of cell killing in serum from patients with aHUS. Additional Factor D Inhibitors: The company’s research team has synthesized approximately 1,200 factor D inhibitor compounds from which it plans to select one or more development candidates to advance into the clinic. The company’s candidate library contains compounds with various pharmacophysical properties for various routes of administration. Collaboration with Janssen Pharmaceuticals, Inc. In May 2015, the company entered into a collaboration and license agreement with Janssen Pharmaceuticals, Inc. (Janssen) and its affiliate, Johnson & Johnson Innovation-JJDC, Inc., which it refers to as the Janssen Agreement. Under the Janssen Agreement, the company granted Janssen worldwide rights to develop and commercialize products that contain one or more of its drug candidates for the treatment of hepatitis C virus (HCV), namely odalasvir, a second-generation NS5A inhibitor; ACH-3422, a NS5B HCV polymerase inhibitor; and sovaprevir, a NS3/4A HCV protease inhibitor. The company established its HCV drug candidate pipeline through its internal discovery capabilities. The compounds from its discovery and development efforts are licensed for further development to Janssen. Odalasvir, a NS5A Inhibitor: The company completed three Phase IIa clinical trials with odalasvir, including the -007 trial with sovaprevir, the -005 study, which examined the use of odalasvir with ribavirin alone; and the Proxy Doublet study, which examined the use of odalasvir in combination with sofosbuvir, a nucleotide NS5B polymerase inhibitor marketed by Gilead Sciences, Inc. under the brand name Sovaldi. ACH-3422, a NS5B Nucleotide Polymerase Inhibitor: ACH-3422 has demonstrated potency and tolerated in a Phase Ib proof of concept study. Sovaprevir, a NS3/4A Protease Inhibitor: The company has completed a Phase II clinical trial that evaluated 12 weeks of treatment consisting of sovaprevir and its NS5A inhibitor, odalasvir, with ribavirin for the treatment of genotype 1 HCV (the -007 trial). In October 2015, the company announced that Janssen had initiated Phase IIa clinical testing of a triple combination regimen consisting of Olysio, a protease inhibitor marketed by Janssen; AL-335, a nucleotide polymerase inhibitor in clinical development by Janssen; and odalasvir. The trial would evaluate the safety, pharmacokinetics and efficacy of the combination treatment in patients with genotype 1 chronic HCV. Strategy The company’s near-term strategy includes advancing its complement Factor D portfolio comprising Advance ACH-4471 in clinical development for rare diseases, advancing back-up series of compounds for rare diseases, and discovering and developing additional complement factor D compounds for other complement-mediated disease indications; and collaborating with Janssen in H
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New Haven, CT 06511
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