Profile: Achillion Pharmaceuticals Inc (ACHN.O)

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Achillion Pharmaceuticals, Inc., incorporated on August 17, 1998, is a biopharmaceutical company focused on the discovery, development and commercialization of treatments for infectious diseases. The Company is engaged in the development of antivirals for the treatment of chronic hepatitis C infection (HCV), and the development of antibacterials for the treatment of resistant bacterial infections. The Company focuses on the development of four drug candidates for the treatment of HCV: ACH-1625, a NS3 protease inhibitor for the treatment of HCV, which is being tested in a phase IIa clinical trial, ACH-2684, a NS3 protease inhibitor for the treatment of HCV, which is being tested in a phase I clinical trial; ACH-2928, a NS5A inhibitor for the treatment of HCV, which is being tested in a phase I clinical trial, and ACH-3102, a NS5A inhibitor for the treatment of HCV, which is being prepared for investigational new drug (IND), filing and initiation of a phase I clinical trial.

ACH-1625, a NS3 Protease Inhibitor for Chronic HCV Infection

The Company is evaluating ACH-1625, a protease inhibitor for the treatment of chronic hepatitis C, in a phase IIa clinical trial to assess the compound’s safety, tolerability, pharmacokinetic properties and efficacy in HCV-infected subjects. ACH-1625 has demonstrated liver partitioning and a safety profile in preclinical studies. ACH-1625 was demonstrated to achieve a complete early virologic response (cEVR), in 100% of patients receiving ACH-1625 at doses of 200 milligram, 400 milligram and 800 milligram once-daily in combination with pegylated interferon alpha and ribavirin (P/R). In December 2011, ACH-1625 was granted Fast Track status by the United States Food and Drug Administration (FDA).

ACH-2684, a NS3 Protease Inhibitor for Chronic HCV Infection

ACH-2684 demonstrates a range of natural variants of HCV, and prevention and treatment of emerging resistant variants. This compound also retains potent in vitro activity against all known HCV genotypes 1-6. In preclinical studies, ACH-2684 was effective in combination with other HCV inhibitors, and in vitro is synergistic with NS5B nucleoside polymerase inhibitors. It is in phase I clinical studies, ACH-2684 reduced viral load by a maximal 4.63 log10 in genotype 1 HCV patients and by a maximal 2.03 log10 in genotype three HCV patients.

ACH-2928, a NS5A Inhibitor for Chronic HCV Infection

ACH-2928 is highly active and potent against HCV genotypes 1a and 1b, as well as across other genotypes. NS5A inhibitors demonstrate in clinical trials to be highly effective in combination with NS3 protease inhibitors, and in vitro studies to be highly effective in combination with NS5B polymerase inhibitors, interferon and ribavirin. In phase I clinical studies, ACH-2928 was demonstrated to reduce viral load by a maximal 4.86 log10 and was safe and well-tolerated. This phase I clinical trial for ACH-2928 is on-going.

ACH-3102, a NS5A Inhibitor for Chronic HCV Infection

In preclinical studies, ACH-3102 has demonstrated potent pan-genotypic activity, meaning activity against HCV subtypes referred to as genotypes one through six, including activity against both the 1a genotype and known mutant variants of genotype 1 HCV. It has IND-enabling preclinical testing for ACH-3102 and focuses to prepare to file an IND with the FDA and to initiate phase I human clinical studies.

Other drug candidates

The Company has established a pipeline of other product candidates for which the Company is seeking appropriate collaborative partners. The Company does not have resources for ACH-702 and ACH-2881 for drug resistant bacterial infections and elvucitabine for HIV infection.

ACH-702 is a preclinical candidate with potency against a range of spectrum of bacterial pathogens, including methicillin-resistant staphylococcus aureus (MRSA). In the Company’s research on compounds similar to ACH-702 for systemic use against MRSA, the Company discovered ACH-2881, a hydroxythienoquinolone (HTQ). The Company’s discovery tools are, including Achillion Automated Chemistry Platform (AACP), Achillion Cheminformatics Engine (ACE), Compound Acquisition and Repository Tracking (CART), CHEM-ACH, Competitive Intelligence & Data Mining (CIDM) and Preclinical Study Tracking System (PSTS). AACP is a software program that facilitates medium and high throughput synthesis of compounds. AACP allows the Company to synthesize thousands of small molecules in support of its drug discovery programs. ACE is a software interface, which provides access to several vendors’ compound libraries, analyzes the physicochemical properties of compounds and assists in designing new compound libraries. CART is a software tool that streamlines the Company’s scientists’ ability to select and acquire compounds for lead identification. CART is integrated with computational chemistry tools and a virtual database of greater than two million small molecules. CHEM-ACH is data mining software that allows compounds synthesized at Achillion to be cross-referenced against biological activities associated with them. CIDM is a Web application. It analyzes publicly available information to display competitive information, including clinical and preclinical development activities, intellectual property and scientific literature. PSTS is a Web interface, which is used for accessing the details of its preclinical studies. It allows scientists to enter, modify, and query preclinical study documents.

The Company competes with Roche, Merck, Vertex, Abbott, Astra-Zeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Enanta, Gilead, GlaxoSmithKline, Human Genome Sciences, Idenix, Johnson & Johnson, Presidio, Medivir, Novartis, Pfizer, Valeant.

Company Address

Achillion Pharmaceuticals Inc

300 George Street
NEW HAVEN   CT   06511
P: +1203.6247000
F: +1203.6247003

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