OSI Pharmaceuticals, LLC, a member of the Astellas US group of companies (Astellas), is an oncology drug discovery and translational research organization primarily focused on the discovery of molecular targeted therapies (MTTs). Following its 2010 acquisition by Astellas, OSI has become Astellas center of excellence for oncology small molecule discovery research.

OSI and its partners received FDA approval for the use of Tarceva® to treat non-small cell lung cancer first in 2004 as a second/third line therapy, and again in 2010 as maintenance therapy. In 2005 we received approval for the use of Tarceva to treat pancreatic cancer in combination with gemcitabine.

Throughout the development and multiple launches of Tarceva, OSI's research teams developed a deep understanding of the mechanisms driving both patient benefit and resistance to epidermal growth factor receptor targeted therapy, and OSI has worked hard to put this knowledge into practice in its pipeline programs. OSI's collective goal is to serve even better the patients treated with OSI's drugs through precision medicine and better biomarkers and to advance the OSI pipeline of targeted agents beyond what has been achieved with Tarceva. OSI has several prospective new oncology drugs in its research pipeline.

Targeting the Needs of Patients

Cancer comprises groups of diseases characterized by the uncontrolled growth and spread of abnormal cells. This definition is a very simple one for such complex and challenging diseases. Cancer is ranked as the second largest cause of death in the United States. Indeed, the American Cancer Society has estimated that more than 570,000 Americans would die from cancer in 2011, with about 1.6 million new cases diagnosed (Cancer Facts & Figures, 2011).

Although these statistics highlight the challenges yet to be overcome in the fight against cancer, they do not accurately reflect the advances in treatments that offer patients a better quality of life while increasing survival rates. OSI strives to be at the forefront of the war against cancer by arming patients with an arsenal of weapons and tools to help them manage their disease and live productive lives.

Together with discovery and development colleagues across the Astellas organization, OSI seeks to integrate both oncology discovery and clinical sciences through focused clinical trial design. OSI's success is driven by the discovery and implementation of proprietary patient selection markers that predict response to targeted therapeutics.

OSI seeks to link patient outcome to response biomarkers within the patient populations we intend to treat. Our goal is to develop drugs that achieve superb efficacy, measured through disease remission and cure, for our patients through a careful and detailed understanding of each individual's disease. Our collective goal is a paradigm shift in how cancer patients are treated.

Shifting the Paradigm

OSI's oncology research strategy is based on achieving the Astellas global expectation of the future of cancer therapy and, hence, of realizing the goal of achieving truly personalized therapy in oncology: This is the Astellas vision of precision medicine. Over the coming years, OSI anticipates the continued development and use of oral targeted therapies both as single agents and in combination with other anti-cancer drugs. To achieve this vision of precision oncology medicine, cancer research at OSI combines the identification of pharmacodynamic, surrogate, and diagnostic markers within the organization's clinical development programs to monitor drug efficacy and to provide the opportunity to select responsive patients.

OSI's research organization is focused on the discovery of novel small molecule compounds targeted at the underlying molecular mechanisms of the pathogenesis of cancer. The OSI strategy is to develop mechanism-based approaches to drug discovery directed at oncogenic drivers and the processes underlying Epithelial Mesenchymal Transition (EMT) in tumor growth and survival in the development and progression of cancer. OSI seeks to develop novel agents that result in disease remission and cure through targeting heterogeneous cell types that comprise solid tumors. Through understanding the biology and pharmacology of cancer EMT and compensatory pathway activation, OSI's discovery efforts target key growth and survival pathways of both epithelial and mesenchymal tumor cells that characterize much of the complexity of human disease.

Science and Research

OSI Pharmaceuticals has developed an extensive, fully integrated drug discovery organization, generating a pipeline of high-quality drug candidates to move into clinical development. This research operation is built upon the organization's strengths, including high-throughput screening, chemical libraries, medicinal and combinatorial chemistry and automated drug profiling technology platforms. The research at OSI's Farmingdale site uses new technologies in drug evaluation and biomarker discovery through a proprietary tumor model platform that we use to (1) better understand the mechanism of action of OSI's compounds, (2) define new cancer indications for OSI's drugs, and (3) discover predictive biomarkers for use in OSI's clinical trials.

Oncology Pipeline

OSI-906 is a potential first-in-class selective small-molecule, dual kinase inhibitor of both insulin-like growth factor-1 receptor (IGF-1R) and insulin receptor (IR). OSI-906 is currently in a Phase III clinical trial in adrenocortical carcinoma (ACC) and in Phase II clinical trials in lung and ovarian cancers.

OSI-027 is a next-generation mammalian target of rapamycin (mTOR) kinase inhibitor that inhibits the kinase activity associated with both the TORC1 and TORC2 complexes of mTOR. OSI-027 is currently completing its Phase I clinical trial.

For more information, visit the Investor Relations Library on the Astellas Pharma Inc. Web site.