Ambit has developed a pipeline of small molecule targeted therapies using our expertise in kinase drug discovery and development. A panel of over 440 kinase assays developed by scientists at Ambit has allowed us to assemble a broad catalog of potential drug candidates annotated against more than 80% of known human kinases for potential interaction and activity. This approach has made it possible for Ambit to move projects from inception to the nomination of high quality candidates for development in 18 months or less, yielding the following pipeline of kinase inhibitor drugs.

The following table summarizes the status of our product pipeline:

Oncology—Striving for New Targeted Therapies

According to the World Health Organization, cancer is a leading cause of death worldwide. Chemotherapy, radiation and surgical resection of tumors are the most common approaches for treating cancer. Heightened vigilance, new diagnostic tests, combination therapies and improved treatment regimens have resulted in improvements in quality of life and overall survival for many cancer patients. Despite these advancements, the treatment of cancer remains inadequate. Currently available treatments typically address each cancer as a single disease based on the originating organ, as opposed to a collection of different disease subtypes driven by different genetic causes. We believe that there remain significant opportunities to improve patient outcomes by developing targeted therapies to treat the underlying cause of these different cancer subtypes.

For most types of cancer, targeted therapies, or the matching of a disease treatment to a specific tumor or disease profile, are anticipated to be more effective and have fewer side effects than traditional chemotherapy drugs. In contrast to the traditional therapies which indiscriminately kill healthy cells along with cancer cells, targeted therapies impact the underlying mechanisms of the disease. The use of targeted therapies, often in combination with other targeted agents and chemotherapy, will be an important component in the evolution of cancer treatment. Physicians administering targeted therapies rely on the use of a diagnostic test where possible to identify patients most likely to respond favorably. For example, Gleevec and Herceptin, both of which are prescribed for treatment of cancer in patients that have tumors driven by a particular genetic signature, are usually administered following a diagnostic test designed to select patients most likely to respond.

Kinases—A Rich Source for Targeted Therapies

Kinases are a large enzyme family with central roles in regulating and promoting cell growth and other cellular processes. In many diseases, kinases function abnormally, making them an important source of therapeutic targets. Over the last decade, the sequencing of the human genome and advances in molecular biology has made it possible to identify more than 500 members of this protein family. This facilitated the discovery of abnormal kinase activity in many cancers. Several of these kinase targets were validated by studying genetic differences between cancerous and normal tissues to identify tumor-specific mutations that may be implicated in the onset of disease. Kinases have also been implicated as targets in a number of other diseases including inflammatory and autoimmune diseases.

Since 2001, more than ten small molecule kinase inhibitors have been approved by the U.S. Food and Drug Administration (FDA) for the treatment of various cancer indications. Combined sales of these therapies in 2009 were approximately $8.4 billion. Gleevec alone generated sales of more than $3.9 billion and has revolutionized the outlook for patients with chronic myeloid leukemia (CML). Notably, Gleevec has significantly increased the five-year survival rate of patients with CML and has resulted in CML being treated as a chronic condition for most patients.