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VITAE PHARMACEUTICALS, INC filed this Form S-1 on 08/12/2014
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Immuno-Oncology Program

        We have initiated a discovery program focused on developing small molecule immuno-modulators for treating cancer. The goal of cancer immunotherapy is to harness the human immune system and direct it against the cancer to achieve durable responses or complete eradication of disease. The genetic and cellular changes that are present in most cancers provide the immune system with new antigens to generate tumor-directed T cells that can recognize and kill these cancer cells. However, the presence of immune suppressive factors from the cancer cells or in response to the cancer cells in the tumor micro-environment blunts an effective immune response, allowing tumors to escape immune-mediated clearance. The key to success in cancer immunotherapy is the ability to overcome tumor-induced mechanisms that antagonize the function of the immune system and limit its capacity to clear the tumors.

        Cancer immunotherapy has been a focus of the pharmaceutical industry and academia over the last several years due to the publicly disclosed clinical successes that immune activation has had in treating many tumor types. There is an emerging class of cell checkpoint blockers to treat cancer by targeting immune checkpoints, which are inhibitory signaling pathways that switch off T cells. These inhibitory pathways are upregulated in the tumor microenvironment, allowing cancers to actively suppress tumor infiltrating T cells and escape targeting. The first clinical success with checkpoint blockade was seen with Yervoy, an antibody shown to bind to and block the signaling of CTLA-4, an inhibitory protein on the T cell surface. Even more promising clinical results have been seen with antibodies targeting other checkpoint inhibitor pathway components (e.g. PD-1, Tim-3, LAG-3). In addition to checkpoint blockers, a number of complementary approaches are being pursued clinically, including novel vaccination strategies, adoptive T cell therapies and direct activation of T cell co-stimulatory pathways.

        With the initial success of anti-CTLA-4 directed therapies, the list of potential "checkpoint" modulators has continued to expand. A number of small molecular weight metabolites have been identified by others as potential immuno-modulators. These metabolites, which are generated by cells in the tumor microenvironment, directly suppress T cell function or activate immune suppressive mechanisms. The ability to inhibit the enzymes that generate these metabolites with small molecule drugs represents a new approach to tumor immunotherapy.

        As clinical experience grows with immuno-therapeutics, both preclinical and clinical studies in the published scientific literature suggest that combination regimens are likely to be the dominant route for development of agents in this area. This applies not only to combinations with other newer agents, but also with conventional therapies such as radiotherapy, surgery and chemotherapy, and multiple groups have demonstrated the potential for additivity or synergy of these combinations to promote broader and more durable responses. Regimens targeting multiple, discrete pathways to activate tumor immunity have the potential to improve patient outcomes dramatically.

        Using our Contour platform, we discovered certain potential compounds that bind to and inhibit our target protein which are candidates to develop within our immuno-oncology program. We plan to conduct preclinical animal studies to optimize these compounds and confirm that they are active.

Contour—Our Structure-Based Drug Design Platform

    Traditional Discovery Approach Compared to Contour

        Small molecule drugs continue to represent the majority of drug approvals in the United States. In 2013, the FDA approved 25 small molecule drugs out of 27 new drug approvals. Small molecule drugs interact with a target molecule, typically a protein, either to induce or to inhibit that molecule's function within the human body. Traditionally, small molecule drugs have been discovered through the screening of thousands to millions of compounds from existing chemical libraries against a predictive biological assay for a disease target. Under this method of small molecule drug discovery, molecules or potential drugs are