2014 MEDICINES IN DEVELOPMENT REPORT Cancer PRESENTED BY AMERICA’S BIOPHARMACEUTICAL RESEARCH COMPANIES Nearly 800 Medicines and Vaccines in Clinical Testing For Cancer Offer New Hope to Patients In recent decades, great progress has America’s biopharmaceutical companies Medicines in Development for been made in the fi ght against cancer. are responding to the needs of cancer Cancer Advances in molecular and genomic patients, working to develop more and research have unveiled complexities better treatments. Researchers are now Application Submitted and changed the perception of cancer, exploring new high-tech weapons to Phase III which we now know is more than 200 fi ght the disease as well as ways to use Phase II unique diseases. Continued research existing medicines, either alone or in has expanded our knowledge of how combination with other therapies, to Phase I cancer develops and how to target treat various cancers. The 771 medicines 98 medicines for specifi c cancer types, and vaccines are either in clinical trials which has resulted in more effective or awaiting review by the U.S. Food and therapies for patients. Drug Administration (FDA) and include: 87 A cancer diagnosis once meant almost • A monoclonal antibody for colorectal 78 certain death but with new therapies, cancer that targets a cell surface 73 better testing, earlier diagnosis and protein that plays a role in tumor follow-up care, patients are often able growth and metastases; to live longer with a better quality of life. According to the American Cancer 56 Contents Society (ACS), the number of cancer Medicines and Vaccines in 48 survivors living in the United States has Development to Treat Cancer ...............2 increased from 3 million in 1971 to 13.7 Advances in Cancer Treatment .............3 million in 2012. Value in Cancer Care Although great progress has been made in the United States ............................5 and continues to be made against Research Collaboration: Public-Private Partnerships ...................................... 6 cancer, it remains the second leading Facts About Cancer in the cause of death in the United States, United States .................................... 8 behind heart disease, accounting Medicines and Vaccines in for nearly 1 of every 4 deaths. The Development Chart ........................... 12 ACS estimates that in 2014, more The Drug Discovery, Development than 1.6 million new cancer cases will and Approval Process ...................... 103 ast er er er er e emia Skin be diagnosed and nearly 600,000 Lunganc arian anc Br C C euk Ov Canc Americans will die from cancer, Canc L ymphoma L approximately 1,600 people per day. Some medicines are listed in more than one category. Key Issues • A monoclonal antibody for non-small-cell lung cancer that blocks a signaling pathway allowing for immune responses that can recognize and destroy cancer cells; • A medicine that targets and disrupts a signaling pathway associated with tumor growth in stomach cancer. The development of new therapies would not be possible without the patients who volunteer to participate in clinical trials. Currently, there are 3,137 active clinical trials for cancer in the United States. Of those, 1,824 are now seeking volunteers to participate or have not yet started recruiting patients; the other 1,313 are ongoing but are not currently recruiting new patients.* The progress made against cancer has resulted in better treatments that help extend lives, improve patients’ quality of life, and increase productivity contributing to economic growth. The hundreds of medicines being developed are our best hope for continuing that progress and lessening the burden of cancer. However, researching and developing new medicines remains a risky investment and a lengthy process. On average, it costs $1.2 billion and takes between 10–15 Medicines and Vaccines in years to bring a new, effective medicine to patients. Despite Development to Treat Cancer those risks, America’s biopharmaceutical research companies are continuing their efforts to develop effective new therapies Many of the medicines in the pipeline today are using for the prevention and treatment of cancer. novel approaches to attack cancer at the molecular level. Approximately 80 percent of cancer pipeline drugs are potentially fi rst-in-class treatments, according to the Tufts Center for the Study of Drug Development. Among the 771 medicines and vaccines in development are potential treatments for: Colorectal Cancer—A humanized monoclonal antibody in development for metastatic colorectal cancer targets the cell surface protein “endosialin,” which is expressed on cells that are part of the tumor blood vessel structure. In preclinical studies, blocking the function of endosialin inhibited tumor growth and metastases. Liver Cancer—A medicine in development for hepatocellular carcinoma (liver cancer) is a small molecule kinase inhibitor designed to selectively block transforming growth factor-beta (TGF-beta) signaling. The overexpression of TGF-beta in cells may enhance tumor growth and intensify metastases. *Source: www.clinicaltrials.gov, search performed 9/25/2014. Search criteria: cancer, United States, Phase 0, 1, 2, 3; industry only; excluding unknown status. 2 Medicines in Development Cancer 2014 Key Issues Lung Cancer—A fully human monoclonal antibody in thought to play a role in tumor growth and metastases in development for non-small-cell lung cancer targets the many cancers, including stomach cancer. An exploratory PD-1 (programmed death-1) checkpoint receptor which is biomarker analysis found that the addition of the medicine to expressed on activated T-cells. Cancer cells may exploit chemotherapy improved median overall survival in patients the checkpoint pathway to hide from the immune system with tumors that expressed high levels of MET protein. and protect the tumor. Blocking this pathway may allow for immune responses that recognize and destroy cancer cells. Another medicine which blocks the PD-1 receptor was “ ere is no question that when we talk recently approved by FDA for the treatment of malignant melanoma. about turning the tide against cancer, the most exciting opportunities, Pancreatic Cancer—In malignant solid tumors, oxygen levels are often low (called tumor hypoxia) relative to oxygen levels the new opportunities in fact, are in healthy tissue. Tumor hypoxia is associated with tumor understanding the biology and progression, metastases, and resistance to chemotherapy applying that biology to new treatments. and radiation treatment. A medicine in development is We are certainly at a turning point.” activated when it reaches the hypoxic region of the tumor environment where it eventually kills tumor cells in its vicinity. —J. Leonard Lichtenfeld, M.D., American Cancer Society The medicine is also being tested in patients with soft tissue sarcoma. Stomach Cancer—A monoclonal antibody in testing for Advances in Cancer Treatment stomach cancer with high MET (mesenchymal-epithelial As we continue to advance cancer treatment, progress can transition factor) expression is designed to inhibit hepatocyte be seen in declining death rates from cancer and increases in growth factor (HGF)/scatter factor from binding to the cancer survivors. According to the ACS, the death rate from c-MET receptor. The HGF/c-Met signaling pathway is cancer has been steadily declining since the 1970s—declining more than 15 percent in the period from 2000-2011 alone. Five-year survival rates have increased 39 percent across all cancer types since 1975, with 2 out of 3 people diagnosed with cancer surviving at least fi ve years. To sustain this progress in an environment of increasing pressure to contain health care costs, it is important to understand the valuable role new medicines play in improving patient outcomes. According to the ACS, 83 percent of survival gains are attributable to new treatments, including medicines. Some key advances in treatment include: Personalized Medicines—Personalized medicines are therapeutics that are tailored to individual patients based on certain biologic and physiologic characteristics, such as genetic makeup. Patients can be identifi ed by their susceptibility to a certain disease or how they will likely respond to a specifi c therapy. By targeting therapies to patients most likely to benefi t, personalized medicines represent an important tool, as they may reduce the use of Medicines in Development Cancer 2014 3 Key Issues Targeted Therapies—Targeted therapies are designed to HOW CANCER COULD ECHO interfere with specifi c molecular targets involved in cancer cell growth or progression, whereas traditional chemotherapy HIV SUCCESS acts against all rapidly dividing cells, including healthy ones. Innovations in cancer treatment could echo the Several targeted therapies have been approved for use by success we have seen in HIV/AIDS treatment. the FDA. According to IMS Health, the role of targeted therapies in oncology treatment has grown from 11 percent in Advances in medicines for HIV/AIDS have 2003 to 46 percent in 2013. helped lower the death rate by 83 percent since the disease was fi rst recognized in 1981. Prior to Therapeutic Cancer Vaccines—In the late 1990s, scientists began experimenting with new vaccines that could harness 1995, when the fi rst antiretroviral medicine was the power of the immune system to fi ght cancer rather approved by the FDA, an HIV diagnosis was than to prevent it. One type of therapeutic vaccine, called a death sentence. Now, thanks to medicines