1. Pancreatic Cancer in the US – 12/2015 TIMOTHY PAULUS - TESTIMONY 1/20/2016

2. Timothy Paulus  Diagnosed in March 2015  Have gone through a complete chemotherapy regiment  Am currently in a second regiment because the tumors grew through the first regiment  Currently exploring clinical trials and specific drugs that might help

3. Basic Statistics  Pancreatic Cancer (Pancreatic ductal adenocarcinoma (PDA)) is a lethal disease and is one of the cancers that is most resistant to traditional therapies. Historically, neither chemotherapy nor radiotherapy has provided any significant increase in the survival of patients with PDA. Despite intensive efforts, any attempts to improve the survival in the past 15 years have failed. This holds true even after the introduction of molecularly targeted agents, chosen on the basis of their involvement in pathways that are considered to be important in PDA development and progression. Recently, however, FOLFIRINOX (5-fluorouracil, leucovorin, irinotecan, and oxaliplatin) treatment has provided a limited survival advantage in patients with advanced PC.

4. Basic Statictics  Although lung, breast, prostate, and colorectal cancers are considered to be the “big four” cancer types in the USA, pancreas and liver cancers are expected to surpass breast, prostate, and colorectal cancers to become the second and third leading causes of cancer-related deaths by 2020 respectively. In the USA, there were an estimated 48,960 cases of new- onset pancreatic cancer in 2015, which led to 40,560 deaths, with a 5- year survival rate of ∼ 7%. Pancreatic Cancer is nearly universally lethal; 20% of patients are suitable candidates for surgery at the time of diagnosis, and the median survival rate is 3.5 months and 12.6 months for nonresected patients and resected patients, respectively.  During the week of 1/11/2016 Pancreatic Cancer moved up to the #3 spot.

5. Snapshot of Pancreatic Cancer  2015 estimates: 48,960 diagnoses, 40,560 deaths. Third leading cause of cancer death, behind only lung and colorectal. Note: Ahead of breast. 6.9% of all cancer deaths. PC will be the second leading cause of cancer death by 2020.  91% are diagnosed with metastatic disease for which there is no effective treatment. Surgery, available only for non-metastatic disease, offers the sole prospect of a cure, but even this leads to an average survival of just 18 - 20 months.  3-6 months average life expectancy at diagnosis. 74% will die within one year. 7% survive 5 years.  Equally affects men and women  Vast majority of incidence is at age 45+  33% of incidence is in minority populations: Black, Hispanic, Asian/Pacific Islander, Native Indian.

6. FDA Approval Process  FDA Approval Process is approximately 12 years  Once a company develops a drug, it undergoes around three and a half years of laboratory testing, before an application is made to the U.S. Food and Drug Administration (FDA) to begin testing the drug in humans.  Phase 1 uses 20-80 healthy volunteers to establish a drug's safety and profile. (about 1 year)  Phase 2 employs 100-300 patient volunteers to assess the drug's effectiveness. (about 2 years)  Phase 3 involves 1000-3000 patients in clinics and hospitals who are monitored carefully to determine effectiveness and identify adverse reactions. (about 3 years)  The company then submits an application (usually about 100,000 pages) to the FDA for approval, a process that can take up to two and a half years.

7. Steps from Test Tube to New Drug Application Review

8. Vice-President Biden’s Cancer Moonshot 2020  1) Increase resources — both private and public — to fight cancer.  2) Break down silos and bring all the cancer fighters together — to work together, share information, and end cancer as we know it.  Several cutting-edge areas of research and care — including cancer immunotherapy, genomics, and combination therapies — could be revolutionary. Innovations in data and technology offer the promise to speed research advances and improve care delivery.  Only 5 percent of cancer patients in the U.S. end up in a clinical trial.

9. Thanks  Clinical Trials are very important for long-term knowledge and research  Safety is a guiding principle for the FDA but…  Due to the life-cycle of Pan Can there can be good outcomes for “Right to Try” procedures – compare the FDA cycle to the expected life expectancy for Pan Can patients