Rafael Pharmaceuticals Receives Fast Track Designation for CPI-613

Rafael Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s lead compound, CPI-613® (devimistat), for the treatment of metastatic pancreatic cancer.

“Receiving Fast Track designation is a significant milestone in our fight against pancreatic cancer,” said Sanjeev Luther, President and CEO of Rafael. “This designation further stresses the severe unmet need in treatment options for this aggressive and devastating disease. We are truly thankful to the doctors, researchers, the FDA and all of our supporters who have made this possible. And most of all, we are grateful to the patients involved in our trials; the patients are the inspiration and driving force behind all of our efforts.”

Rafael has continued to reach milestones throughout the year, including achieving its target enrollment of 500 patients for its Phase 3 trial for metastatic pancreatic cancer ahead of schedule. The Company also recently announced that the FDA granted devimistat Orphan Drug Designation for the treatment of soft tissue sarcoma.

“Pancreatic cancer is notoriously challenging to treat and long overdue for a new approach,” said Philip A. Philip, M.D., Ph.D., FRCP, Professor of Oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University and a medical advisor to Rafael. “We have remained hopeful throughout our pancreatic cancer trials, and now with Fast Track designation, our optimism is further fueled. We believe with this designation, cancer metabolism is truly being propelled forward, with devimistat at the helm.”

CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500®) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, Burkitt’s lymphoma and soft tissue sarcoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.