To develop highly selective, well tolerated, and highly effective anti-cancer agents, by selectively targeting altered metabolism in cancer cells. Our immediate goal is to improve the quality of life of patients with Pancreatic Cancer, which is the deadliest cancer worldwide and has limited treatment options.
To extend and enhance the lives of patients with Gastrointestinal (GI) Cancers.
Learn more about our Clinical Trials:
Rafael Pharmaceutical’s first-in-class clinical lead compound, CPI-613® (devimistat), targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. CPI-613® is being evaluated in multiple Phase I, I/II, II, and III clinical studies as a single agent, as well as in combination with standard drug therapies, in patients diagnosed with advanced solid tumors or blood cancers.
The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal clinical trials in pancreatic cancer and acute myeloid leukemia (AML), and has designated CPI-613® as an orphan drug for the treatment of pancreatic cancer, AML, Myelodysplastic syndromes (MDS), peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to CPI-613® for pancreatic cancer and AML.
Chi Van Dang, M.D., Ph.D.
Chairman of Rafael’s Scientific and Medical Advisory Boards
Director of the Abramson Cancer Center of the University of Pennsylvania. Dr. Dang is considered by many to be the world’s leading authority on cancer metabolism. Dr. Dang currently serves as Rafael’s Chairman of the Scientific and Medical Advisory Boards.
Jorge Cortes, M.D.
Acute Myeloid Leukemia Advisor
Director of The Georgia Cancer Center at Augusta University. He is designated as a Georgia Research Alliance Eminent Scholar in Cancer in the Medical College of Georgia.
Steven I. Park, M.D.
Division Chief, Hematology and Oncology, Levine Cancer Institute, CHS-Northeast; Director of Investigational Therapeutics in Hematologic Malignancies, Levine Cancer Institute/Carolinas HealthCare System; Associate Professor of Medicine, University of North Carolina, Chapel Hill
Dafna Bar-Sagi, Ph.D.
Senior Vice President, Vice Dean for Science, Chief Scientific Officer, Professor, Departments of Biochemistry and Molecular Pharmacology, and Medicine, NYU Langone Medical Center
Philip A. Philip, M.D., Ph.D., FRCP
Pancreatic Cancer Advisor
Professor of Oncology and Pharmacology, Leader, GI and Neuroendocrine Oncology, Vice President of Medical Affairs, Karmanos Cancer Center, Wayne State University, Detroit, MI.
Greg Semenza, M.D., Ph.D.
Noble Prize in Physiology and Medicine
Director, Vascular Program, Institute for Cell Engineering; Professor of Pediatrics, Radiation Oncology and Molecular Radiation Sciences, Biological Chemistry, Medicine, and Oncology at the Johns Hopkins University School of Medicine. Dr. Semenza won The Nobel Prize in Physiology or Medicine in 2019 and 2016 Lasker Award.
World Class Team
Innovation in oncology drug development requires a deep well of scientific, medical and business expertise, and we have assembled an unparalleled team of directors and advisors who are working with Rafael to revolutionize anticancer treatment.
Our Board of Directors and Scientific & Medical Advisory Boards are drawn from the highest echelons of oncology and hematology researchers and physicians, and include seasoned managers from the biotech and pharmaceutical sectors.