Mission: 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.

Vision: To extend and enhance the lives of patients with Gastrointestinal (GI) Cancers.

At Rafael Pharmaceuticals, Inc. we are a pioneering, clinical-stage, metabolic oncology therapeutics company, focused on transforming the lives of patients with hard to treat cancers. Our primary objective is to produce highly selective and effective anti-cancer agents with minimal toxic effects on normal cells and tissues.

CPI-613

Rafael Pharmaceutical’s first-in-class clinical lead compound, CPI-613, 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, and II 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 an orphan drug for the treatment of pancreatic cancer, AML, and myelodysplastic syndromes (MDS).

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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.

Boards of Directors

Medical Advisors

Scientific Advisors

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.
Greg Semenza, M.D., Ph.D., Scientific Advisor

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 2016 Lasker Award.
Dafna Bar-Sagi, Ph.D., Scientific Advisor

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.
Jorge Cortes, M.D., Acute Myeloid Leukemia Advisor

Deputy Chair and Professor of Medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center
Steven I. Park, M.D., Lymphoma Advisor

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

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 will work with Cornerstone 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.

Boards of Directors

Medical Advisors

Scientific Advisors

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.
Greg Semenza, M.D., Ph.D., Scientific Advisor

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 2016 Lasker Award.
Dafna Bar-Sagi, Ph.D., Scientific Advisor

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.
Jorge Cortes, M.D., Acute Myeloid Leukemia Advisor

Deputy Chair and Professor of Medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center
Steven I. Park, M.D., Lymphoma Advisor

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

News & Events

January 17, 2019

Rafael Pharmaceuticals to Present Plan for Phase 3 Trial (AVENGER 500) of devimistat (CPI-613®) in Combination with Modified FOLFIRINOX as a First Line Treatment for Metastatic Pancreatic Cancer at 2019 Gastrointestinal Cancers Symposium

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January 10, 2019

EMA Grants Orphan Drug Designation to Rafael Pharmaceuticals’ devimistat (CPI-613®) for Treatment of Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Webcast Recording >>

January 10, 2019

EMA Grants Orphan Drug Designation to Rafael Pharmaceuticals’ devimistat (CPI-613®) for Treatment of Metastatic Pancreatic Cancer

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January 09, 2019

Rafael Pharmaceuticals Announces Initiation of Phase II Trial of CPI-613 in Combination with Modified FOLFIRINOX in Locally Advanced Pancreatic Cancer

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January 09, 2019

Rafael Pharmaceuticals Announces Initiation of Patient Enrollment for Phase II Clinical Trial of CPI-613 for Patients with Relapsed or Refractory Burkitt Lymphoma/Leukemia

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January 07, 2019

Rafael Pharmaceuticals to Present at the 37th Annual J.P. Morgan Healthcare Conference

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