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).
News & Events
April 2018
A letter to shareholders, from Sanjeev Luther, Rafael Pharmaceuticals’ CEO
April 12, 2018
Clinical Cancer Research Publishes Phase I Data from a Study of Rafael Pharmaceuticals’ Lead CPI-613 Compound in Combination with High-Dose Cytarabine and Mitoxantrone for Relapsed or Refractory Acute Myeloid Leukemia
December 07, 2017
Rafael Pharmaceuticals to Present New Data Validating Efficacy of CPI-613 in Treating Acute Myeloid Leukemia at the 2017 American Society of Hematology Annual Meeting & Exposition
November 16, 2017
Rafael Pharmaceuticals, in Collaboration with Atlantic Health System, Announces Initiation of a Phase I Study of CPI-613 in Combination with Gemcitabine and Nab-Paclitaxel for Patients with Locally Advanced or Metastatic Pancreatic Cancer
May 9, 2017
The Lancet Oncology Publishes Phase I Data Demonstrating the Safety, Tolerability, and Preliminary Efficacy of Cornerstone Pharmaceuticals’ CPI-613 in Combination with Modified FOLFIRINOX in Patients with Metastatic Pancreatic Cancer
March 23, 2017
FDA Provides Cornerstone Pharmaceuticals Approval to Initiate Pivotal Study for CPI-613, its Metabolism-Directed Anticancer Compound