CPI-613

Rafael’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.


CPI-613

Rafael’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.


Developed as part of Rafael’s proprietary Altered Energy Metabolism Directed (AEMD) drug platform, CPI-613 was discovered at Stony Brook University. CPI-613 is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, an indispensable process essential to tumor cell multiplication and survival, selectively in cancer cells.

CPI-613’s attack on the TCA cycle also substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for combinations of CPI-613 with lower doses of these generally toxic drugs to be highly effective with lower patient side effects. Combinations with CPI-613 represent a diverse range of potential opportunities to substantially improve patient benefit in many different cancers.

The U.S. Food and Drug Administration (FDA) has given Cornerstone 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). As a next step, Rafael intends to apply for Orphan Drug designation for MYC amplified lymphoma/Burkitt lymphoma and T-cell lymphoma.


Learn more about recent developments involving CPI-613: CPI-613 Fact Sheet

Developed as part of Rafael’s proprietary Altered Energy Metabolism Directed (AEMD) drug platform, CPI-613 was discovered at Stony Brook University. CPI-613 is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, an indispensable process essential to tumor cell multiplication and survival, selectively in cancer cells.

CPI-613’s attack on the TCA cycle also substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for combinations of CPI-613 with lower doses of these generally toxic drugs to be highly effective with lower patient side effects. Combinations with CPI-613 represent a diverse range of potential opportunities to substantially improve patient benefit in many different cancers.

The U.S. Food and Drug Administration (FDA) has given Cornerstone 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). As a next step, Rafael intends to apply for Orphan Drug designation for MYC amplified lymphoma/Burkitt lymphoma and T-cell lymphoma.


Learn more about recent developments involving CPI-613: CPI-613 Fact Sheet