Following successful End-of-Phase I Type B meetings with the FDA, where updated results from phase I trials of CPI-613 in patients with acute myeloid leukemia (AML) and pancreatic cancer were presented, the initiation of pivotal trials of the drug in these patient populations has been approved.
CPI-613, a first-in-class anticancer compound, is designed to disrupt the altered energy production pathways in cancer cells by targeting the mitochondrial tricarboxylic acid cycle. The FDA has previously designated CPI-613 an orphan drug for the treatment of AML, pancreatic cancer, and myelodysplastic syndromes.
"We believe the phase II trial in AML can potentially lead to approval for treatment of a disease that historically has been extremely difficult to treat," said Jorge Cortes, MD, lead investigator of the AML trials and Deputy Chair and Professor of Medicine, MD Anderson Cancer Center, Houston.
"We are encouraged the FDA has granted us the opportunity to continue clinical trials in pancreatic cancer, a disease with large unmet clinical need, and are optimistic we will continue to see positive results in advanced pancreatic cancer patients," noted Philip A. Philip, MD, PhD, FRCP, lead investigator of the pancreatic cancer trials and Professor of Oncology and Pharmacology; Leader, GI and Neuroendocrine Oncology; and Vice President of Medical Affairs at Karmanos Cancer Center, Detroit.