FDA Provides Cornerstone Pharmaceuticals Approval to Initiate Pivotal Study for CPI-613, its Metabolism-Directed Anticancer Compound

CRANBURY, N.J., March 23, 2017

CPI-613 is Cornerstone Pharmaceuticals' first-in-class anticancer compound from its proprietary Altered Energy Metabolism Directed (AEMD) platform. The drug is designed to disrupt the altered energy production pathways in cancer cells by targeting the mitochondrial tricarboxylic acid (TCA) cycle, an indispensable process essential to cell multiplication and survival. The FDA has designated CPI-613 an orphan drug for the treatment of acute myeloid leukemia (AML), pancreatic cancer and myelodysplastic syndromes (MDS).

"Our productive dialogue with the FDA has cleared a path forward for initiating pivotal trials of CPI-613, which may be sufficient to support our proposed indications for the treatment of AML and pancreatic cancer," said Sanjeev Luther, Chief Operating Officer, Cornerstone Pharmaceuticals. "The FDA has provided valuable feedback to inform a design that will enhance the robustness of our trials. This would allow the Agency to assess them as registrational trials. We are confident in our ability to meet the FDA's requirements for the trials, and look forward to continued interaction with the FDA as the CPI-613 clinical development program advances."

During the EOP1 Type B meetings with the FDA, Cornerstone Pharmaceuticals presented updated results from Phase I trials of CPI-613 in patients with AML and pancreatic cancer.

"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, M.D., lead investigator of the AML trials and Deputy Chair and Professor of Medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center.

"We are encouraged that the FDA has granted us the opportunity to continue clinical trials in pancreatic cancer, a disease with large unmet clinical need, and are optimistic that we will continue to see positive results in advanced pancreatic cancer patients," commented Philip A. Philip, M.D., Ph.D., F.R.C.P., 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, Wayne State University.

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