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# FDA Approves Cornerstone Pivotal Trial on Leukemia
- URL: https://www.fdaweb.com/fda-approves-cornerstone-pivotal-trial-on-leukemia/
- Published: 2017-03-23T12:00:00.000Z
- Updated: 2026-09-14T22:13:28.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5138277

FDA has given Cornerstone Pharmaceuticals a green light to conduct pivotal trials of its lead compound, CPI-613, in patients with acute myeloid leukemia (AML) and pancreatic cancer. CPI-613 is the company’s first-in-class anticancer compound from its proprietary Altered Energy Metabolism Directed platform. It 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 company says. The agency has designated CPI-613 an orphan drug status for treating AML, pancreatic cancer and myelodysplastic syndromes.

  
Recent clinical trial data involving 67 patients treated for AML showed anoverall response rate of 50%, including 26 patients in complete remission (CR) and five in CR with incomplete blood count recovery; median survival was 6.7 months, according to the company. “Response was significantly associated with survival, with a median survival of 13.2 months in responders versus three months for all others,” it says.