7:51 PM
 | 
Mar 24, 2017
 |  BioCentury  |  Product Development

Pivotal meeting

How Cornerstone’s CPI-613 leapfrogged to pivotal trials in AML, pancreatic cancer

Cornerstone Pharmaceuticals Inc. was planning a traditional development pathway for CPI-613 in acute myelogenous leukemia and pancreatic cancer, but FDA nudged the company to go from Phase I to pivotal trials for both indications.

According to COO Sanjeev Luther it was FDA, not the company, that suggested an accelerated development timeline based on the strength of the Phase I data.

Cornerstone is finalizing its pivotal study protocols and plans to launch two trials this year to evaluate CPI-613 plus standard of care (SOC). One will enroll relapsed and refractory AML patients aged 60 and older, and the other newly diagnosed stage IV pancreatic cancer patients.

CPI-613 is an analog of alpha-lipoic acid that disrupts abnormal metabolic pathways specific to cancer cells.

In normal cells, the primary metabolic goal is to generate energy in the form of ATP, which is produced in mitochondria. In most types of cancer cells, however, the function of mitochondria is shifted toward producing nutrients needed for biosynthesis via the tricarboxylic acid (TCA) cycle.

Cancer cells bring carbons into the TCA cycle to create backbones for synthesizing amino acids and nucleotides, which enables them to maintain high rates of replication and quickly repair damage following chemotherapy.

The mitochondrial enzymes pyruvate dehydrogenase (PDH) and alpha-ketoglutarate control the entry of carbon into the TCA cycle.

CPI-613 inhibits both enzymes, blocking the pathway cancer cells rely on for replication and repair. In doing so, the compound aims to increase the efficacy of SOC.

There’s a lot of potential to combine with targeted agents.”

Tim Pardee, Cornerstone


The compound has Orphan Drug designation in AML, pancreatic cancer and myelodysplastic syndrome (MDS).

Chief Oncologist Tim Pardee told BioCentury Cornerstone chose to start with AML and...

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