Cornerstone Pharmaceuticals gets Approval to Conduct Phase I/II Clinical Trial of CPI-613 in Cancer Patients by US FDA

25 th July 2008

Cornerstone Pharmaceuticals, Inc., a privately held pharmaceutical company, has received clearance from the US Food and Drug Administration (FDA) to begin a Phase I/II clinical trial evaluating the safety and early efficacy of its first-in-class Altered Energy Metabolism-Directed (AEMD) compound, CPI-613, in a variety of cancer types.

CPI-613 targets distinctive changes in the energy generating processes associated with the vast majority of solid tumor types, according to preclinical studies. CPI-613 has shown possible utility in multiple preclinical studies including studies conducted using human tumor biopsies from patients bearing lung, colon, pancreatic and breast tumors as well as cancer cell lines resistant to traditional chemotherapeutics.

The approved clinical trial will be open to patients with a variety of tumor types who have failed previous therapies. It will be conducted at a limited number of clinical trial sites in North America.

CPI-613 represents a subclass of compounds from Cornerstone’s AEMD platform which the company has named “Thioctans,” and which it believes kill cancer cells by an entirely new and highly selective mechanism.  Cornerstone has been granted Orphan Drug Designation by the US FDA for the use of CPI-613 in the treatment of pancreatic cancer.