Initiation Of Clinical Trials Of CPI-613 For The Treatment Of Cancer - First-In-Class Altered Energy Metabolism-Directed Compound

Cornerstone Pharmaceuticals Inc., a privately held pharmaceutical company, announced today that it has begun dosing patients in a recently approved safety and early efficacy clinical trial of CPI-613, its novel first-in-class Altered Energy Metabolism-Directed (AEMD) compound being evaluated for potential use in a wide array of cancers. Health Canada and the US FDA have approved the initiation of studies of CPI-613, which will be conducted at clinical trial sites in both the US and Canada.

"The recent regulatory approvals and commencement of this clinical trial in both the US and Canada represent significant milestones in the global development of the company's AEMD compound platform. Given the novelty of the proposed mechanism of action and the highly encouraging pre-clinical efficacy and safety data, we look forward to advancing the development of this promising class of agents to therapeutic fruition for patients suffering from many types of cancer," said Robert J. Rodriguez, President and Chief Operating Officer of Cornerstone.

Recent molecular oncology studies have focused on the significant genetic differences between different types of cancers. However, it has long been recognized that metabolic energy processes in the majority of cancer cells are similar to each other, but quite distinct from that of normal cells. This observation, first made by Nobel Laureate Otto Heinrich Warburg in 1924 (the "Warburg Effect"), forms the basis for recent significant advances in cancer imaging by positron emission tomography (PET). Because this altered energy metabolism is common to many types of cancer but not normal cells, it is also an attractive target for therapeutic development.

CPI-613 and related compounds are well tolerated in several different animal tumor models at doses that significantly exceed the effective anti-tumor doses in these models. These findings, among others, led to the decision to seek regulatory approval to begin clinical trials.  The approved trial is designed to be an open label, dose-escalation study to evaluate safety, tolerability, maximum tolerated dose, efficacy, and pharmacokinetics of CPI-613 in multiple types of cancer. Additional information regarding this clinical trial may be found at http://www.clinicaltrials.gov.

The AEMD technology platform being developed by Cornerstone is based upon the research of Paul M. Bingham, Ph.D. and Zuzana Zachar, Ph.D., Stony Brook University, Stony Brook, NY. Recently, Cornerstone Pharmaceuticals and Professors Bingham and Zachar were awarded a matching Innovative Technology Development grant from the Center for Biotechnology, a NYSTAR Center for Advanced Technology at Stony Brook University.  "Given the competitive nature of these grants and the extensive peer review required, we are encouraged by the outside expert evaluation and validation of our efforts and the support of the Center for Biotechnology," said Cornerstone CEO Rob Shorr, Ph.D., D.I.C.

About Cornerstone Pharmaceuticals
Cornerstone Pharmaceuticals, Inc. is a privately held pharmaceutical company singularly focused on the discovery and development of innovative cancer therapies that exploit the metabolic pathways that are common to different cancer types but different from normal cells and tissues. This unique approach, i.e. understanding and addressing what is similar to multiple cancer types rather than the differences between each, offers a significant opportunity to make a profound impact on the clinical treatment of a variety of cancers.  Cornerstone is currently developing two distinct technology platforms that show strong specificity for cancer cells of multiple types:
- Altered Energy Metabolism-Directed (AEMD) small molecule drugs; and
- Emulsiphan, a cancer-selective nanotechnology-based delivery system