Phase 2 Safety, Tolerability and Efficacy Study of CPI-613 in Cancer Patients

Phase 2 Safety, Tolerability and Efficacy Study of CPI-613 in Cancer Patients

Solid Tumors

Terminated

2

Start Date 06/01/2013

Age of Trial (yrs) 9.8

Gleevec-resistant

SDH-directed

Krebs cycle modulation

GIST not specified. GIST patient enrollment unknown.

CL-CPI-613-023

Cornerstone Pharmaceuticals, Inc.

King C Lee, Ph.D. 203-239-2021 king@cornerstonepharma.com Claudia B Maturo, M.Sc. 631-444-6868 claudia@cornerstonepharma.com

Intravenous

This Phase II study is conducted to assess the safety and efficacy of CPI-613 in patients with advanced and/or metastatic solid tumors for whom there there is no available therapy to provide clinical benefit or for those who have refused further standard therapy. The primary outcome measure is Overall Survival (OS). The secondary outcome measures are: Response Rate (RR), Progression-Free Survival (PFS), and safety.

From Cornerstone's website:

"Altered Energy Metabolism Directed (AEMD)

Cornerstone’s proprietary AEMD small molecule compounds are believed to exploit the aberrant energy metabolism of cancer cells and its link to pathways controlling cancer cell growth, development, apoptosis and necrosis. CPI-613, the lead product candidate from the Company’s AEMD platform has undergone extensive in-vitro and in-vivo anti-tumor studies as well as animal toxicology studies which have demonstrated highly desirable characteristics that strongly warrant clinical evaluation of these agents in humans.

CPI-613 is being evaluated as a single agent therapy in Phase I/II clinical trial in multiple cancers in both the United States and Canada. The US FDA has also granted Orphan Drug Status to CPI-613 for the treatment of pancreatic cancer. Accordingly, the Company has initiated a Phase I/II trial in combination with gemcitabine for newly diagnosed pancreatic cancer patients or those with tumors intended to be treated with gemcitabine. Among the more salient AEMD compound characteristics observed in the preclinical setting are:
* A high degree of cancer cell selectivity
* Broad spectrum activity against a wide variety of cancer cell lines representing different genetic mutations, including cancer cell lines characterized as being multiple drug resistant
* A high degree of tolerability in animal models resulting in an unusually significant therapeutic index"