Cornerstone Pharmaceuticals announced its first –in-class cancer metabolism inhibitor drug will be featured in a poster session at the 53rd Annual Meeting and Exposition of the American Society of Hematology (ASH).  Findings from the study will be presented along with preclinical findings at ASH from a Phase 1 clinical trial of CPI-613 in refractory acute leukemias. In August 2011, CPI-613 received FDA orphan drug designation for the treatment of acute myeloid leukemia (AML).

CPI-613 is the subject of a Phase 1 clinical single agent trial to evaluate safety, tolerability, maximum tolerated dose, efficacy, and pharmacokinetics for patients with relapsed and refractory hematologic malignancies. To date, eleven patients have been treated with at least one cycle of CPI-613. Interim clinical data suggests that altered lipid and mitochondrial metabolism are viable targets in acute leukemias. CPI-613 is a novel agent that has activity against several acute leukemia cell lines in vitro and in vivo and demonstrates activity in patients with relapsed AML and Myelodysplastic Syndrome (MDS). Several patients experienced significant clinical benefit, including a sustained complete remission when treated with CPI-613 as a single agent.  Only minor toxicities were observed.

According to the National Cancer Institute, one in 77 men and women will be diagnosed with leukemia during their lifetime. In adults these diseases disproportionally affect the elderly with a median age of onset of 72 for acute myeloid leukemia. Patients over the age of 60 have less than 10 percent survival at five years. It is estimated that 12,950 Americans (6,830 men and 6,120 women) will be diagnosed with acute myeloid leukemia in 2011. Chemotherapy is the standard of treatment for untreated adult.  Cornerstone’s CPI-613 may represent a potential new alternative treatment for this disease.

“This study demonstrates early proof of concept for this class of agents, which is very important because relatively few treatment options exist for patients with myeloid leukemia, especially with relapsed/refractory disease,” said the CEO of Cornerstone Pharmaceuticals, Robert Shorr, Ph.D. “Preliminary results from the ongoing Phase 1 study demonstrate the safety of this first-in-class agent, CPI-613, with very few adverse events, and support further exploration of CPI-613 as a potential treatment for AML and other hematologic malignancies.”

Date: December 12, 2011
Source: Cornerstone Pharmaceuticals