Cornerstone Pharmaceuticals and Wake Forest Baptist Medical Centre announced key findings from a phase I clinical trial of Cornerstone’s first –in-class cancer metabolism inhibitor drug, CPI-613 in Acute Myeloid Leukaemia (AML). Data will be presented at the 47th Annual meeting of American Society of Clinical Oncology (ASCO).

Cornerstone is a leader in the discovery and development of cancer therapies based on the science of cancer metabolism. CPI-613 is being studied at Wake Forest Baptist’s Comprehensive Cancer Centre to evaluate safety, tolerability, maximum tolerated dose, efficacy, and pharmacokinetics for patients with relapsed and refractory haematologic malignancies.

The novel agent, CPI-613, has activity against several acute leukaemia cell lines and may have activity in patients with relapsed disease. The mode of cytotoxicity and degree of synergy with more traditional agents remains a focus of ongoing study. “This study is important because relatively few treatment options exist for patients with AML,” said Robert Shorr, PhD, CEO Cornerstone Pharmaceuticals. “Preliminary results from the ongoing phase I study demonstrate the safety of 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 haematologic malignancies” he added.

According to the National Cancer Institute, 1 in 77 men and women will be diagnosed with leukaemia during their lifetime. It is estimated that of the 43,050 Americans diagnosed annually, 21,840 of those will die of leukaemia in 2011. Chemotherapy is the standard of treatment for untreated adult Acute Myeloid Leukaemia (AML). Cornerstone’s CPI-613 may represent a potential new alternative treatment for this disease “I am very excited at the opportunity to present our data from the lab as well as our ongoing phase I trial at ASCO,” said Timothy S Pardee, MD, PhD, lead investigator for the study. “CPI-613 is a very intriguing compound and I hope our studies will lead to improved care for patients who suffer haematologic malignancies.” Pardee will present the data at ASCO June 3-7 in Chicago. The poster session for the study will be on Monday, June 6 from 1 p.m. to 5 p.m. at McCormick Place Hall A.

CPI-613 is the first drug in a new chemical class that, through a novel mechanism, targets metabolic changes considered to be common to many, if not all, cancer types and not present in normal cells. Patients with solid tumours and haematologic cancers are currently being enrolled in multiple phase I and phase I/II human clinical trials evaluating CPI-613. These trials include a phase I/II single agent trial for patients with solid tumours who have failed all other therapy options, a phase I/II combination trial with gemcitabine in newly diagnosed or relapsed patients, and a single agent trial in haematologic malignancies. CPI-613 was granted orphan drug status by the US FDA for pancreatic cancer, which has a poor prognosis, spreads rapidly and often goes undetected in its early stages.

Cornerstone Pharmaceuticals, Inc. is a privately held company that is committed to changing the way cancer is treated through the discovery and development of innovative therapies capitalizing on the unique metabolic processes of cancer cells.

Wake Forest Baptist Medical Centre is a fully integrated academic medical centre and Wake Forest School of Medicine directs the education and research components, with the medical school ranked among the nation’s best and recognized as a leading research centre in regenerative medicine, cancer, the neurosciences, ageing, addiction and public health sciences. Piedmont Triad Research Park, a division of Wake Forest Baptist, fosters biotechnology innovation in an urban park community.