Cornerstone Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, has started phase II clinical trial of CPI-613, its lead Altered Energy Metabolism Directed (AEMD) drug candidate. The pilot study, sponsored by the Comprehensive Cancer Centre of Wake Forest Baptist Medical Centre, will evaluate the overall response rate for CPI-613 in patients with myelodysplastic syndrome (MDS) who have been unresponsive to previous therapies.

Secondary endpoints include overall survival, progression-free survival, safety, and transfusion frequency.

Earlier this year, the Company announced the initiation of a Wake Forest Baptist-sponsored phase I trial investigating the combination of CPI-613 with high dose cytarabine and mitoxantrone in relapsed or refractory acute meylogenous leukaemia (AML) patients. Both of these trials have been launched following the encouraging results of a recently completed phase I single agent trial of CPI-613 in patients with hematological malignancies similarly sponsored by CCCWFU.

The results from this study were presented by Dr Pardee at the 2013 American Society for Clinical Oncology (ASCO) Annual Meeting and was hand-selected for inclusion in the Best of ASCO 2013 educational series.

“The signals of activity and the tolerability profile demonstrated by CPI-613 in the earlier phase I study have motivated us to move forward in the clinic with this novel compound,” said Dr Timothy Pardee, MD, Ph.D. of Wake Forest Baptist, who also serves as the Principal Investigator for this trial. “We look forward to further evaluating CPI-613 in treatment-resistant MDS patients, with the hope of improving their outcomes and quality of life.”

“It has been a special honor to continue working with Wake Forest Baptist and its world class team of investigators,” said Robert Rodriguez, Cornerstone’s president and chief operating officer. “In the earlier phase I study we observed complete remission or stable disease in several relapsed/refractory MDS patients. We look forward to the possibility of extending these encouraging results and offering a novel, better-tolerated, therapeutic alternative treatment for MDS patients.”

CPI-613 is the lead drug candidate from Cornerstone's proprietary  AEMD platform. Cornerstone’s AEMD drug platform disrupts biochemical alterations in the conversion of glucose to energy that occur in many types of cancer cells. These essential "bioenergetic" differences are linked to pathways that support, among other things, cancer cell growth and development. The platform is designed to produce drugs, such as the company’s lead drug CPI-613, that disrupt energy-production pathways whose organization or regulation are altered specifically in cancer cells. CPI- 613 is currently being evaluated in phase I, I/II and phase II trials.

Wake Forest Baptist Medical Centre is a fully integrated academic medical centre and comprises of Wake Forest School of Medicine, a leading centre for medical education and research; Wake Forest Baptist Health, the integrated clinical structure that includes nationally ranked Brenner Children’s Hospital; Wake Forest Innovations, which promotes the commercialization of research discoveries and operates Wake Forest Innovation Quarter, an urban research and technology park; plus a network of affiliated community hospitals, physician practices, outpatient services and other medical facilities.