PFK-158 is a small molecule therapeutic candidate that inactivates a novel cancer metabolism target never before examined in human clinical trials.It has been licensed by ACT from the James Graham Brown Cancer Center at UofL. Last spring, the U.S. Food and Drug Administration (FDA) approved Phase 1 dose escalation study that is evaluating the safety, tolerability and anti-tumor activity of PFK-158 in cancer patients with solid tumors such as melanoma, lung, colon, breast and pancreatic cancer.

Randall B. Riggs, President and CEO of ACT, presented during Informa’s Therapeutic Area Partnerships (TAP) meeting held November 19-21, 2014 at the Hyatt Regency in Boston. Mr. Riggs’ presentation, on November 20 within Track 3 (Oncology), highlighted PFK-158, a first-in-man / first-in-class inhibitor of PFKFB3, an enzyme that controls glycolysis and that is overexpressed in most hematological and solid tumors.

A product selected as one of the “2014 Top 10 Most Interesting Oncology Projects to Watch” list has met rigorous criteria, including unmet medical need, market potential, diversity of indications, strong science, partnering opportunities, and potential for new opportunities beyond initial indications.

Selected products have been screened using a strict set of judging criteria for the Top 10 award and represent what Informa and Kantar Health consider among the most attractive opportunities the industry has to offer, said Bill Bagwell, General Manager, Oncology, Kantar Health. “As an industry leader in oncology strategic research and consulting, it is exciting to give these companies exposure to potential investors, partners and acquirers.”

PFK-158 is the first 6-phosphofructo-2-kinase/fructose-2,6-biphosphatase 3 (PFKFB3) inhibitor to undergo clinical trial testing in cancer patients. The target, PFKFB3, is activated by oncogenes and the low oxygen state in cancers, stimulates glucose metabolism and is required for the growth of cancer cells as tumors in mice. PFK-158, which has been licensed by ACT from the James Graham Brown Cancer Center at the University of Louisville, inhibits the substrate binding domain of PFKFB3 causing a marked reduction in the glucose uptake and growth of multiple cancer types in mice.  

PFK-158 human clinical trials began recruiting patients in May 2014 with the first clinical trial site located at the James Graham Brown Cancer Center, a part of KentuckyOne Health. Within weeks of opening the first clinical trial site, ACT was able to open the second clinical trial site, Georgetown University Medical Center in Washington, D.C., also in May 2014.

Mr. Riggs added, “We were excited to present PFK-158 at this prestigious conference. PFK-158 is a first-in-man, novel anti-cancer drug that prevents tumor cells from using glucose as a fuel source for tumor survival, growth and metastasis and is currently in a Phase 1 clinical study in the US.”  

About Advanced Cancer Therapeutics (ACT):

ACT is a privately held company dedicated to advancing novel therapeutics for the prevention and treatment of cancer. ACT has successfully established a unique and innovative business model with the University of Louisville’s James Graham Brown Cancer Center (Brown Cancer Center) whereby ACT is able to obtain exclusive worldwide licenses to novel cancer therapeutics discovered at Brown Cancer Center under preset business terms. ACT then fast-tracks these discoveries, including the selection process for partnership, commercialization and manufacture, to the pharmaceutical industry, and ultimately to the patients who need them. Led by Dr. Donald M. Miller, the Brown Cancer Center employs more than 50 scientists focused on the discovery and advancement of breakthrough cancer therapeutics for patients suffering from cancer. For more information, please visit