Phase 1 trials begin for first-in-class anticancer drug

Clinical trials have begun for PFK-158, a small-molecule therapeutic candidate that inactivates a novel cancer metabolism target never before examined in human clinical trials. The FDA-approved, Phase 1, dose-escalation study is evaluating the safety, tolerability, and antitumor activity of PFK-158 in cancer patients with solid tumors, such as melanoma, lung, colon, breast, and pancreatic cancers.

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 Advanced Cancer Therapeutics (ACT) from the James Graham Brown Cancer Center at the University of Louisville, in Kentucky, inhibits the substrate binding domain of PFKFB3. This causes 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 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 its second clinical trial site at Georgetown University Medical Center in Washington, DC, also in May.

“PFK-158 is not only a first-in-class cancer drug but also the first to target glucose metabolism by inhibiting PFKFB3. This unique mechanism of action has resulted in efficacy against a broad spectrum of human cancers caused by common mutations as well as synergy with targeted agents that are FDA approved for several cancer types,” said Jason A. Chesney, MD, PhD, deputy director of the Brown Cancer Center and a global thought leader and researcher in cancer metabolism.

“As a researcher, it is incredibly rewarding to witness your group's studies move into clinical trials and potentially save the lives of cancer patients,” Chesney said.

“This is a significant milestone for ACT and it supports our dedication to develop significant treatment advancements for cancer patients with first-in-class, potential breakthrough therapeutics like PFK-158,” said Randall B. Riggs, president and chief executive officer of ACT.

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