Instant Report

Date: 23-Feb-2021

Eisai Gets Japanese Orphan Drug Status To Novel FGF Receptor Selective TKI E7090 With Prospective Indication For Unresectable Biliary Tract Cancer Wit

Eisai Co, Ltd announced that it has received orphan drug designation for a prospective indication for unresectable biliary tract cancer with FGFR2 gene fusion by the Ministry of Health, Labour and Welfare, Japan (MHLW) for its in-house discovered fibroblast growth factor (FGF) receptor (FGFR1, FGFR2, FGFR3) selective tyrosine kinase inhibitor (TKI) E7090, which is currently under development as an orally available novel anti-cancer agent.

FGFRs with genetic aberrations are known to play an important role in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. These genetic aberrations in FGFRs have been observed in various types of cancers, therefore, there is growing interest in FGFRs as a promising target for cancer therapy. By selectively inhibiting FGFR1, 2 and 3, and blocking those signals, E7090 has the potential to become a new molecular targeted therapy for cancers with FGFR genetic aberrations.

In Japan, a phase I clinical trial of E7090 was conducted, and E7090 has been designated as the target drug for the SAKIGAKE Designation System of the MHLW for the treatment of unresectable biliary tract cancer. Currently, a phase II clinical trial (Study 201) of E7090 is underway in patients with cholangiocarcinoma with FGFR2 gene fusion in Japan and China.

Discovered in-house by Eisai’s Tsukuba Research Laboratories, E7090 is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against fibroblast growth factor receptors (FGFR) FGFR1, FGFR2 and FGFR3. Distinct from prior known FGFR inhibitors, E7090 has a basic structure which lacks the dimethoxyphenyl moiety, and in a kinetic interaction analysis study, it was observed that E7090 demonstrates antitumor effects due to inhibition of kinase activity with a binding mode (Type V) that exhibits rapid and potent binding as well as high selectivity to FGFR.

A phase II clinical trial (Study 201) of E7090 is underway in Japan and China to evaluate efficacy and safety in patients with cholangiocarcinoma with FGFR2 gene fusion. A phase I clinical trial of E7090 is also underway in Japan in patients with estrogen receptor-positive and HER2-negative breast cancer.

The five-year survival rate for biliary tract cancer is approximately 20%, which makes it an intractable cancer with the second worst prognosis following pancreatic cancer. Drug therapy options are limited in comparison with other cancers, and as such it is a disease with significant unmet medical needs. The estimated number of patients with biliary tract cancer is approximately 32,000 in Japan. FGFR2 gene fusion is observed in approximately 14% of intrahepatic cholangiocarcinoma, which account for 15-30% of biliary tract cancers