On June 20, 2018 Taiho Oncology, Inc. reported a Phase I study to examine the efficacy of TAS-120, an investigational irreversible pan-fibroblast growth factor receptor (FGFR) inhibitor as a potential treatment for patients with advanced solid tumors, including cholangiocarcinoma (CCA), who were previously treated with chemotherapy or other therapies including other FGFR inhibitors (Press release, Taiho, JUN 20, 2018, View Source [SID1234527409]). This is a presentation of updated clinical data for TAS-120 in cholangiocarcinoma, a rare cancer with limited treatment options. These data were presented as oral and poster presentations on Wednesday, June 20 at 2:50 PM CEST at the ESMO (Free ESMO Whitepaper) 20th World Congress on Gastrointestinal Cancer 2018 (ESMO-GI) in Barcelona, Spain, June 20 to 23.

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"We’re very pleased to see that TAS-120 has shown great promise among CCA patients in this study," said Robert Winkler, MD, senior vice president and head of Clinical Development, Taiho Oncology, Inc. "With such a broad range of tumor types with high unmet medical need, Taiho Oncology has remained committed to pioneering innovative research in specific areas that we hope will ultimately lead to an expanded range of potential therapies."

In this study of 45 patients with CCA harboring FGF/FGFR aberrations (e.g., FGFR2 gene fusions, FGF/FGFR mutations, amplifications and re-arrangements), TAS-120 demonstrated clinical activity in patients with CCA with FGFR2 gene fusions, and showed efficacy in patients who progressed on prior FGFR inhibitors. In 28 patients with FGFR2 gene fusions, 71 percent experienced tumor shrinkage and seven achieved confirmed partial response (cPR). The objective response rate in these patients was 25 percent, and 15 patients (54%) experienced stable disease as their best response, with seven still on treatment. The overall disease control rate was 79 percent. In 17 patients with other FGF/FGFR aberrations who received TAS-120, 18 percent achieved cPR. In 13 patients who had received prior FGFR inhibitors, 31 percent achieved cPR.

"These results are encouraging for this rare and difficult-to-treat cancer," said Milind Javle, MD, professor, Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, and investigator on the Phase I TAS-120 study. "Given the limited number of patients diagnosed each year with cholangiocarcinoma in the U.S., we recognize the sense of urgency for and importance of ongoing research to advance potential new therapies."

The most common treatment-related adverse events (AEs) of all grades in all patients were hyperphosphatemia (78%), increased aspartate aminotransferase (29%), dry skin (29%), diarrhea (27%) and dry mouth (27%). Grade ≥3 treatment-related AEs were reported in 51 percent of patients (51%); the most common was hyperphosphatemia in 22 percent of patients.

A Phase II study of TAS-120 in CCA patients has been initiated. The abstract for this presentation is available on the ESMO (Free ESMO Whitepaper)-GI website at View Source

In May 2018, the U.S. Food and Drug Administration Office of Orphan Drug Development granted Taiho’s investigational TAS-120 orphan drug status for the treatment of cholangiocarcinoma.

About FDA Orphan Drug Designation
The mission of the FDA Office of Orphan Products Development (OOPD) is to advance the valuation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or preventive of rare diseases/disorders that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. The program has successfully enabled the development and marketing of more than 350 drugs and biologic products for rare diseases since 1983. In contrast, the decade prior to 1983 saw fewer than 10 such products come to market.

About Cholangiocarcinoma
Cholangiocarcinoma (CCA), also known as bile duct cancer, is not common. About 8,000 people in the United States are diagnosed with CCA each year. This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) cancers. CCA can occur at younger ages, but it is seen mainly in older people. The average age of people in the United States diagnosed with cancer of the intrahepatic bile ducts is 70, and for cancer of the extrahepatic bile ducts it is 72. The chances of survival for patients with CCA depend to a large extent on its location and how advanced it is when it is found.1

The main treatment for CCA is surgery. Radiation therapy and chemotherapy may be used if the cancer cannot be entirely removed with surgery and in cases where the edges of the tissues removed at the operation show cancer cells (also called a positive margin). Both stage III and stage IV cancers cannot be completely removed surgically. Currently, standard treatment options are limited to radiation, palliative therapy, liver transplantation, surgery, chemotherapy and interventional radiology.2