Cholangiocarcinoma Treatment Granted Orphan Drug Designation

TransThera Biosciences received Orphan Drug Designation from the US Food and Drug Administration (FDA) for TT-00420, a clinical stage investigational drug, for the treatment of cholangiocarcinoma.

"Cholangiocarcinoma lacks effective therapies and remains a huge unmet medical need around the world. TT-00420 has demonstrated great potential in multiple experiments,” Dr. Frank Wu, Founder and CEO of TransThera, said. “We believe that today's orphan drug designation will help accelerate the development of this potential product in the clinical trials and bring meaningful benefit to cholangiocarcinoma patients. We are very excited about this news, which expands the horizon of our lead product in development. In addition to triple negative breast cancer, cholangiocarcinoma is identified by our scientists to be second indication for TT-00420."

TT-00420 is in global Phase I trial both in the US and China. Dose expansion Phase Ib/II trials in cholangiocarcinoma and triple negative breast cancer are planned to start in 2020.

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TT-00420 is a novel, small molecule, spectrum-selective multiple kinase inhibitor, developed by TransThera Biosciences. A global, phase I, first-in-human trial is currently ongoing to evaluate the safety and tolerability of TT-00420 in patients with advanced solid tumors.

Cholangiocarcinoma is a rare cancer that forms in the bile ducts with poor prognosis. Common treatment options for cholangiocarcinoma include surgery and chemotherapy, but recurrence is common and disease-free survival time is low. Thus, clinical trials are necessary to develop and evaluate effective therapies for treatment of this disease.

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