TransThera Receives Fast Track Designation from FDA for its Core Product TT-00420 to Treat Cholangiocarcinoma

TransThera Sciences (Nanjing) Inc., a clinical-stage biopharmaceutical company focusing on discovering and developing innovative small molecule drug therapies for oncology, inflammatory and cardiovascular diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its phase II stage product TT-00420 for the treatment of patients with Cholangiocarcinoma (CCA) who have no standard treatment options.

TT-00420 is a spectrum selective kinase inhibitor that is found to show high potency to a variety of clinically identified FGFR2 mutations in preclinical experiments. The unique combination of TT-00420's target profile and mechanism of action also allow it to effectively treat patients with heterogeneous tumors, such as CCA patients without clear biomarkers.

The grant of Fast Track Designation is primarily based upon the clinical results of TT-00420 in its completed phase I study, in which nine CCA patients were enrolled and treated with TT-00420 at different dose levels. Seven patients had at least one post treatment efficacy assessment. Two achieved PR and five achieved SD. Among these evaluable patients, (i) Five harbored FGFR2 fusion or rearrangement and developed acquired resistance to prior treatment of FGFR inhibitors. One patient achieved PR with PFS about ten months. The other four patients achieved SD. (ii) One patient with primary resistance achieved PR with PFS about eight months. (iii) One patient without FGFR alterations achieved SD with shrinkage of target lesion. The clinical evidence supports the continuing exploration of TT-00420 in treating CCA patients with no standard treatment options.

"Receiving Fast Track Designation is an important milestone for the development of TT-00420." commented excitedly by Dr. Frank Wu, CEO of TransThera, "We have been and will continue to actively work with FDA, expediting the clinical development of TT-00420 in CCA field."

In November 2019, TT-00420 was already granted Orphan Drug Designation by FDA to treat Cholangiocarcinoma.

About TT-00420

TT-00420 is a highly innovative clinical-stage spectrum-selective kinase inhibitor that exerts antitumor effects by targeting tumor cells and improving the tumor microenvironment. A large number of preclinical studies have found that TT-00420 has promising inhibitory effect on triple-negative breast cancer, cholangiocarcinoma and other malignant tumors. In September 2018, TT-00420 was approved by the US FDA for the first human clinical trial; in February 2019, it was approved by China's NMPA for human clinical trials; and in November of the same year, TT-00420 was granted the "Orphan Drug Designation" status (ODD) by FDA for the treatment of cholangiocarcinoma. In November 2020, it was again approved by the US FDA targeting the clinical trial of new indications for cholangiocarcinoma.

About TransThera Sciences (Nanjing), Inc.

TransThera Sciences (Nanjing), Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics to target diseases with major unmet medical needs via the internal research platform and open innovation. TransThera's current portfolio covers therapeutic areas such as oncology, cardiovascular, and inflammatory diseases. For more information, please visit www.transtherabio.com.

Source: TransThera Sciences (Nanjing) Inc.