TransThera Announced Global Phase 3 Clinical Trial for Cholangiocarcinoma Authorized in the European Union and Orphan Drug Designation for Tinengotinib to Treat Biliary Tract Cancer Granted by European Medicines Agency

NANJING, China and GAITHURSBURG, Md., March 8, 2024 /PRNewswire/ -- TransThera, a clinical-stage biopharmaceutical company dedicated to innovating differentiated drugs globally, today announced that the randomized, controlled, global multicenter Phase 3 trial (FIRST-308) of tinengotinib versus physician's choice to evaluate the efficacy and safety in subjects with FGFR-altered, chemotherapy- and FGFR Inhibitor-refractory/relapsed cholangiocarcinoma (CCA), has been authorized by regulatory agencies in the European Union (EU) after the authorizations from US, South Korea and Taiwan region. Furthermore, European Medicines Agency (EMA)  granted the Orphan Drug Designation(ODD) for tinengotinib for the treatment of biliary tract cancer (BTC) .

Tinengotinib, a next-generation FGFR inhibitor with high potency against a variety of FGFR2 kinase domain mutations, has shown promising clinical benefit in subjects with FGFR-altered metastatic CCA who were heavily pretreated with chemotherapy and refractory/relapsed to FGFRi(s). The results of tinengotinib in CCA from the phase I/II clinical trials were presented orally at 2023 ESMO and 2024 ASCO GI conferences. The FRIST-308 clinical trial is enrolling to further confirm the efficacy and safety of tinengotinib in CCA. In December 2023, first patient was dosed in the United Sates (US). 

"We are very delighted to have achieved the significant regulatory progresses for tinengotinib in global development at various countries and regions. These milestones are not only the recognition by global regulatory authorities of the potential clinical benefit of tinengotinib to treat CCA patients, but also a reflection of the company's international regulatory capabilities and unwavering commitment to bringing innovative therapies to global patients," said Jean Fan, M.D., Chief Medical Officer of TransThera Sciences. "Leveraging the regulatory agency's support from the EU in addition to the US and other countries and regions, we will continue to expedite the global clinical development and commercialization of tinengotinib. We are hopeful that these efforts will enable us to bring this novel drug to patients around the world as quickly as we can."

About ODD in EU

The term "orphan medicines" refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions affecting less than 5 in 10,000 people in the EU. The EMA offers a range of incentives to encourage the development of designated orphan medicines. This Orphan Drug Designation for tinengotinib qualifies it for great regulatory supports in the subsequent clinical development and commercialization in the EU, including protocol assistance, fee reductions, and most importantly, 10 years of market exclusivity upon approval.

About tinengotinib

Tinengotinib is an innovative, global phase III stage spectrum-selective kinase inhibitor that exerts antitumor effects by targeting tumor cells proliferation, angiogenesis and immune-oncology pathways by inhibiting the cytokine signaling and angiogenesis (FGFRs and VEGFRs), mitotic kinases Aurora A/B and Janus kinases (JAK). Ongoing clinical trials in the US and China have revealed the potential of tinengotinib to be efficacious in various solid tumors. It was granted the Orphan Drug Designation and Fast Track Designation by the FDA for the treatment of CCA. In July 2023, tinengotinib was granted the Breakthrough Therapy Designation (BTD) by NMPA in China. In March 2024, tinengotinib was granted the Orphan Drug Designation(ODD) for the treatment of biliary tract cancer by EMA.

About TransThera 

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, inflammatory, and cardiovascular diseases.

For more information, please visit www.transthera.com

Forward-looking statements

This news contains forward-looking statements that involve risks, uncertainties, and assumptions. If risks or uncertainties ever materialize or the assumptions prove incorrect, our actual results may differ from those expressed or implied by such forward-looking statements. All statements other than statements of historical facts could be deemed forward-looking, including, but not limited to, any statement of the plans, strategies, and objectives of management for future operations, including but not limited to our clinical development and commercialization plans; any projections of financial information; any statements about historical results that may suggest trends for our business; any statements of expectation or belief regarding future events, potential markets or market size, technology developments, our product pipeline, clinical data, results or practices or the implications thereof, enforceability of our intellectual property rights, competitive strengths or our position within the industry; and any statements of assumptions underlying any of the items mentioned.

SOURCE TransThera Sciences (Nanjing) Inc.