NANJING, China and GAITHERSBURG, Md., Aug. 7, 2026 /PRNewswire/ -- TransThera Sciences' Yochanra^® is the first drug independently developed in China to address the longstanding global challenge of acquired resistance to FGFR inhibitor therapy in patients with cholangiocarcinoma. On 6 August 2026, the National Medical Products Administration (the "NMPA") formally approved the drug for marketing. As a global frontrunner in this field, the product had previously been granted Fast Track Designation and Orphan Drug Designation by the U.S. Food and Drug Administration (the "FDA"), Orphan Drug Designation for the treatment of biliary tract cancer by the European Medicines Agency (the "EMA"), and inclusion in the List of Products for Priority Review and the List of Breakthrough Therapeutic Drugs by the NMPA in China. The product has also been featured and presented orally on multiple occasions at leading international scientific conferences, filling the clinical gap arising from the absence of a standard treatment following the development of resistance to FGFR inhibitor therapy.

The indication approved by the NMPA is the first indication under the multi-indication development strategy for Yochanra^®. It is indicated for adult patients with advanced, metastatic or unresectable cholangiocarcinoma harboring an FGFR2 fusion or rearrangement who have previously received systemic therapy and FGFR inhibitor therapy. The approval of this indication was based on a multicenter, open-label, single-arm, pivotal Phase II clinical trial conducted in China. The results of the study were presented at the 2026 Annual Meeting of the American Society of Clinical Oncology (ASCO). As of 27 December 2025, with a median follow-up of 12 months, all 50 patients with advanced cholangiocarcinoma enrolled in the study had previously received at least one line of chemotherapy and treatment with one FGFR inhibitor. Assessments by blinded independent central review (BICR) showed that:

• The objective response rate (ORR) was 28.0%, with 14 patients achieving a confirmed partial response. The median duration of response (DoR) was 8.5 months (95% CI: 5.6~12.5 months), and the disease control rate (DCR) was 82.0%.

• The median progression-free survival (PFS) was 6.0 months (95% CI: 4.4~8.3 months).

• The median overall survival (OS) was 20.7 months (95% CI: 11.8 months ~ not estimable).

In the trial, Yochanra^® demonstrated durable antitumor activity and a manageable safety and tolerability profile.

The global market for cholangiocarcinoma therapeutics reached US$2.0 billion in 2024 and is projected to grow to US$3.2 billion by 2027, with approximately 25.2% of patients with cholangiocarcinoma harboring FGFR alterations. According to a research paper published in "Cancer Treatment Reviews" in 2023, patients with cholangiocarcinoma develop acquired resistance following FGFR inhibitor treatment. Patients who develop resistance typically harbor at least one, and often multiple, mutations, creating extremely high barriers to drug development in this field. Several overseas biotech companies have initiated related research programs, but such programs ultimately ended in failure. Neither Chinese nor U.S. treatment guidelines provide any high-level recommendation for the treatment of patients with cholangiocarcinoma who have developed resistance, leaving such patients without an established standard treatment option. Through its unique FGFR-binding mode, Yochanra^® can overcome resistance mutations at multiple sites while maintaining high binding activity. Clinical trials have demonstrated that it can effectively overcome acquired resistance, bringing new hope to patients with FGFR2 fusion- or rearrangement-positive cholangiocarcinoma who have developed resistance to prior FGFR inhibitor therapy.

This marketing approval marks the approval in China of the first indication for Yochanra^®. Meanwhile, for cholangiocarcinoma, enrollment has been completed in the global multicenter registrational Phase III study of Yochanra^®, while the Phase III confirmatory study in China is progressing in parallel. For other indications, the Company will continue to advance clinical trials at various stages, with a view to providing potentially better clinical treatment options for more patients, including, but not limited to, patients with prostate cancer, breast cancer and liver cancer.

ABOUT YOCHANRA^®

Yochanra^® (Tinengotinib) is an innovative multi-target small-molecule kinase inhibitor independently developed by the Company targeting FGFR/VEGFR, JAK and Aurora kinases. It exerts antitumor effects through three mechanisms: precision targeting, lineage remodeling and immune activation. Yochanra^® is currently being evaluated in multiple clinical trials conducted globally for solid tumors including cholangiocarcinoma, prostate cancer, breast cancer and liver cancer. It has been granted Orphan Drug Designation and Fast Track Designation by the U.S. FDA for the treatment of cholangiocarcinoma and Orphan Drug Designation by the EMA for the treatment of biliary tract cancer.

ABOUT TRANSTHERA

TransThera is a biopharmaceutical company that focuses on clinical needs. It is dedicated to discovering and developing small molecule innovative therapies for tumors, inflammation, and cardiometabolic diseases. Further aided by in-depth study of translational medicine and drug design, TransThera aims to develop first-in-class or best-in-class drug candidates strategically positioned to meet urgent clinical needs on a global scale.