Highlightll Pharmaceutical today announced that its oral highly selective JAK1/TYK2 dual inhibitor girocitinib (TLL-018) has been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for the treatment of rheumatoid arthritis (RA).
This determination is based on the positive topline data from the Girocitinib Phase III Registration Study (TARA). After review, the CDE determined that girocitinib meets the criteria for breakthrough therapy for the prevention and treatment of diseases that seriously threaten life or seriously affect quality of life, and that compared to existing treatments, it has more significant therapeutic effects.
The TARA study is the world's first phase III RA clinical trial using the marketed JAK inhibitor Xeljanz® (tofacitinib) as a positive control with a superiority primary endpoint. Data showed that girocitinib was significantly superior to tofacitinib (p<0.0001) in both primary and all secondary efficacy endpoints: the 24-week ACR50 response rate; ACR20/ACR70 response rate, DAS28 disease activity score, and CDAI low disease activity. In particular，patients in the tofacitinib group who did not reach ACR50 showed significant efficacy after switching to girocitinib, demonstrating potential in treating the “difficult-to-treat” RA patient (who failed at least one biologic and a JAK inhibitor), a significant unmet medical need. In terms of safety, girocitinib was generally well tolerated, with no new safety signals observed.
Professor Zeng Xiaofeng, the principal investigator of the study at the Peking Union Medical College Hospital, stated: "Girocitinib receiving breakthrough therapy designation reflects regulatory recognition of its significant clinical value. Currently, there is still an unmet need for RA treatment, especially for difficult-to-treat patients. With solid Phase III evidence, girocitinib is expected to offer patients a new option that is both effective and safe.”
Breakthrough therapy designation provides an accelerated review channel established by the NMPA for drugs with significant clinical advantages over existing therapies.
About Highlightll
Highlightll is a clinical stage biopharmaceutical company focusing on both peripheral and central autoimmune/inflammatory diseases. We strive to discover breakthrough medicines which would have a high impact on health care. We leverage resources and expertise worldwide and combine them with our unique perspectives to solve challenging medical problems. Our lead programs are the world's first highly potent and selective dual JAK1/TYK2 inhibitors. Girocitinib (TLL-018), a peripheral JAK1/TYK2 inhibitor, met the primary endpoints in its Phase III trial for chronic spontaneous urticaria (CSU) and is also in Phase III trial for rheumatoid arthritis (RA), while TLL-041, the world first brain-penetrant JAK1/TYK2 inhibitor was licensed to Biohaven in 2023. Biohaven advanced the BHV-8000/TLL-041 program to a global Phase II/III pivotal trial in Parkinson’s disease. NLRP3 is our second platform where we are developing HL-400 for CNS/neurodegenerative diseases and HL-450 for peripheral diseases.
