Penpulimab (PD-1) Obtained Breakthrough Therapy Designation from FDA for Third-Line Treatment of Metastatic Nasopharyngeal Carcinoma

On March 30, 2021, Akeso, Inc. (9926.HK) announced that Penpulimab(AK105), an PD-1 monoclonal antibody drug co-developed by Akeso with Sino Biopharmaceutical Limited (1177.HK), has obtained breakthrough therapy designation from the Food and Drug Administration of the United States (FDA) for third-line treatment of patients with metastatic nasopharyngeal carcinoma. This is another significant progress Penpulimab made in the United States after receiving the fast track designation and orphan drug designation from the FDA for the thirdline treatment of metastatic nasopharyngeal carcinoma. This is also Akeso's second oncology drug to receive breakthrough therapy designation.


Breakthrough therapy designation aims to accelerate new drug development. These drugs are designed to treat severe diseases and have shown encouraging results in early clinical studies. This shows that these drugs have a significant improvement in clinical endpoints over existing therapies or when there are unmet medical needs. Drugs with breakthrough therapy designation will receive closer guidance and various forms of support from senior FDA officials during the development process to ensure that patients will be provided with new treatment options in the shortest time.


The success in obtaining breakthrough therapy designation for Penpulimab from the FDA in respect of the third-line treatment of metastatic nasopharyngeal carcinoma will significantly support and accelerate the commercialization plan of Penpulimab in the United States. Akeso and Sino Biopharm will work closely with the FDA to swiftly bring Penpulimab to patients with nasopharyngeal carcinoma. Currently, Penpulimab applied to major indications such as liver cancer, gastric cancer, lung cancer, Hodgkin's lymphoma, nasopharyngeal cancer, etc.




Penpulimab (AK105, PD-1 monoclonal antibody) is jointly developed and commercialized by a joint venture established by the Company and Chia Tai-Tianqing Pharmaceutical Group Co., Ltd. ("Chia Tai-Tianqing"), a subsidiary of Sino Biopharm. Penpulimab's fragment crystallisable ("Fc") receptor and complement mediated effector are completely removed by mutations of Fc region, it also has a slower antigen binding offrate compared with the PD-1 antibodies that are already launched in foreign market. These features have made Penpulimab more effective in blocking the activity of the PD-1 pathway and evaded the T-cell anti-tumor activity, thus it has the potential to become an anti-PD-1 drug that can achieve better clinical efficacy.


In May 2020, the National Medical Products Administration (NMPA) of the People's Republic of China ("PRC") has accepted the new drug application of Penpulimab monoclonal antibody for the treatment of patients with classical Hodgkin's lymphoma (cHL) that has relapsed or refractory (r/r) after two or more lines of systemic chemotherapy (r/r cHL). The registrational clinical trial for third-line metastatic nasopharyngeal cancer with Penpulimab has also met key endpoints.



The Company is a biopharmaceutical company dedicated to the research, development, manufacturing and commercialization of new innovative antibody drugs that are affordable to patients worldwide. Since the Company's establishment, the Company has established an end-to-end comprehensive drug development platform (ACE Platform) and system, encompassing fully integrated drug discovery and development functions, including target validation, antibody drug discovery and development, CMC production process development, and GMP compliant scale production. The Company has also successfully developed a bi-specific antibody drug development technology (Tetrabody technology). The Company currently has a pipeline of over 20 innovative drugs for the treatment of major diseases like tumors, autoimmune diseases, inflammation and metabolism diseases, 13 of which have entered clinical stage, including two first-in-class bi-specific antibody drugs (PD-1/CTLA-4 and PD-1/VEGF). The Company's vision is to become a global leading biopharmaceutical company through research and development ("R&D") of high efficacy and breakthrough new drugs that are first-in-class and best-in-class therapies.