Myeloma: CT053 fully human anti-BCMA autologous CAR T cell
Myeloma: CT053 fully human anti-BCMA autologous CAR T cell. Carsgen Bio-CT053 fully human anti-BCMA autologous CAR T cell injection is included in the breakthrough therapy drug variety for the treatment of relapsed and refractory multiple myeloma.
Shanghai, China, December 8, 2020-CARsgen Therapeutics Inc. (CARsgen Therapeutics Inc., hereinafter referred to as “Carsgen Bio”) today announced that the company’s CT053 fully human anti-BCMA autologous CAR T cell injection passed During the publication period of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA), breakthrough treatment drugs were included, and the proposed indication is relapsed and refractory multiple myeloma (R/R MM).
Dr. Li Zonghai, Chairman, CEO and Chief Scientist of Keji Biotech, said: “This CT053 whole-human anti-BCMA autologous CAR T cell clinical trial is included in the CDE breakthrough therapeutic drug program, which is expected to accelerate the development and development of this drug in China. The speed of approval will benefit Chinese patients earlier.”
It is worth mentioning that CT053 is the first cell therapy candidate in China to receive the U.S. Food and Drug Administration’s (FDA) Regenerative Medicine Advanced Therapy (RMAT) and was included in the European Medicines Agency’s Priority Medicine (PRIME) program. Therefore, CT053 has become China’s first cell therapy candidate that integrates CDE breakthrough therapy, RMAT and PRIME qualifications.
1 About CT053
Multiple myeloma is a malignant disease with abnormal proliferation of clonal plasma cells. Relapsed/refractory multiple myeloma (R/R MM) has significant unmet medical needs. T cells genetically modified by chimeric antigen receptor (CAR) targeted by B cell maturation antigen (BCMA) are considered to be a promising new method for curing multiple myeloma. CT053 is a fully human BCMA-targeted CAR T cell product independently developed by Keji Bio to treat patients with R/R MM. It uses a fully human anti-BCMA scFv (single chain antibody) domain, which is speculated to reduce immunogen And improve safety. Currently, CT053 is conducting clinical trials in China, the United States, Canada and other countries and regions.
2 About Breakthrough Therapeutic Drug Program
During the drug clinical trial, it is used to prevent and treat diseases that are severely life-threatening or seriously affect the quality of life, and there is no effective prevention and treatment method or compared with existing treatment methods, there is sufficient evidence that innovative drugs or improved new drugs have obvious clinical advantages. Applicants can apply for the breakthrough drug program. For drugs that are included in the breakthrough therapeutic drug program, there will be more opportunities to communicate with the drug regulatory agency during the drug review and approval process, thereby speeding up the drug’s marketing process.
3 About RMAT
RMAT (Regenerative Medical Advanced Therapy) is a fast-track system developed to accelerate the development and approval of innovative regenerative therapies when the United States revised the regenerative medical provisions of the “21st Century Cures Act” in December 2016. RMAT can be cell therapy, therapeutic tissue engineering products, human cells and tissue products, or other combination therapies that include regenerative medicine technology products. To obtain RMAT qualification for a drug under research, there must be preliminary clinical research data to prove that the drug has a positive result in treating, delaying, reversing or curing serious or life-threatening diseases or unmet medical needs. Obtaining RMAT qualifications can simultaneously enjoy all the preferential policies of FDA’s Breakthrough Therapy designation and Fast Track qualifications, thereby obtaining FDA’s intensive guidance on effective drug development, including the ability to interact with senior FDA management personnel early Discuss alternative or intermediate endpoints, support potential methods for accelerating approval and meeting post-approval requirements, priority review of potential biological product licensing applications (BLA), and other opportunities for accelerated development and review.
4 About PRIME
In March 2016, the European Medicines Agency (EMA) launched the Priority Medicines (PRIME) program, which aims to accelerate the review process of key drugs in the field of medical shortages and benefit patients as soon as possible. PRIME refers to drugs that can provide more obvious therapeutic advantages than existing drugs for some diseases for which medical needs are not met, or can provide potential clinical benefits for patients without drugs. The program also provides a channel for drug developers to have early dialogue and enhanced interaction with EMA, thereby accelerating drug development.
About Keji Bio
Keji Biopharmaceutical (Shanghai) Co., Ltd. is a biomedical company dedicated to the development of innovative CAR T cells and other tumor immunotherapy drugs with the mission of providing treatment and even cure for tumor patients. The company has a number of CAR T cell drug candidates, covering hematological tumors and solid tumors, and is conducting clinical trials in China, the United States, Canada and other countries and regions.