Domestic anti BCMA car-t cell therapy! Keji biological ct053 has obtained the qualification of American FDA orphan drug to treat multiple myeloma!

August 31, 2019 / BIOON / -- carsgen therapeutics (Shanghai) Co., Ltd (hereinafter referred to as "carsgen therapeutics") is a clinical stage immunooncology company, which is committed to the development and commercialization of car-t cell immunotherapy for insufficient medical needs The company has cooperated with China's top hospitals to carry out a number of first-in-human studies, such as anti GPC3 car-t therapy for HCC and squamous lung cancer, anti EGFR / egfrv3 car-t therapy for glioblastoma multiforme, anti caludin 18.2 car-t therapy for gastric cancer and pancreatic adenocarcinoma Recently, the company announced that the U.S Food and Drug Administration (FDA) has granted its orphan drug design (odd) qualification for anti BCMA car-t cell therapy ct053 in the treatment of multiple myeloma (BCMA) Orphan drugs are drugs used to prevent, treat and diagnose rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases in less than 200000 people After the orphan drug qualification (odd) is recognized, pharmaceutical companies are entitled to various incentives for research and development of rare disease drugs, including tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a 7-year market monopoly period after the drug is approved for marketing "FDA's orphan drug qualification is an important regulatory milestone in the ongoing development and commercialization of anti BCMA car-t cell therapy ct053," said Dr Li Zonghai, founder, CEO and chief scientific officer of Keji biology Ct053 has shown excellent efficacy in an exploratory phase I clinical study in China Among 24 patients with relapsed refractory multiple myeloma (R / RMM), 19 showed complete remission, with a complete remission rate of 79.2% Importantly, no class 3 or higher CRS events were observed The results of this study will be presented orally at the International Symposium on multiple myeloma (imw2019) in Boston on September 14 Multiple myeloma (mm) is the second most common hematological malignancy after non Hodgkin's lymphoma In recent years, although great progress has been made in chemotherapy, proteasome inhibitors, immunomodulator thalidomide derivatives and CD38 targeted antibodies, almost all patients will eventually relapse Therefore, there is an urgent need for new treatment Mm market is close to $14 billion in 2017 and is expected to reach nearly $29 billion in 2027 BCMA is one of the most important biomarkers of B cells, which widely exists on the surface of MM cells and has become a very popular target of immunotherapy for mm and other hematological malignancies At present, there are more than 20 immunotherapies developed for BCMA, mainly divided into three categories: chimeric antigen receptor T-cell therapy (car-t, represented by new base / Bluebird, Novartis), bispecific antibody (BsAb, represented by Amgen), antibody drug conjugate (ADC, represented by GSK) Recently, GSK announced the success of dreamm-2 (nct03525678), a key clinical study to evaluate the efficacy of belantamab mafodotin (gsk2857916) in the treatment of recurrent or refractory multiple myeloma (R / RMM), and plans to submit an application for listing at the end of the year Anti BCMA car-t cell therapy ct053 is an innovative drug independently developed by Keji biology It is a T cell modified by the chimeric antigen receptor (car) targeting B cell mature antigen (BCMA) with all human antibodies The main target indication is recurrent / refractory multiple myeloma At present, Keji biology has 11 car-t cell treatment products for a variety of tumor targets, such as GPC3, CD19, BCMA, claudin18.2, EGFR / EGFRvIII and so on At present, it has entered the following five clinical trials On June 19 this year, Keji biology announced that the application for the new drug clinical trial (ind) of ct053 in the treatment of relapsed and refractory multiple myeloma received the notice of FDA approval of the product to enter the clinical trial The clinical trial was approved in the United States, which is the second national or regional clinical license for the product after it was implicitly approved by the National Drug Administration (nmpa) on February 27, 2019 This approval is also the first time for China's original car-t project to independently apply for ind in the United States, and successfully obtained FDA approval On August 29, Keji biology successfully held the inauguration ceremony of the commercial production base of car-t cell therapy products in Jinshan, Shanghai The base is the first commercial production base of car-t cell therapy products in China, and its completion marks Keji biology's solid step on the industrialization road of car-t cell therapy products Original source: carsgen therapeutics receives US FDA orphan drug design for full human anti BCMA (B cell matching antigen) autologous Chinese Anti receiver (car) T cells for the treatment of multiple myeloma