Car-t cell therapy for leukemia! Mb-102 (CD123 car T) was approved by FDA to carry out phase I / II clinical trials

August 8, 2019 news / Biovalley bio / -- Mustang bio is a clinical stage biopharmaceutical company, focusing on the development of cell and gene therapy for hematological cancer, solid tumors and rare genetic diseases Recently, the company announced that the US Food and Drug Administration (FDA) has approved the new drug clinical trial application (ind) of mb-102 (CD123 car T), launched a multicenter phase I / II clinical trial to evaluate the efficacy of mb-102 in the treatment of acute myeloid leukemia (AML), maternal plasma cell like dendritic cell tumor (bpdcn) and high-risk myelodysplastic syndrome (MDS) Previously, FDA has granted mb-102 orphan drug qualification (odd) to treat AML and bpdcn Mb-102 (CD123 car T) is a car T-cell therapy, which is used to engineer T cells of patients to recognize and eliminate tumor cells expressing CD123 CD123 is widely expressed in myeloid cells of patients with myelodysplastic syndromes and hematological malignancies, including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL), hairy cell leukemia (HCl), bpdcn, chronic myeloid leukemia (CML) and Hodgkin's lymphoma (HL) The structure of mb-102 CD123 is expressed in 75-89% of AML and more than 90% of bpdcn patients, so it is a very attractive target for T cell adoptive immunotherapy In a phase I clinical trial conducted by researchers in the city of hope in the United States, mb-102 showed a promising response rate in early small groups of these patients According to data from the American Cancer Society (ACS), 19520 new AML cases were estimated in the United States in 2018, with a five-year survival rate of 25% In 2016, the American Society of Hematology (ash) education program reported about 700 new bpdcn cases annually in the United States and 1000 in Europe, with an average survival of 12-14 months Mustang bio's pipeline asset is the first human clinical trial conducted in the city of hope according to the report of the annual meeting of the American Society of Hematology (ash) in December 2017 and the special meeting of the American Association for cancer research (AACR) in December 2018 In (nct02159495), mb-102 showed complete remission and no dose limiting toxicity in the treatment of AML and bpdcn at low dose At present, the dose increase of two indications in this study is in progress Manuel litchman, MD, President and CEO of Mustang bio, said: "FDA approved our ind application for mb-102, which is an important milestone for the company, because it is our first ind We are pleased to launch our multicenter phase I / II clinical trial later in 2019 and process patient cells at our manufacturing facility, which opened in June 2018 We are keen to meet the needs of AML, bpdcn and MDS patients, and look forward to further development of mb-102 to help solve these devastating diseases " Original source: FDA clear ind for Mustang bio's mb-102 (CD123 car T)