FDA Clears Mustang Bio’s IND for MB-102 (CD123-CAR T)

Aug 5, 2019 | AML, CAR-T, IND

FDA Clears Mustang Bio’s IND for MB-102 (CD123-CAR T)

Mustang Bio, Inc (MBIO) reports that the U.S. Food and Drug Administration (FDA) has approved the Company’s investigational new dug application (IND) in acute myeloid leukemia (AML) blastic plasmacytoid dendritic cell neoplasm (BPDCN) and high-risk myelodysplastic syndrome (MDS).

The Problem

The American Cancer Society reports there were an estimated 19,520 new U.S. cases of AML in 2018 and the disease has an estimated five year survival rate of 25%. In 2016, an American Society of Hematology Education Program article reported that there were about 700 new BPCN cases in the U.S. and 1,000 in Europe per year, with median survival of 12 to 14 months.

The Drug

MB-102 is a CAR T cell therapy that is produced by engineering T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with myelodysplastic syndrome and hematologic malignancies, including 75%-89% of AML patients and over 90% in BPDCN patients. MB-102 has shown promising response rates in early small populations of these patients in an investigator-sponsored Phase I clinical trial being conducted by City of Hope, where the CAR T cell therapy was also developed. Clinical trials will start later in 2019.

FDA Recognition

The FDA granted Orphan Drug Designation to MB-102 for the treatment of AL on July, 24 2019. It previously granted the same status for the treatment of BPDCN.

MB-102 (CD123 CAR T)

MB-102 (CD123 CAR T) is a CAR T cell therapy that is produced by engineering patient T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with myelodysplastic syndromes, as well as hematologic malignancies, including AML, B-cell acute lymphoblastic leukemia, hairy cell leukemia, BPDCN, chronic myeloid leukemia and Hodgkin’s lymphoma.

First-in-Human Study

MB-102 was utilized in a first-in-human clinical trial at City of Hope. It has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported by the American Society of Hematology (ASH Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) Special Conference on Tumor Immunology and Immunotherapy in November 2018.  Dose escalation at City of Hope in both indications MB-102 has received Orphan Drug Designation from the U.S. FDA for AML and BPDCN.

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