On December 20, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the
development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR T)
technology and gene therapies for rare diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and incurable blood cancer with a median survival of less than 18 months and no standard of care (Press release, Mustang Bio, DEC 20, 2018, View Source [SID1234532176]).
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Martina Sersch, M.D., Ph.D., Chief Medical Officer of Mustang, said, "We are pleased to receive Orphan Drug
Designation for MB-102, which has shown the potential to address an area of high unmet medical need. This significant milestone for Mustang will provide additional market exclusivity and financial benefits to advance MB-102, which we believe is an important new treatment for patients with BPDCN. Based on the Phase 1 data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in November 2018, we expect to initiate a multicenter Phase 1/2 clinical trial in patients with acute myeloid leukemia (AML), BPDCN and high-risk myelodysplastic syndrome in 2019."
MB-102 is currently being studied in a single center, first-in-human Phase 1 dose-escalation clinical trial evaluating the
safety and anti-tumor activity of escalating doses of MB-102 in patients with relapsed or refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a single dose of MB-102 with an option for a second infusion if they continue to meet safety and eligibility criteria and still have CD123+ disease. MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities.
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.
About Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and incurable blood cancer with a median survival of less
than 18 months and no standard of care. High levels of CD123 expression is one of the diagnostic hallmarks of BPDCN, making CD123 an attractive target for T cell-based adoptive immunotherapy.
About 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 in hematologic malignancies including AML, B-cell acute lymphoblastic leukemia, hairy cell leukemia, BPDCN, chronic myeloid leukemia and Hodgkin’s lymphoma.
In the first-in-human clinical trial at City of Hope (NCT02159495), MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in November 2018. Dose escalation continues at City of Hope in both indications.