Mustang Bio, Inc. was granted Orphan Drug Designation by U.S. FDA for MB-102 (CD123 CAR T) for the treatment of acute myeloid leukemia (AML). The FDA also previously granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).
 
MB-102 is currently being studied in a 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. 
 
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are pleased that MB-102 has received Orphan Drug Designation in two indications, AML and BPDCN. AML most commonly occurs in senior adults, many of whom have to forgo chemotherapy due to other health conditions. MB-102 has the potential to be an important new treatment for these and other patients, and to potentially address multiple areas of high unmet medical need. We expect to initiate a multicenter Phase 1/2 clinical trial in patients with AML, BPDCN and high-risk myelodysplastic syndrome in the coming months.”