Mustang Bio has announced City of Hope’s report that their MB-102 (CD123 CAR) CAR T therapy is safe and has achieved the first-ever complete response from a CAR T therapy in blastic plasmacytoid dendritic cell neoplasm (BPDCN), as well as a CR in acute myeloid leukemia (AML), in an ongoing Phase 1 clinical trial. 

Manuel Litchman, M.D., president and chief executive officer of Mustang, said, “According to investigators at City of Hope, these data demonstrate MB-102’s potential to be a safe, well tolerated and effective CAR T therapy that can achieve complete disease response. In addition, MB-102’s promising anti-leukemic activity in both AML and BPDCN supports further evaluation in clinical trials in transplant eligible and ineligible patients. 

This single center, first-in-human Phase 1 dose-escalation clinical trial is evaluating the safety and 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. To date, 14 patients have been enrolled and seven have been treated in this first in-human trial for AML and BPDCN patients using a CD123 CAR T therapy.

In the AML cohort, two patients were treated at dose level 1. Trial investigators reported that one achieved a morphologic leukemic-free state at day 28 post-infusion.