Leukemia: Acute Lymphoblastic Leukemia (ALL)

    Actionable Target Type Prevalence (%) Clinical Experience with Targeted Agent
    CD19 Precursor B cell 80 Blinatumomab (CD3-CD19 bispecific monoclonal antibody):
    • Phase 2 in pts with R/R B-ALL: CR/CRh rate of 43%, median RFS 5.9 mo (22)
    • Phase 3, blinatumomab vs. chemotherapy in R/R AML: Blinatumomab superior in complete remission rates (34% vs. 16%; p < 0.001) and OS (7.7 vs. 4.0 mo; p = 0.01) (23)
    CD19 Precursor B cell 80 Tisagenlecleucel (murine anti-CD19 CAR T-cell therapy):
    • Phase 2, single-arm ELIANA trial in pts with R/R B-ALL: Overall 75% and 64% at 6 and 12 mo; OS 89% and 79% at 6 and 12 mo (pivotal Phase II global ELIANA trial)
    Other topics in Targets by Organ Site