Major pharmaceutical companies presented the data of gene therapies at ASH 2019 conference

Novartis presented the data of Kymriah in patients suffering from DLBCL and ALL. Janssen presented the data of JNJ-4528 in patients suffering from multiple myeloma. Kite has shown the data of Yescarta in relapsed or refractory large B-cell lymphoma. BMS presented results of bb21217 in relapsed/refractory multiple myeloma.

Novartis presented the data of Kymriah (tisagenlecleucel) approved for diffuse large B cell lymphoma (DLBCL) and B-cell acute lymphoblastic leukemia (ALL). Kymriah efficacy in patients suffering from DLBCL was the 24-month analysis of JULIET in adults. In the 24-month study, 52% is the overall response rate (ORR) and 38% complete response (CR). In patients suffering from ALL, the real-world setting had more favorable outcomes than ELIANA pivotal trial. CR was 85% compared to 82% in the ELIANA trial.

Janssen Pharmaceuticals presented the Phase 1b/2 CARTITUDE-1 study of JNJ-4528. JNJ-4528 is an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) indicated for the treatment of relapsed or refractory multiple myeloma. Twenty-nine patients included in the trial, 69% achieved an overall response rate (ORR), 66% achieved stringent CR. 86% achieved a very good partial response (VGPR) or better. Cytokine release syndrome, neutropenia, anemia and thrombocytopenia were the common adverse events reported in the trial.

Kite and CIBMTR presented the real-world use results of Yescarta (axicabtagene ciloleucel) in patients suffering from relapsed or refractory large B-cell lymphoma. The company post-marketing study showed comparable safety and efficacy to ZUMA-1 pivotal trial. The study included 533 patients. 84% of patients achieved an overall response rate (ORR), and 66% of patients achieved a complete response.

Bluebird bio, Inc and Bristol-Myers Squibb presented the data of bb21217 in the patients suffering from relapsed/refractory multiple myeloma. bb21217 is a BCMA-targeted chimeric antigen receptor (CAR) T cell therapy. “Early data from the CRB-402 study in heavily pre-treated patients (median of six prior lines) with relapsed/refractory multiple myeloma demonstrate the potential for durable responses following bb21217 CAR T cell treatment, with a median duration of response of 11.1 months at the 150 x 106 CAR+ T cell dose level,” said David Davidson, M.D., chief medical officer, bluebird bio.


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