Overall response rate (ORR) of 94% in patients in active dose cohorts (doses above 50 x 106 CAR + T-cells)
Complete Response (CR) rate of 56% observed in patients in active dose cohorts
Median progression free survival (PFS) not reached with median follow up of 40 weeks in active dose cohorts
Nine of 10 (90%) of patients evaluable for minimal residual disease (MRD) status were found to be MRD-negative
The objective of this Phase 1 dose-escalation study is to evaluate safety and efficacy of bb2121 and determine a recommended Phase 2 dose.
"Celgene has a longstanding commitment to patients with multiple myeloma
through our extensive research efforts in this deadly blood cancer,"
"The growing body of bb2121 clinical data are building a compelling
story, further supporting the importance of the therapy's unique
Durable clinical responses in heavily pretreated patients with relapsed/refractory multiple myeloma: Updated results from a multicenter study of bb2121 anti-BCMA CAR T cell therapy (Abstract #740)
Location: Hall C1 (
Session Title: Myeloma: Therapy, excluding Transplantation I
The open-label Phase 1 CRB-401 study (NCT02658929) is evaluating the preliminary safety and efficacy of bb2121 anti-BCMA CAR T cell in patients with relapsed and/or refractory multiple myeloma. The study also evaluated the recommended dose of bb2121 for future studies.
Patients on study were heavily pre-treated, with a median of 7 prior therapies (range: 3 - 14):
As of the
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 anti-BCMA CAR T cells. The CAR T cells were produced from each patient's own blood cells, which were modified using a proprietary lentiviral vector encoding the anti-BCMA CAR.
Results in the active dose cohorts (150 x 106, 450 x 106 and 800 x 106 CAR+ T cells; N=18) were:
In the dose-escalation phase, 15/21 (71%) of patients had cytokine release syndrome (CRS), mostly Grade 1 & 2, with 2 patients experiencing Grade 3 CRS (9%). Four patients received tocilizumab, 1 (Grade 2 CRS) received steroids and in each case the CRS resolved within 24 hours. The most common treatment-emergent Grade 3-4 AEs in 21 infused patients were cytopenias commonly associated with lymphodepleting chemotherapy including neutropenia (86%), anemia (57%) and thrombocytopenia (43%). There were two deaths in the active cohorts at 22 and 69 weeks following infusion, respectively. The first was due to cardiac arrest and the second was due to myelodysplastic syndrome; both subjects were in a myeloma CR at their last study assessment prior to death. Based on the findings during dose escalation, a dose expansion phase of 12 subjects has started testing doses between 150-450 x 106 CAR+ T cells. In the dose expansion phase, one patient treated at the 450 x 106 CAR+ T cells dose experienced Grade 4 neurotoxicity including focal cerebral edema and subarachnoid hemorrhage. This patient had a high tumor burden, and a history of subarachnoid hemorrhage. The event was successfully managed, and the patient remains in the response group. No other Grade 3/4 neurotoxicity was observed in the escalation or expansion cohort.
"To see these types of responses after one treatment with bb2121 in a
heavily pre-treated patient population is very promising, and we are
hopeful that CAR T therapy with bb2121 may become an important therapy
in the fight against multiple myeloma, which remains an insidious and
incurable disease," said
bb2121 is an investigational compound that is not approved for any use
in any country. bb2121 recently received Breakthrough Therapy
Designation from the
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio's gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin® product candidate, currently in five clinical studies for the treatment of transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease. bluebird bio's oncology pipeline is built upon the company's leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio's lead oncology programs, bb2121 and bb21217, are anti-BCMA CAR T programs partnered with Celgene. bb2121 and bb21217 are each currently being studied in Phase 1 trials for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
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