– 100% of patients (n=6) in second and third dose cohorts achieved an
objective response; two patients MRD-negative; overall response rate
– Two patients achieved stringent complete responses, with 6 and 4 months follow-up –
– Among all dosed patients (n=11), no dose-limiting toxicities to date, no Grade 3 or Grade 4 cytokine release syndrome or Grade 3 or Grade 4 neurotoxicity observed –
– Company to hold conference call and webcast with slides at
“We are pleased that these early data from our ongoing Phase 1 study of
bb2121 demonstrate objective anti-tumor responses in heavily pre-treated
patients with multiple myeloma, with all patients in the 15.0 x 107
and 45.0 x 107 CAR+ T cell dose cohorts achieving responses,
including among them, patients with stringent complete responses and
elimination of minimal residual disease,” said
Clinical remissions and limited toxicity in a first-in-human multicenter study of bb2121, a novel anti-BCMA CAR T cell therapy for relapsed/refractory multiple myeloma (Abstract #14, LBA)
Session: Plenary Session 7
The open-label Phase 1 CRB-401 study (NCT02658929) is investigating the
administration of bb2121 anti-BCMA CAR T cells in patients with relapsed
and/or refractory multiple myeloma. The primary endpoint of the study is
incidence of adverse events (AEs) and abnormal laboratory test results,
including dose-limiting toxicities (DLTs). The study also seeks to
assess disease-specific response criteria including: complete response
(CR), very good partial response (VGPR), and partial response (PR)
according to 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 lentiviral vector encoding the anti-BCMA CAR.
Results, as of
|CAR+ T Cell dose||5.0 x 107||15.0 x 107||45.0 x 107|
sCR (time to response: 2 months)
sCR* (time to response: 4 months)
*Both patients with
All patients in cohorts 2 and 3 with bone
6 (range: 5-13); all patients had a prior autologous stem cell
No dose-limiting toxicities and no Grade 3 or higher
Investor Webcast Information
bluebird bio will host a conference call to discuss these data at
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™ BB305 product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-thalassemia 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 program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial 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,
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s bb2121 product candidate to treat relapsed/refractory multiple myeloma. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks that the preliminary positive results from our ongoing CRB-401 clinical trial of bb2121 will not continue or be repeated in our ongoing or planned clinical trials of bb2121, the risk of a delay in the enrollment and treatment of patients in our CRB-401 clinical study, and the risk that our bb2121 product candidate will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the
Source: bluebird bio, Inc.
bluebird bio, Inc.
Manisha Pai, 617-245-2107
bluebird bio, Inc.
Elizabeth Pingpank, 617-914-8736
Pure Communications, Inc.
Dan Budwick, 973-271-6085