First pivotal study of BCMA-directed CAR T cell therapy in triple-class exposed relapsed and refractory multiple myeloma
Biologics License Application (BLA) for ide-cel was accepted by FDA for Priority Review, with a target action date of
The KarMMa study met its primary endpoint of overall response rate and key secondary endpoint of complete response rate. The data from the study demonstrates deep and durable responses with ide-cel treatment in triple-class exposed RRMM patients (n=128).
“The publication of KarMMa, the first pivotal study of a CAR T cell therapy in multiple myeloma, in The
Clinically meaningful responses were reported in heavily pre-treated patients across all dose levels and in multiple high-risk subgroups, including those with high-risk cytogenetics, triple- or penta-refractory disease, high tumor burden at baseline, and extramedullary disease. Clinically meaningful improvement was also observed across measures for median duration of response, median progression-free survival and overall survival in treated patients.
In the KarMMa study, ide-cel demonstrated a safety profile consistent with known toxicities of CAR T cell therapies, regardless of dose level. The most frequently reported adverse events were cytopenia and cytokine release syndrome.
“Despite the progress made in the treatment of multiple myeloma over the past decade, long-term disease-free survival is uncommon and relapses are inevitable. Currently, the patients who have progressed through the three main classes of therapy do not have very effective therapeutic options and their outcome are often poor,” said
The research included in this manuscript was first presented at the
Ide-cel is not approved for any indication in any geography.
About Idecabtagene Vicleucel (ide-cel, bb2121)
Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 (4-1BB) and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.
bluebird bio and Bristol Myers Squibb’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including high risk newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.
Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between
KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders, including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.
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bluebird bio Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of ide-cel. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, the possibility that ide-cel may not receive the FDA’s approval for the indication described in this release in the currently anticipated timeline or at all, and, if approved, whether ide-cel will be commercially successful, that the positive results for ide-cel may not continue in additional clinical trials, that ide-cel may not receive marketing approval in the EU or in any jurisdictions outside of the US and the EU, and that the collaboration with
- Munshi NC, Anderson LD, Shah N, et al. Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021 February 25;384:705-716.
- Munshi NC, et al. Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): initial KarMMa results. ASCO 2020 Virtual Scientific Program. Abstract #39T850339T.
Raje N, Berdeja J, Lin Y, et al. Anti-BCMA CAR T-cell therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019
ClinicalTrials.gov. Efficacy and safety study of bb2121 in subjects with relapsed and refractory multiple myeloma (KarMMa). Available at: https://clinicaltrials.gov/ct2/show/NCT03361748. Accessed
Source: bluebird bio, Inc.