Bluebird bio: NEJM Publishes Results from Pivotal Phase 2 on IBCMA-Directed CAR T Cell Therapy

 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 March 27, 2021

Results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of bluebird bio, Inc. (Nasdaq: BLUE) and Bristol Myers Squibb’s (NYSE: BMY) investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, idecabtagene vicleucel (ide-cel; bb2121), in adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, were published today in The New England Journal of Medicine.

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 New England Journal of Medicine, underscores the importance of these data and the unprecedented outcomes observed in this triple-class exposed patient population, following a single infusion of ide-cel,” said David Davidson, M.D., chief medical officer, bluebird bio. “Together with our partners at Bristol Myers Squibb, we look forward to the prospect of bringing this first-in-class BCMA-directed CAR T therapy to patients.”

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.

https://www.businesswire.com/news/home/20210224006141/en/The-New-England-Journal-of-Medicine-Publishes-Results-from-Pivotal-Phase-2-KarMMa-Study-of-Idecabtagene-Vicleucel-Ide-cel-bb2121-an-Investigational-BCMA-Directed-CAR-T-Cell-Therapy