Try our mobile app
Study met its primary endpoint and key secondary endpoint, demonstrating deep and durable responses in a heavily pre-treated multiple myeloma patient population
Safety results are consistent with the data presented in CRB-401 study
PRINCETON, N.J. & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Bristol-Myers Squibb Company (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) today announced positive top-line results from KarMMa, a pivotal, open-label, single arm, multicenter, Phase 2 study of idecabtagene vicleucel (ide-cel; bb2121). KarMMa, which evaluated the efficacy and safety of the companies’ lead investigational BCMA-targeted chimeric antigen receptor (CAR) T cell therapy candidate for patients with relapsed and refractory multiple myeloma, met its primary endpoint and key secondary endpoint.
KarMMa enrolled 140 patients, of whom 128 patients were treated with ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells. All treated patients were exposed to at least three prior therapies, including an immunomodulatory (IMiD) agent, a proteasome inhibitor (PI) and an anti-CD38 antibody, and all were refractory to their last regimen. Ninety-four percent of patients were refractory to an anti-CD38 antibody and 84% percent were triple refractory (refractory to an IMiD agent, PI and anti-CD38 antibody).
Results for the primary endpoint (overall response rate [ORR]) and key secondary endpoint (complete response rate [CR]), as well as duration of response (DoR) and progression-free survival (PFS) across the target dose levels and at each of the three target doses explored in the study are presented in the table below. The median follow-up duration for all subjects was 11.3 months.
| Ide-cel Treated Population | |||
| 150 x 106 CAR+ T cells (N=4) | 300 x 106 CAR+ T cells (N=70) | 450 x 106 CAR+ T cells (N=54) | 150-450 x 106 CAR+ T cells (N=128) |
ORR, n (%) | 2 (50.0) | 48 (68.6) | 44 (81.5) | 94 (73.4) |
CR/sCR, n (%) | 1 (25.0) | 20 (28.6) | 19 (35.2) | 40 (31.3) |
Median DoR, months | --- | 9.9 | 11.3 | 10.6 |
Median PFS, months | --- | 5.8 | 11.3 | 8.6 |
Median DOR and median PFS are not reported for the 150 x 106 CAR+ T cells dose group due to the small number of evaluable patients | ||||
Overall, the safety results were consistent with those observed in the phase 1 CRB-401 study, which evaluated the preliminary safety and efficacy of ide-cel. Instances of grade 3 or higher cytokine release syndrome (CRS) occurred in 5.5% (7/128) of patients, including one fatal CRS event. Investigator identified grade 3 or higher neurotoxicity events (iiNT) occurred in 3.1% (4/128) of patients and there were no Grade 4 iiNT events reported. Grade 3 or higher CRS and iiNT events were reported in
