Results from the KarMMa study, a pivotal, open-label, single arm, multicenter, phase II study of idecabtagene vicleucel met its primary endpoint of overall response rate in the treatment of relapsed and refractory multiple myeloma.
The study is sponsored by Bristol-Myers Squibb Co. and bluebird bio Inc.
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 also met a key secondary endpoint, complete response rate.
The primary endpoint overall response rate was 73.4% (n=94/128) across three doses. The response rate was dose-dependent, 50.0% (n=2/4) in the lowest, 68.6% (n=48/70) in the middle and 81.5% (n=44/54) in the highest. The complete response rates were 25%, 28.6% and 35.2%, respectively.
Median duration of response was 10.6 months and median progression-free survival was 8.6 months.
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 agent, a proteasome inhibitor 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).
The median follow-up duration for all subjects was 11.3 months.
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.
“For multiple myeloma patients who have relapsed and become refractory to current treatment options, there remains a high unmet need, as these patients typically experience low response rates, short response durations and poor survival,” Kristen Hege, senior vice president of Hematology/Oncology and Cell Therapy and Early Clinical Development for Bristol-Myers Squibb, said in a statement.
“The KarMMa study provides further support for ide-cel as a potential therapeutic option in this heavily pre-treated patient population, and we are encouraged by these data, especially the outcomes observed at the highest target dose of 450 x 106 CAR+ T cells,” Hege said.
“We are actively preparing for submission of these data to health authorities for proposed initial registration of ide-cel as a first-in-class BCMA-targeted CAR T-cell therapy.”
