Celgene Corp. and Bluebird Bio Inc. said its investigational therapy for multiple myeloma — a bone marrow cancer that affects plasma cells — demonstrated benefit in updated results from an early stage trial.
The two biotechs are jointly developing bb2121 — an anti-B-cell maturation antigen CAR-T cell therapy. The chimeric antigen receptor T-cell, or CAR-T cell therapy uses engineered versions of the patients' own cells to target the disease.
The ongoing CRB-401 phase 1 study of bb2121 showed an overall response rate of 94% in patients with late-stage relapsed or refractory multiple myeloma in active dose cohorts and a complete response rate of 56%.
But the trial failed to achieve the median progression-free survival with median follow up of 40 weeks.
Progression-free survival was 81% and 71% at six months and nine months, respectively.
The trial's objective is to evaluate the preliminary safety and efficacy of bb2121 and determine a recommended phase 2 dose.
"The responses achieved in this relapsed and refractory patient population, combined with the generally tolerable safety profile, reinforce the potential role of bb2121 as a groundbreaking CAR-T therapy in multiple myeloma," said Dave Davidson, Bluebird Bio's chief medical officer.
There were two deaths in the active cohorts at 22 and 69 weeks following the infusion. The first was due to cardiac arrest and the second was due to myelodysplastic syndrome, a group of cancers where immature blood cells in the bone marrow do not mature.
In the dose expansion phase, one patient experienced grade 4 neurotoxicity which was successfully managed. As of the data cut-off date of Oct. 2 for the updated results, 21 patients had been enrolled and dosed in the dose-escalation phase of the study.
