28 November 2018 - - US-based biopharmaceutical company Celgene Corp. (NASDAQ: CELG) and US-based bluebird bio, Inc. (NASDAQ: BLUE) have completed enrollment for the KarMMa pivotal study of bb2121, the companies' lead investigational anti-BCMA CAR T cell therapy candidate for patients with relapsed and refractory multiple myeloma, the companies said.

bb2121 is being developed as part of a Co-Development, Co-Promote and Profit Share Agreement between Celgene and bluebird bio.

KarMMa is a pivotal, open-label, single-arm, multi-center phase 2 study evaluating the efficacy and safety of bb2121 in patients with relapsed and refractory multiple myeloma.

In November 2017, bb2121 was granted Breakthrough Therapy Designation by the US Food and Drug Administration and PRIority Medicines (PRIME) eligibility by the European Medicines Agency. The BTD designation and PRIME eligibility were based on preliminary clinical data from the phase 1 CRB-401 study.

The FDA action date for the bb2121 NDA is anticipated in 2020. bb2121 is currently an investigational therapy; safety and efficacy have not yet been established. bb2121 has not been approved for use by any health authority.

Celgene Corp., headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation.

bluebird bio, with its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built a pipeline with broad potential application in severe genetic diseases and cancer.

The company's gene therapy clinical programmes include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and sickle cell disease.

bluebird bio's oncology pipeline is built upon the company's lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor therapies. The company's lead oncology programs are anti-BCMA CAR T programs partnered with Celgene.
The company's bio's discovery research programs include utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.

bluebird bio has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina and Zug, Switzerland.