US-based bluebird bio has started dosing in a Phase I trial (CRB-401) of its product candidate bb2121 for patients with relapsed / refractory multiple myeloma.
The company is committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer.
Being developed in collaboration with Celgene, bb2121 is a chimeric antigen receptor T cell (CAR T) therapy that targets B-cell maturation antigens (BCMA).
Celgene is to exclusively licence bb2121 as part of a collaboration agreement between the two firms.
The trial’s primary objective is to evaluate the maximum tolerated dose of bb2121, and determine the recommended Phase II dose.
Its secondary objective is patient response, measured using the International Myeloma Working Group (IMWG) Response Criteria for multiple myeloma.
bluebird chief Nick Leschly said: "bb2121 is bluebird bio’s first oncology program to enter the clinic, and the treatment of this first patient marks an important milestone for us as we build a broad, fully integrated T cell immunotherapy franchise.
"We are pleased that Celgene has exercised their option to license bb2121. We believe our combined manufacturing, development and commercial expertise will enable us to rapidly advance bb2121 through clinical trials."
Both firms amended and restated their collaboration agreement last June to focus on developing product candidates targeting BCMA during a three-year collaboration term.
CRB-401 study investigator James Kochenderfer said: "BCMA is one of the most exciting targets in multiple myeloma, and we are eager to explore the potential of bb2121 to become an important new treatment option for patients living with multiple myeloma."
Celgene will be responsible for worldwide development and commercialisation of bb2121 after Phase I, while bluebird bio will develop the product throughout the trial and has an optional share in the drug’s US development, promotion and profits.
The deal will see bluebird bio receive a $10m option exercise payment from Celgene. The company is also eligible to receive specified development, regulatory and commercial milestone payments and royalty payments on net sales.
In the trial’s second phase, patients will receive bb2121 to further evaluate the safety, tolerability and clinical activity.