• Celgene has agreed to exercise its op­tion to ex­clu­sively license bb2121 under global stra­te­gic col­lab­o­ration
• bluebird bio to re­ceive $10 million op­tion exercise pay­ment from Celgene

Cambridge, MA (Press Release) – bluebird bio, Inc. (Nasdaq:BLUE), a clin­i­cal-stage com­pany com­mit­ted to devel­op­ing poten­tially trans­for­ma­tive gene ther­a­pies for severe ge­netic dis­eases and T cell-based immuno­therapies for cancer, an­nounced treat­ment of the first patient in a Phase 1 study of its prod­uct can­di­date bb2121 in patients with re­lapsed / re­frac­tory mul­ti­ple myeloma. bb2121 is a chi­meric an­ti­gen re­cep­tor T cell (CAR T) ther­apy targeting B cell maturation an­ti­gen (BCMA), and bluebird bio is devel­op­ing bb2121 in col­lab­o­ration with Celgene Corpo­ra­tion. bluebird bio also an­nounced to­day that Celgene has exercised its op­tion to ex­clu­sively license bb2121, under the terms of the col­lab­o­ration agree­ment be­tween the two com­pa­nies.

“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,” said Nick Leschly, chief bluebird. “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.”

“Despite many recent ad­vances in the field, mul­ti­ple myeloma remains incurable, with almost all patients becoming re­frac­tory to ther­apy even­tu­ally,” said James N. Kochenderfer, M.D., National Cancer Institute, an in­ves­ti­ga­tor for the CRB-401 study. “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.”

bluebird bio and Celgene amended and restated their col­lab­o­ration agree­ment in June 2015 to focus on devel­op­ing prod­uct can­di­dates targeting BCMA during a three-year col­lab­o­ration term. By exercising its ex­clu­sive op­tion under the terms of the agree­ment, Celgene will be responsible for world­wide devel­op­ment and com­mer­cial­iza­tion of bb2121 after Phase 1. bluebird bio is responsible for the devel­op­ment of bb2121 through the com­ple­tion of the CRB-401 Phase 1 study and has an op­tion to share in the devel­op­ment, promotion and profits in the United States. bluebird bio will re­ceive a $10 million op­tion exercise pay­ment from Celgene, and bluebird bio is also eli­gible to re­ceive specified devel­op­ment, regu­la­tory and com­mer­cial mile­stone pay­ments and royalty pay­ments on net sales.

About the CRB-401 Study

The pri­mary objective of the CRB-401 study is to eval­u­ate the max­i­mum tolerated dose of bb2121 and de­ter­mine the rec­om­mended Phase 2 dose. The sec­ond­ary objective is patient re­sponse, measured using the Inter­na­tional Myeloma Work­ing Group (IMWG) Re­sponse Criteria for Multiple Myeloma. The first portion of the study in­cludes a dose-escalation phase in which cohorts of patients will re­ceive ascending doses of bb2121 to de­ter­mine the max­i­mum tolerated dose and estab­lish­ a rec­om­mended Phase 2 dose. The sec­ond portion of the study is a dose ex­pan­sion phase where patients will re­ceive bb2121 to fur­ther eval­u­ate the safety, tolerability and clin­i­cal ac­­tiv­ity at the rec­om­mended Phase 2 dose.

About bluebird bio, Inc.

With its lentiviral-based gene ther­a­pies, T cell immuno­therapy ex­per­tise and gene edit­ing capabilities, bluebird bio has built an integrated prod­uct plat­form with broad poten­tial appli­ca­tion to severe ge­netic dis­eases and cancer. bluebird bio’s gene ther­apy clin­i­cal pro­grams in­clude its Lenti-D™ prod­uct can­di­date, cur­rently in a Phase 2/3 study, called the Starbeam Study, for the treat­ment of cerebral adreno­leuko­dys­trophy, and its LentiGlobin® BB305 prod­uct can­di­date, cur­rently in three clin­i­cal stud­ies for the treat­ment of transfusion-dependent ß-thalassemia, also known as ß-thalassemia major, and severe sickle cell dis­ease. bluebird bio’s on­col­ogy pipe­line is built upon the com­pany’s leadership in 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 (TCR) ther­a­pies. bluebird bio’s lead on­col­ogy pro­gram, bb2121, is an anti-BCMA CAR T pro­gram part­nered with Celgene. bb2121 is cur­rently being studied in a Phase 1 trial for the treat­ment of re­lapsed / re­frac­tory mul­ti­ple myeloma. bluebird bio also has discovery re­search pro­grams uti­liz­ing megaTALs/homing endonuclease gene edit­ing tech­nolo­gies with the poten­tial for use across the com­pany’s pipe­line.

bluebird bio has op­er­a­tions in Cambridge, Massachusetts, Seattle, Washington, and Paris, France.

LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.

Forward-Looking State­ments

This release con­tains “forward-looking state­ments” within the meaning of the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995, in­­clud­ing state­ments re­gard­ing the clin­i­cal and mar­ket poten­tial of the Com­pany’s anti-BCMA on­col­ogy pro­gram, in­­clud­ing its bb2121 prod­uct can­di­date. Any for­ward-looking state­ments are based on man­agement’s current ex­pec­ta­tions of future events and are subject to a number of risks and un­cer­tain­ties that could cause actual results to differ ma­teri­ally and adversely from those set forth in or im­plied by such for­ward-looking state­ments. These risks and un­cer­tain­ties in­clude, but are not limited to, the risk that the pre­clin­i­cal ef­fi­cacy and safety data for our bb2121 prod­uct can­di­date will not be observed in the CRB-401 clin­i­cal study, the risk of cessation or delay of any of the on­go­ing or planned clin­i­cal stud­ies and/or our devel­op­ment of our prod­uct can­di­dates, the risk of a delay in the en­roll­ment of patients in our clin­i­cal stud­ies, the risk that our col­lab­o­ration with Celgene Corpo­ra­tion will not con­tinue or will not be suc­cess­ful, and the risk that any one or more of our prod­uct can­di­dates will not be suc­cess­fully devel­oped and com­mer­cial­ized. For a dis­cus­sion of other risks and un­cer­tain­ties, and other im­por­tant factors, any of which could cause our actual results to differ from those con­tained in the for­ward-looking state­ments, see the section entitled “Risk Factors” in our most recent quar­ter­ly report on Form 10-Q, as well as dis­cus­sions of poten­tial risks, un­cer­tain­ties, and other im­por­tant factors in our sub­se­quent filings with the Se­cu­ri­ties and Ex­change Com­mis­sion. All in­­for­ma­tion in this press release is as of the date of the release, and bluebird bio under­takes no duty to up­date this in­­for­ma­tion unless re­quired by law.

Source: bluebird bio, Inc.