Summit, NJ and Cambridge, MA (Press Release) – Celgene Corpo­ra­tion (Nasdaq: CELG) and bluebird bio, Inc. (Nasdaq: BLUE) to­day an­nounced the com­ple­tion of 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. bb2121 is being devel­oped as part of a Co-Development, Co-Promote and Profit Share Agreement be­tween Celgene and bluebird bio.

“We con­tinue to be ex­cited about bb2121 as a poten­tial first-in-class BCMA-targeted ther­apy for patients with mul­ti­ple myeloma,” said Alise Reicin, M.D., Pres­i­dent, Global Clinical De­vel­op­ment for Celgene. “We would like to thank everyone who enabled this achieve­ment, especially the patients and care­givers, and we congratulate the physicians and others in­volved in the KarMMa study, in­­clud­ing our ded­i­cated part­ners at bluebird bio. We look for­ward to seeing the data from this study and are progressing our broader bb2121 devel­op­ment pro­gram as we ad­vance closer to­ward de­livering this im­por­tant new op­tion to appro­pri­ate patients in need.”

“We are com­mit­ted to devel­op­ing new treat­ment op­tions to im­prove the care of patients with mul­ti­ple myeloma, and com­plet­ing en­roll­ment of the KarMMa study moves us closer to this goal,” said David Davidson, M.D., chief med­i­cal officer, bluebird bio. “As we ad­vance our clin­i­cal stud­ies of bb2121 in earlier lines of ther­apy in col­lab­o­ration with our part­ners at Celgene, we remain very grateful to the patients, families and health­care providers who have made this pro­gram possible.”

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 (BTD) by the U.S. 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.

About Celgene

Celgene Corpo­ra­tion, 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. For more in­for­ma­tion, please visit www.celgene.com.

Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Face­book and YouTube.

About bluebird bio, Inc.

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.

bluebird bio's gene ther­apy clin­i­cal pro­grams 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 (TCR) ther­a­pies. The com­pany’s lead on­col­ogy pro­grams are anti-BCMA CAR T pro­grams part­nered with Celgene.

bluebird 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.

Forward-Looking State­ments

This press release con­tains for­ward-looking state­ments within the meaning of The Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995. Such for­ward-looking state­ments in­clude those re­gard­ing the poten­tial benefits of, and plans relating to the col­lab­o­ration be­tween bluebird bio and Celgene in the devel­op­ment of bb2121; the poten­tial of bb2121 as a thera­peutic drug; and the benefit of each com­pany’s stra­te­gic plans and focus. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “would,” “could,” “potential,” “possible,” “hope” and similar ex­pres­sions are in­tended to identify for­ward-looking state­ments, although not all for­ward-looking state­ments con­tain these identifying words. Such state­ments are subject to nu­mer­ous im­por­tant factors, risks and un­cer­tain­ties that may cause actual events or results to differ ma­teri­ally from current ex­pec­ta­tions and beliefs. For example, there can be no guar­an­tee that any prod­uct can­di­date will be suc­cess­fully devel­oped or com­plete nec­es­sary pre­clin­i­cal and clin­i­cal phases, or that devel­op­ment of any of prod­uct can­di­dates will suc­cess­fully con­tinue, or that mar­ket­ing ap­prov­al will be granted. There can be no guar­an­tee that any pos­i­tive devel­op­ments will result in stock price ap­pre­ci­a­tion. Man­age­ment's ex­pec­ta­tions and, there­fore, any for­ward-looking state­ments in this press release could also be affected by risks and un­cer­tain­ties relating to a number of other im­por­tant factors, in­­clud­ing: results of clin­i­cal trials and pre­clin­i­cal stud­ies, in­­clud­ing sub­se­quent analysis of existing data and new data re­ceived from on­go­ing and future stud­ies; the content and timing of de­ci­sions made by the U.S. FDA and other regu­la­tory author­i­ties, inves­ti­ga­tional review boards at clin­i­cal trial sites and pub­li­ca­tion review bodies; the ability to obtain and main­tain requisite regu­la­tory ap­prov­als and to en­roll patients in planned clin­i­cal trials; unplanned cash re­quire­ments and ex­pen­di­tures; competitive factors; the ability to obtain, main­tain and enforce pat­ent and other in­tel­lec­tual property pro­tec­tion for any prod­uct can­di­dates; the ability to main­tain key col­lab­o­rations; and general economic and mar­ket con­di­tions. These and other risks are described in greater detail under the caption "Risk Factors" in­cluded in each com­pany’s pub­lic filings with the Se­cu­ri­ties and Ex­change Com­mis­sion. Any for­ward-looking state­ments con­tained in this press release speak only as of the date hereof, and neither com­pany has any obli­ga­tion to up­date any for­ward-looking state­ments, whether as a result of new in­for­ma­tion, future events or other­wise, except as may be re­quired by law.

Hyperlinks are provided as a con­ve­nience and for in­for­ma­tional pur­poses only. Neither Celgene nor bluebird bio bears re­spon­si­bil­ity­ for the se­cu­ri­ty or content of ex­ternal websites or websites outside of their re­spec­tive­ con­trol.

All registered trademarks are owned by Celgene Corpo­ra­tion.

Source: Celgene.