Designations based on pre­lim­i­nary clin­i­cal data from on­go­ing phase I study of bb2121 in heavily pre-treated mul­ti­ple myeloma

Summit, NJ and Cambridge, MA (Press Release) – Celgene Corpo­ra­tion (NASDAQ:CELG) and bluebird bio, Inc. (NASDAQ:BLUE) to­day an­nounced that bb2121, a chi­meric an­ti­gen re­cep­tor T-cell (CAR-T) ther­apy targeting b-cell maturation an­ti­gen (BCMA) in pre­vi­ously treated patients with mul­ti­ple myeloma, has been granted Break­through Therapy Desig­na­tion (BTD) by the U.S. Food and Drug Admin­istra­tion (FDA) and PRIority MEdicines (PRIME) eligibility by the Euro­pean Medicines Agency (EMA).

BTD desig­na­tion and PRIME eligibility for bb2121 were based on pre­lim­i­nary clin­i­cal data from the on­go­ing phase 1 study CRB-401. Updated data from CRB-401 is scheduled to be pre­sented at the 59th annual meeting of the American Society of He­ma­tol­ogy in Atlanta during an oral pre­sen­ta­tion on Dec. 11.

"Receiving Break­through Therapy Desig­na­tion and PRIME eligibility for bb2121 fur­ther underscores the poten­tial of this novel cellular immuno­therapy ap­proach to mul­ti­ple myeloma treat­ment," said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. "We will work closely with these agencies as we ac­cel­er­ate devel­op­ment of bb2121, a novel tech­nology and ther­apy for patients with mul­ti­ple myeloma."

"Despite recent ad­vances, mul­ti­ple myeloma remains an incurable dis­ease, and heavily pre­treated patients have limited thera­peutic op­tions," said David Davidson, M.D., Chief Medical Officer for bluebird bio. "Early data sug­gest that treat­ment with bb2121 has the poten­tial to induce durable re­sponses in this patient pop­u­la­tion. It is en­cour­ag­ing for both the FDA and EMA to identify bb2121 as a can­di­date for ac­cel­er­ated devel­op­ment as we con­tinue our work with Celgene to bring this ther­apy to patients in need of new op­tions."

Breakthrough Therapy Desig­na­tion is in­tended to expedite the devel­op­ment and review of drugs that are in­tended to treat serious or life-threatening con­di­tions. The criteria for break­­through ther­apy desig­na­tion re­quire pre­lim­i­nary clin­i­cal evi­dence that dem­onstrates the drug may have sub­stan­tial im­prove­ment on at least one clin­i­cally sig­nif­i­cant end­point over avail­able ther­apy.

PRIME is a pro­gram launched by the EMA to en­hance sup­port for the devel­op­ment of med­i­cines that target an unmet med­i­cal need. This voluntary pro­gram is based on en­hanced inter­action and early dialogue with de­vel­opers of promising med­i­cines, to op­ti­mize devel­op­ment plans and speed up evaluation so these med­i­cines can reach patients earlier. The pro­gram focuses on med­i­cines that may offer a major thera­peutic ad­van­tage over existing treat­ments, or benefit patients without treat­ment op­tions. These med­i­cines are con­sidered priority med­i­cines by EMA. To be ac­cepted for PRIME, a med­i­cine must show its poten­tial to benefit patients with unmet med­i­cal needs based on early clin­i­cal data.

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 an integrated prod­uct plat­form with broad poten­tial appli­ca­tion to severe ge­netic dis­eases and cancer. 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 has op­er­a­tions in Cambridge, Massachusetts, Seattle, Washington and Europe.

About the bluebird bio-Celgene Collaboration

In March 2013, bluebird bio and Celgene entered into a col­lab­o­ration to de­vel­op chi­meric an­ti­gen re­cep­tor (CAR) T cell ther­a­pies to target and destroy cancer cells. In June 2015, the col­lab­o­ration was amended and restated to focus on devel­op­ing prod­uct can­di­dates targeting B-cell maturation an­ti­gen (BCMA). bluebird bio's lead on­col­ogy pro­gram, bb2121, is an anti-BCMA CAR T pro­gram cur­rently being studied in a Phase 1 trial for the treat­ment of re­lapsed / re­frac­tory mul­ti­ple myeloma. bluebird bio and Celgene are also work­ing to­geth­er on a sec­ond anti-BCMA CAR T pro­gram, bb21217.

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; 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. 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.

Source: Celgene.