• Bristol Myers Squibb to host an in­vestor call to­day at 8:00 a.m. EDT
• bluebird bio to host an in­vestor call to­day at 8:45 a.m. EDT

Princeton, NJ and Cambridge, MA (Press Release) – Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) to­day an­nounced that the com­pa­nies re­ceived a Refusal to File letter from the U.S. Food and Drug Admin­istra­tion (FDA) re­gard­ing the Biologics License Appli­ca­tion (BLA) for idecabtagene vicleucel (ide-cel; bb2121) for patients with heavily pre-treated re­lapsed and re­frac­tory mul­ti­ple myeloma, which was sub­mitted in March 2020.

Upon pre­lim­i­nary review, the FDA de­ter­mined that the Chemistry, Manufacturing and Control (CMC) module of the BLA re­quires fur­ther detail to com­plete the review. No addi­tional clin­i­cal or non-clinical data have been re­quested or are re­quired. Bristol Myers Squibb is planning to resubmit the BLA no later than the end of July 2020.

Bristol Myers Squibb Investor Call and Webcast

Bristol Myers Squibb will hold an in­vestor conference call to discuss this up­date to­day at 8:00 a.m. EDT.

Investors are invited to listen to a live webcast of the call at bms.com/investors or by dialing toll free in the U.S. 877-257-8599 or inter­na­tional 1-631-291-4581, con­firmation code: 5938714. Please dial in at least 15 min­utes in ad­vance to ensure a timely con­nec­tion to the call.

bluebird bio Investor Call and Webcast

bluebird bio will hold an in­vestor conference call to discuss the up­date to­day at 8:45 a.m. EDT.

Investors are invited to listen to a live webcast of the call on the in­vestors page of www.bluebirdbio.com or by dialing toll free in the U.S. (844) 825-4408 or inter­na­tional (315) 625-3227, con­firmation code: 2892826. Please dial in at least 15 min­utes in ad­vance to ensure a timely con­nec­tion to the call.

For Holders of Contingent Value Rights (CVR), Ticker BMY-RT

U.S. FDA ap­­prov­al of ide-cel by March 31, 2021 is one of the re­quired remaining mile­stones of the Contingent Value Rights issued upon the close of the Celgene acquisition in the fourth quarter of 2019. The other is U.S. FDA ap­­prov­al of liso-cel by De­cem­ber 31, 2020. The com­pany is com­mit­ted to work­ing with FDA to progress both appli­ca­tions and achieve the remaining regu­la­tory mile­stones re­quired by the CVR.

About Ide-Cel

Ide-cel is a B-cell maturation an­ti­gen (BCMA)-directed ge­net­ic­ally modified au­tol­o­gous chi­meric an­ti­gen re­cep­tor (CAR) T cell immuno­therapy. The ide-cel CAR is com­prised of a murine extracellular single-chain variable fragment (scFv) spe­cif­ic for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3-ζ chain, in tandem. Ide-cel recog­nizes and binds to BCMA on the surface of mul­ti­ple myeloma cells lead­ing to CAR T cell pro­lif­er­a­tion, cytokine secretion, and sub­se­quent cytolytic kill­ing of BCMA-expressing cells.

In addi­tion to the pivotal KarMMa trial eval­u­ating ide-cel in patients with re­lapsed and re­frac­tory mul­ti­ple myeloma, Bristol Myers Squibb and bluebird bio’s broad clin­i­cal de­vel­op­ment pro­gram for ide-cel in­cludes clin­i­cal stud­ies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treat­ment for patients with mul­ti­ple myeloma, in­clud­ing newly diag­nosed mul­ti­ple myeloma. For more in­for­ma­tion visit clin­i­caltrials.gov.

Ide-cel was granted Break­­through Therapy Desig­na­tion (BTD) by the FDA and PRIority Medicines (PRIME) desig­na­tion, as well as Accelerated Assessment status, by the Euro­pean Medicines Agency for re­lapsed and re­frac­tory mul­ti­ple myeloma.

Ide-cel is being devel­oped as part of a Co-Development, Co-Promotion and Profit Share Agreement be­tween Bristol Myers Squibb and bluebird bio.

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer re­search is to in­crease patients’ quality of life, long-term sur­vival and make cure a possibility. We har­ness our deep scientific ex­peri­ence, cutting-edge tech­nolo­gies and discovery plat­forms to discover, de­vel­op and de­liver novel treat­ments for patients.

Building upon our trans­formative work and legacy in hema­tol­ogy and Immuno-Oncology that has changed sur­vival ex­pec­ta­tions for many cancers, our re­searchers are ad­vanc­ing a deep and diverse pipe­line across mul­ti­ple modalities. In the field of immune cell ther­apy, this in­cludes reg­is­tra­tional CAR T cell agents for nu­mer­ous dis­eases, and a growing early-stage pipe­line that ex­pands cell and gene ther­apy targets, and tech­nolo­gies. We are devel­op­ing cancer treat­ments directed at key bio­logical path­ways using our pro­tein homeo­stasis plat­form, a re­search ca­pa­bil­i­ty that has been the basis of our approved ther­a­pies for mul­ti­ple myeloma and several promising com­pounds in early- to mid-stage de­vel­op­ment. Our scientists are targeting dif­fer­en­t immune sys­tem path­ways to address inter­actions be­tween tumors, the microenvironment and the immune sys­tem to fur­ther ex­pand upon the progress we have made and help more patients respond to treat­ment. Combining these ap­proaches is key to de­livering poten­tial new op­tions for the treat­ment of cancer and addressing the growing issue of re­sis­tance to immuno­therapy. We source inno­va­tion in­ternally, and in col­lab­o­ration with academia, gov­ern­ment, advocacy groups and bio­technol­ogy com­pa­nies, to help make the prom­ise of trans­formational med­i­cines a reality for patients.

About Bristol Myers Squibb

Bristol Myers Squibb is a global bio­pharma­ceu­tical com­pany whose mis­sion is to discover, de­vel­op and de­liver inno­va­tive med­i­cines that help patients prevail over serious dis­eases. For more in­for­ma­tion about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Face­book and Insta­gram.

Celgene and Juno Thera­peutics are wholly owned sub­sid­i­aries of Bristol-Myers Squibb Com­pany. In cer­tain countries outside the U.S., due to local laws, Celgene and Juno Thera­peutics are referred to as, Celgene, a Bristol Myers Squibb com­pany and Juno Thera­peutics, a Bristol Myers Squibb com­pany.

About bluebird bio, Inc.

bluebird bio is pioneering gene ther­apy with pur­pose. From our Cambridge, Mass., headquarters, we’re devel­op­ing gene ther­a­pies for severe ge­netic dis­eases and cancer, with the goal that people facing poten­tially fatal con­di­tions with limited treat­ment op­tions can live their lives fully. Beyond our labs, we’re work­ing to pos­i­tively disrupt the health­care sys­tem to create access, transparency and education so that gene ther­apy can be­come avail­able to all those who can benefit.

bluebird bio is a human com­pany powered by human stories. We’re putting our care and ex­per­tise to work across a spectrum of disorders in­clud­ing cerebral adrenoleukodystrophy, sickle cell dis­ease, β-thalassemia and mul­ti­ple myeloma using three gene ther­apy tech­nolo­gies: gene addi­tion, cell ther­apy and (megaTAL-enabled) gene edit­ing.

bluebird bio has addi­tional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more in­for­ma­tion, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Insta­gram and YouTube.

bluebird bio is a trademark of bluebird bio, Inc.

Bristol Myers Squibb Cautionary State­ment Regarding Forward-Looking State­ments

This press release con­tains “forward-looking state­ments” within the meaning of the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995 re­gard­ing, among other things, the re­search, de­vel­op­ment and com­mer­cial­iza­tion of pharma­ceu­tical prod­ucts. All state­ments that are not state­ments of historical facts are, or may be deemed to be, for­ward-looking state­ments. Such for­ward-looking state­ments are based on historical per­for­mance and current ex­pec­ta­tions and pro­jec­tions about our future fi­nan­cial results, goals, plans and objectives and in­volve­ in­her­ent risks, assump­tions and un­cer­tainties, in­clud­ing in­ternal or ex­ternal factors that could delay, divert or change any of them in the next several years, that are dif­fi­cult to predict, may be beyond our con­trol and could cause our future fi­nan­cial results, goals, plans and objectives to differ ma­teri­ally from those ex­pressed in, or im­plied by, the state­ments. These risks, assump­tions, un­cer­tainties and other factors in­clude, among others, that we may not be able to re-submit the BLA for ide-cel in the time frame described in this release, that the FDA may not ac­cept our re-submitted BLA for ide-cel, and that ide-cel may not re­ceive FDA ap­­prov­al by March 31, 2021 or at all. No for­ward-looking state­ment can be guar­an­teed. Forward-looking state­ments in this press release should be eval­u­ated to­geth­er with the many risks and un­cer­tainties that affect Bristol Myers Squibb’s business and mar­ket, par­tic­u­larly those identified in the cautionary state­ment and risk factors dis­cus­sion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended De­cem­ber 31, 2019, as up­dated by our sub­se­quent Quar­ter­ly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Se­cu­ri­ties and Ex­change Com­mis­sion. The for­ward-looking state­ments in­cluded in this doc­u­ment are made only as of the date of this doc­u­ment and except as other­wise re­quired by appli­cable law, Bristol Myers Squibb under­takes no obli­ga­tion to pub­licly up­date or revise any for­ward-looking state­ment, whether as a result of new in­for­ma­tion, future events, changed cir­cum­stances or other­wise.

bluebird bio Cautionary State­ment Regarding Forward-Looking State­ments

This press release con­tains “forward-looking state­ments” within the meaning of the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995 re­gard­ing, among other things, the re­search, de­vel­op­ment and com­mer­cial­iza­tion of pharma­ceu­tical prod­ucts. All state­ments that are not state­ments of historical facts are, or may be deemed to be, for­ward-looking state­ments. Such for­ward-looking state­ments are based on historical per­for­mance and current ex­pec­ta­tions and pro­jec­tions about our future fi­nan­cial results, goals, plans and objectives and in­volve­ in­her­ent risks, assump­tions and un­cer­tainties, in­clud­ing in­ternal or ex­ternal factors that could delay, divert or change any of them in the next several years, that are dif­fi­cult to predict, may be beyond our con­trol and could cause our future fi­nan­cial results, goals, plans and objectives to differ ma­teri­ally from those ex­pressed in, or im­plied by, the state­ments. These risks, assump­tions, un­cer­tainties and other factors in­clude, among others, that the BLA for ide-cel may not be re-submitted in the time frame described in this release, that the FDA may not ac­cept the re-submitted BLA for ide-cel, and that ide-cel may not re­ceive FDA ap­­prov­al by March 31, 2021 or at all, and that the col­lab­o­ration with Bristol Myers Squibb may not con­tinue or be suc­cess­ful. No for­ward-looking state­ment can be guar­an­teed. Forward-looking state­ments in this press release should be eval­u­ated to­geth­er with the many risks and un­cer­tainties that affect bluebird bio’s business, par­tic­u­larly those identified in the risk factors dis­cus­sion in bluebird bio’s Annual Report on Form 10-K for the year ended De­cem­ber 31, 2019, as up­dated by our sub­se­quent Quar­ter­ly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Se­cu­ri­ties and Ex­change Com­mis­sion. The for­ward-looking state­ments in­cluded in this doc­u­ment are made only as of the date of this doc­u­ment and except as other­wise re­quired by appli­cable law, bluebird bio under­takes no obli­ga­tion to pub­licly up­date or revise any for­ward-looking state­ment, whether as a result of new in­for­ma­tion, future events, changed cir­cum­stances or other­wise.

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

Source: Bristol Myers Squibb and bluebird bio.