BLA sub­mission in­cludes results from pivotal Phase 2 KarMMa study eval­u­ating ide-cel in a heavily pre-treated patient pop­u­la­tion with re­lapsed and re­frac­tory mul­ti­ple myeloma

{{image}}Princeton, NJ and Cambridge, MA (Press Release) – Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) to­day an­nounced the sub­mission of their Biologics License Appli­ca­tion (BLA) to the U.S. Food and Drug Admin­istra­tion (FDA) for idecabtagene vicleucel (ide-cel; bb2121), the com­pa­nies’ lead inves­ti­ga­tional B-cell maturation an­ti­gen (BCMA)-directed chi­meric an­ti­gen re­cep­tor (CAR) T cell immuno­therapy, for the treat­ment of adult patients with mul­ti­ple myeloma who have re­ceived at least three prior ther­a­pies, in­clud­ing an immuno­modu­la­tory agent, a pro­te­a­some in­hib­i­tor and an anti-CD38 anti­body.

The sub­mission is based on results from the pivotal Phase 2 KarMMa study. The KarMMa study eval­u­ated the ef­fi­cacy and safety of ide-cel in heavily pre-treated patients with re­lapsed and re­frac­tory mul­ti­ple myeloma. Topline data from KarMMa, reported in De­cem­ber 2019, in­di­cated the study met its pri­mary end­point of over­all re­sponse rate, and the key sec­ond­ary end­point of com­plete re­sponse rate in this patient pop­u­la­tion treated with ide-cel. The safety results were con­sis­tent with those observed in the sup­port­ive Phase 1 CRB-401 study, which eval­u­ated the pre­lim­i­nary safety and ef­fi­cacy of ide-cel. Comprehensive results of the KarMMa study will be pre­sented at a future med­i­cal meeting.

BCMA is a pro­tein that is nearly universally ex­pressed on cancer cells in mul­ti­ple myeloma, making it an im­por­tant poten­tial target for the treat­ment of this aggressive blood cancer. Ide-cel is the first CAR T cell ther­apy sub­mitted for regu­la­tory ap­­prov­al to target this an­ti­gen and for mul­ti­ple myeloma.

Ide-cel was granted Break­­through Therapy Desig­na­tion (BTD) by the FDA and PRIority MEdicines (PRIME) desig­na­tion by the Euro­pean Medicines Agency for re­lapsed and re­frac­tory mul­ti­ple myeloma.

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.

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

Ide-cel is not approved for any in­di­ca­tion in any ge­­og­ra­phy.

About KarMMa

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multi­center, multinational, Phase 2 study eval­u­ating the ef­fi­cacy and safety of ide-cel in adult patients with re­lapsed and re­frac­tory mul­ti­ple myeloma in North America and Europe. The pri­mary end­point of the study is over­all re­sponse rate as assessed by an independent review com­mit­tee (IRC) according to the Inter­na­tional Myeloma Work­ing Group (IMWG) criteria. Complete re­sponse rate is a key sec­ond­ary end­point. Other ef­fi­cacy end­points in­clude time to re­sponse, duration of re­sponse, pro­gres­sion-free sur­vival, over­all sur­vival, minimal residual dis­ease eval­u­ated by Next-Generation Sequencing (NGS) assay and safety. The study en­rolled 140 patients, of whom 128 re­ceived ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after re­ceiv­ing lym­pho­de­plet­ing chemo­ther­apy. All en­rolled patients had re­ceived at least three prior treat­ment regi­mens, in­clud­ing an immuno­modu­la­tory agent, a pro­te­a­some in­hib­i­tor and an anti-CD38 anti­body, and were re­frac­tory to their last regi­men, defined as pro­gres­sion during or within 60 days of their last ther­apy.

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 quality, 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 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, the possibility of un­fa­vor­able results from addi­tional stud­ies involving ide-cel, or bb2121, that such prod­uct can­di­date may not re­ceive regu­la­tory ap­­prov­al for the in­di­ca­tion described in this release in the cur­rently antic­i­pated timeline or at all and, if approved, whether such prod­uct can­di­date for such in­di­ca­tion described in this release will be com­mer­cially 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 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, the possibility of un­fa­vor­able results from addi­tional clin­i­cal trials of ide-cel, that ide-cel may not re­ceive regu­la­tory ap­­prov­al for the in­di­ca­tion described in this release in the cur­rently antic­i­pated timeline or at all and, if approved, whether such prod­uct can­di­date for such in­di­ca­tion described in this release will be com­mer­cially suc­cess­ful, 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.