• FDA set a target action date of March 27, 2021
• Ide-cel is the first CAR T cell ther­apy ac­cepted for regu­la­tory re­view for mul­ti­ple myeloma

{{image}}Princeton, NJ and Cambridge, MA (Press Release) – Bristol Myers Squibb (NYSE: BMY) and blue­bird bio, Inc. (Nasdaq: BLUE) to­day an­nounced that the U.S. Food and Drug Admin­istra­tion (FDA) has ac­cepted for Priority Review their Biologics License Appli­ca­tion (BLA) for idecabtagene vicleucel (ide-cel; bb2121), the com­pa­nies’ 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 FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.

“Today’s Priority Review mile­stone recog­nizes the po­ten­tial of this first anti-BCMA CAR T cell ther­apy to address a crit­i­cal unmet need of patients with mul­ti­ple myeloma,” said Stanley Frankel, M.D., senior vice pres­i­dent, Cellular Therapy De­vel­op­ment, Bristol Myers Squibb. “We are pleased by the sig­nif­i­cant progress that is being made in part­ner­ship with patients and the mul­ti­ple myeloma com­munity to bring ide-cel to adults with re­lapsed and re­frac­tory mul­ti­ple myeloma who are triple-class exposed and may ben­e­fit from an im­por­tant new thera­peutic op­tion.”

The BLA is based on re­­sults from the pivotal Phase 2 KarMMa study eval­u­ating the ef­fi­cacy and safety of ide-cel in 128 adults with heavily pre-treated and highly re­frac­tory mul­ti­ple myeloma exposed to an immuno­modu­la­tory agent, a pro­te­a­some in­hib­i­tor and an anti-CD38 anti­body. Results from the study were shared during an oral pre­sen­ta­tion as part of the American Society of Clinical Oncology 2020 (ASCO20) Virtual Scientific Program.¹

“Today’s ac­ceptance of the BLA for ide-cel for Priority Review by the FDA marks a key moment in our journey to bring this BCMA-directed CAR T cell ther­apy to mul­ti­ple myeloma patients who are in des­per­ate need of new op­tions,” said Joanne Smith-Farrell, Ph.D., chief op­er­at­ing of­fi­cer on­col­ogy, blue­bird bio. “Based on the body of evi­dence we have gen­er­ated in an ad­vanced, heavily pre-treated patient pop­u­la­tion, our con­fi­dence in the po­ten­tial of ide-cel as an im­por­tant treat­ment op­tion re­mains high. Together with our part­ners at Bristol Myers Squibb, we are com­mit­ted to con­tinue work­ing with the FDA to de­liver this promising ther­apy to patients in an expeditious manner.”

Ide-cel was granted Break­­through Therapy Desig­na­tion (BTD) by the FDA, and PRIority MEdicines (PRIME) desig­na­tion and val­i­da­tion of its Marketing Autho­ri­za­tion Appli­ca­tion (MAA) by the Euro­pean Medicines Agency for re­lapsed and re­frac­tory mul­ti­ple myeloma. Bristol Myers Squibb plans regu­la­tory sub­missions for ide-cel in addi­tional mar­kets out­side the U.S. and EU.

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

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 KarMMa

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multi­center, multinational, Phase 2 study eval­u­ating the ef­fi­cacy and safety of ide-cel in adults 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 sec­ond­ary 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.

About Multiple Myeloma

Multiple myeloma is a can­cer of plasma cells in the bone mar­row.² The exact cause of mul­ti­ple myeloma is not known and cur­rently there is no cure, how­ever, there are a num­ber of treat­ment op­tions avail­able to help man­age the dis­ease.² Patients who have already been treated with some avail­able ther­a­pies but con­tinue to have pro­gres­sion of their dis­ease have “relapsed” and “refractory” mul­ti­ple myeloma, meaning their can­cer has re­turned after they have re­ceived ini­tial treat­ments.³

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.

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 ide-cel, or bb2121, 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 ide-cel. 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 that ide-cel may not re­ceive the FDA’s ap­prov­al for the in­di­ca­tion described in this re­lease in the cur­rently antic­i­pated timeline or at all, and, if ap­prov­ed, whether ide-cel will be com­mer­cially suc­cess­ful, that the pos­i­tive re­­sults for ide-cel may not con­tinue in addi­tional clin­i­cal trials, that ide-cel may not re­ceive mar­ket­ing ap­prov­al in the EU or in any jurisdictions out­side of the US and the EU, 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.

References

1. Munshi NC, et al. Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell ther­apy, in patients with re­lapsed and re­frac­tory mul­ti­ple myeloma (RRMM): ini­tial KarMMa re­­sults. ASCO 2020 Virtual Scientific Program. Abstract #8503.
2. International Myeloma Foundation. What is Multiple Myeloma? Available at: https://www.myeloma.org/what-is-multiple-myeloma. Accessed August 2020.
3. International Myeloma Foundation. Glossary of Myeloma Terms and Definitions. Available at: https://www.myeloma.org/resource-library/glossary-myeloma-terms-definitions. Accessed August 2020.

Source: Bristol Myers Squibb and blue­bird bio.