• 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 …

• Bristol Myers Squibb to buy out its ex-U.S. mile­stone and royalty obli­ga­tions to bluebird bio for $200 million
• Bristol Myers Squibb assumes re­spon­si­bil­ity­ for vector manu­fac­tur­ing in ex-US territories
• bluebird to hold conference call and webcast to­day, May 11, 2020 at 8:00AM ET

Cambridge, MA (Press Release) – bluebird bio, Inc. (NASDAQ: BLUE) to­day an­nounced that it has amended its existing co-promotion / co-development agree­ment with Bristol Myers Squibb (BMS) to enable the com­pa­nies to focus their efforts on efficient com­mer­cial­iza­tion of idecabtagene vicleucel (ide-cel; bb2121) in the U.S., the com­pa­nies’ lead inves­ti­ga­tional B-cell maturation an­ti­gen (BCMA)-directed chi­meric an­ti­gen re­cep­tor (CAR) T cell immuno­therapy, cur­rent in review with the FDA.

“Under our amended col­lab­o­ration, we and BMS are redoubling our com­mitment to ide-cel …

Shanghai, China (Press Release) – CARsgen Thera­peutics Co., Ltd., a clin­i­cal-stage bio­pharma­ceu­tical com­pany, to­day an­nounced the Euro­pean Medicines Agency (EMA) Com­mit­tee for Orphan Medicinal Products (COMP) adopted a pos­i­tive opinion on CARsgen's appli­ca­tion for orphan desig­na­tion of its inves­ti­ga­tional CT053 CAR T-cell ther­apy, fully human anti-BCMA (B cell maturation an­ti­gen) au­tol­o­gous chi­meric an­ti­gen re­cep­tor (CAR) T cells for the treat­ment of mul­ti­ple myeloma. CT053 was pre­vi­ously granted orphan drug desig­na­tion by the US Food and Drug Admin­istra­tion in August 2019.

"The COMP's adoption of a pos­i­tive opinion for CT053 orphan drug desig­na­tion is …

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

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 …

Phase 1 trial in re­lapsed and re­frac­tory mul­ti­ple myeloma con­tinues to en­roll patients

Gaithersburg, MD (Press Release) – Arcellx, a clin­i­cal-stage bio­pharma­ceu­tical com­pany devoted to devel­op­ing immune cell ther­a­pies for cancer and other dis­eases, to­day an­nounced that the U.S. Food and Drug Admin­istra­tion (FDA) has granted Orphan Drug Desig­na­tion to CART-ddBCMA, its engi­neered T cell ther­apy uti­liz­ing a novel binding domain, for the treat­ment of patients with mul­ti­ple myeloma. Arcellx con­tinues to en­roll patients in its Phase 1 trial of CART-ddBCMA ther­apy for the treat­ment of re­lapsed and re­frac­tory mul­ti­ple myeloma, the first in a series of clin­i­cal trials planned for the stepwise devel­op­ment of …

• Phase 1 trial is de­signed to val­i­date novel binding domain; first step in eval­u­ating Arcellx T cell ther­apy plat­form in the clinic
• Future trials will use Arcellx plat­form to direct T cell ac­­tiv­ity and target mul­ti­ple tumor an­ti­gens
• Therapy granted Fast Track Desig­na­tion for treat­ment of re­lapsed and re­frac­tory mul­ti­ple myeloma

Gaithersburg, MD (Press Release) – Arcellx to­day an­nounced that the first patient has been dosed with its engi­neered T cell ther­apy uti­liz­ing a novel deimmunized syn­thet­ic binding domain in the treat­ment of patients with re­lapsed and re­frac­tory mul­ti­ple myeloma. This first-in-human Phase 1 trial is the first in a series of clin­i­cal trials planned for efficient, stepwise devel­op­ment of the Arcellx ARC-T + sparX cell ther­apy plat­form, with BCMA as an initial target.

“Validating this novel binding domain facilitates sub­se­quent …

• Study met its pri­mary end­point and key sec­ond­ary end­point, demonstrating deep and durable re­sponse­s in a heavily pre-treated mul­ti­ple myeloma patient pop­u­la­tion
• Safety results are con­sis­tent with the data pre­sented in CRB-401 study

Princeton, NJ and Cambridge, MA (Press Release) – Bristol-Myers Squibb Com­pany (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) to­day an­nounced pos­i­tive top-line results from KarMMa, a pivotal, open-label, single arm, multi­center, Phase 2 study of idecabtagene vicleucel (ide-cel; bb2121). KarMMa, which eval­u­ated the ef­fi­cacy and safety of the com­pa­nies’ lead inves­ti­ga­tional BCMA-targeted chi­meric an­ti­gen re­cep­tor (CAR) T cell ther­apy can­di­date for patients with re­lapsed and re­frac­tory mul­ti­ple myeloma, met its pri­mary end­point and key sec­ond­ary end­point.

KarMMa en­rolled 140 patients, of whom 128 …