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 …

• Phase 1 clin­i­cal trial of off-the-shelf (allogeneic) anti-BCMA CAR T ther­apy can­di­date for patients with re­lapsed / re­frac­tory mul­ti­ple myeloma ex­pec­ted to begin dosing patients in 2020
• PBCAR269A has re­ceived Orphan Drug Desig­na­tion from the FDA for the treat­ment of mul­ti­ple myeloma
• First pro­gram for which clin­i­cal trial ma­teri­al will be gen­er­ated fully in-house at Precision’s Manu­factur­ing Center for Advanced Thera­­peutics (MCAT) in Durham, N.C.

Durham, NC (Press Release) – Precision BioSciences, Inc. (Nasdaq: DTIL), a genome edit­ing com­pany ded­i­cated to im­prov­ing life through the appli­ca­tion of its pioneering, pro­pri­e­tary ARCUS® plat­form, to­day an­nounced that the U.S. Food and Drug Admin­istra­tion (FDA) has ac­cepted its Inves­ti­ga­tional New Drug (IND) appli­ca­tion for PBCAR269A, the Com­pany’s third allo­geneic chi­meric an­ti­gen re­cep­tor (CAR) T cell ther­apy can­di­date. The FDA has also granted Orphan Drug Desig­na­tion to PBCAR269A for the treat­ment of mul­ti­ple myeloma. Wholly-owned by Precision, PBCAR269A is an allo­geneic CAR …

Increasing BCMA Surface Expression with Gamma Secretase Inhibitor Niro­gace­stat May Enable Deep and Durable Responses to ALLO-715, an Inves­ti­ga­tional Anti-BCMA Allogeneic CAR T Therapy

South San Francisco, CA and Stamford, CA (Press Release) – Allogene Thera­peutics, Inc. (Nasdaq: ALLO), a clin­i­cal-stage bio­technol­ogy com­pany pioneering the de­vel­op­ment of allo­geneic chi­meric an­ti­gen re­cep­tor T cell (AlloCAR T™) ther­a­pies for cancer and Spring­Works Thera­peutics, Inc. (Nasdaq: SWTX), a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on devel­op­ing life-changing med­i­cines for patients with severe rare dis­eases and cancer, to­day an­nounced that they have entered into a clin­i­cal trial col­lab­o­ration agree­ment. This agree­ment will eval­u­ate ALLO-715, Allogene’s inves­ti­ga­tional anti-B-cell maturation an­ti­gen (BCMA) AlloCAR T ther­apy in com­bi­na­tion with Spring­Works’ inves­ti­ga­tional gamma secretase in­hib­i­tor (GSI), niro­gace­stat, …

• 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 …

• Newest desig­na­tion for JNJ-4528 is sup­ported by Phase 1b/2 CARTITUDE-1 study in adults with re­lapsed or re­frac­tory mul­ti­ple myeloma
• Initial re­­sults from CARTITUDE-1 study to premiere at the American Society of He­ma­tol­ogy Annual Meeting

Raritan, NJ (Press Release) – The Janssen Pharma­ceu­tical Com­panies of John­son & John­son an­nounced to­day that the U.S. Food and Drug Admin­istra­tion (FDA) has granted Break­­through Therapy Desig­na­tion for JNJ-68284528 (JNJ-4528), an inves­ti­ga­tional B cell maturation an­ti­gen (BCMA)-directed chi­meric an­ti­gen re­cep­tor T cell (CAR-T) ther­apy in pre­vi­ously treated patients with mul­ti­ple myeloma. Break­­through Therapy Desig­na­tion is granted to expedite the devel­op­ment and regu­la­tory re­view of an inves­ti­ga­tional med­i­cine that is in­tended to treat a serious or life-threatening con­di­tion. The criteria for Break­­through Therapy Desig­na­tion …