Updated results from ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin™ for sickle cell disease gene therapy (bb1111)
Long-term efficacy and safety results for betibeglogene autotemcel gene therapy in adult and pediatric patients with beta-thalassemia across genotypes (LTF-303, HGB-207, HGB-212)
Updated safety and efficacy results from ongoing studies of idecabtagene vicleucel and bb21217 in multiple myeloma
Updated results from patients in Group C of the company’s Phase 1/2 HGB-206 study of LentiGlobin™ for SCD gene therapy (bb1111) will be presented.
bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia) in TDT.
Data from across the company’s multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.
Sickle Cell Disease Data at ASH
Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Date/Time: Oral #365,
Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Date/Time: Oral #677,
The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD
Date/Time: Poster #1730,
Transfusion-Dependent β-Thalassemia Data at ASH
Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up
Date/Time: Oral #153,
Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (
Date/Time: Oral #154,
Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia in the Phase 3 HGB-207 Study
Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit,
Date/Time: Poster #776,
Response of patients with transfusion-dependent β-thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for β-thalassemia) gene therapy based on HBB genotype and disease genetic modifiers
Date/Time: Poster #1699,
Multiple Myeloma Data at ASH
Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes
Date/Time: Oral #130,
Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study
Date/Time: Oral #131,
Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial
Date/Time: Oral #437,
Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis
Date/Time: Poster #1367,
Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma
Date/Time: Poster #1378,
Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy
Date/Time: Poster #1401,
Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma
Date/Time: Poster #1405,
KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma
Date/Time: Poster #1418,
Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial
Date/Time: Poster #1598,
A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma
Date/Time: Poster #1653,
Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype
Date/Time: Poster #2315,
KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma
Date/Time: Poster #2323,
Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study
Date/Time: Poster #3234,
Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMA‑directed CAR T Cell Therapy, Idecabtagene Vicleucel (ide‑cel, bb2121): Results from the KarMMa Trial
Date/Time: Poster #3465,
Abstracts outlining bluebird bio’s accepted data at ASH are available on the ASH conference website.
About LentiGlobin for SCD (bb1111)
SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complications—such as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.
LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bio’s clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.
LentiGlobin for SCD was designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbA-T87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.
LentiGlobin for SCD received orphan medicinal product designation from the
bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.
LentiGlobin for SCD is investigational and has not been approved in any geography.
About betibeglogene autotemcel
Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.
Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbA-T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.
Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.
Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On
bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.
About idecabtagene vicleucel (ide-cel, bb2121)
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. Ide-cel was granted accelerated assessment by the
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 RRMM in
CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.
In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibb’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.
Ide-cel is not approved for any indication in any geography.
bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.
bluebird bio’s clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.
bb21217 is not approved for any indication in any geography.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent β-thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD, and the potential for idecabtagene vicleucel and bb21217 to treat multiple myeloma; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the
Source: bluebird bio, Inc.