Updated safety and efficacy results from
ongoing Phase 1 CRB-402 study of bb21217 in relapsed/refractory
multiple myeloma
Updated results from ongoing Phase 1/2
(HGB-206) study of LentiGlobin™ gene therapy for patients with
sickle cell disease
New data from ongoing Phase 3 studies of
LentiGlobin™ gene therapy for β-thalassemia in pediatric,
adolescent and adult patients
bluebird bio, Inc. (Nasdaq: BLUE) announced today that new and
updated data from its investigational gene and cell therapy
programs for multiple myeloma, sickle cell disease (SCD) and
transfusion-dependent β-thalassemia (TDT) will be presented at the
61st American Society of Hematology (ASH) Annual Meeting and
Exposition in Orlando, Florida, December 7 - 10.
bluebird bio will present updated safety and efficacy data from
the ongoing Phase 1 clinical study (CRB-402) of bb21217. bb21217 is
an investigational BCMA-targeted chimeric antigen receptor (CAR) T
cell therapy being studied, in partnership with Celgene, in
patients with relapsed/refractory multiple myeloma (RRMM).
In addition, data from clinical studies of LentiGlobin™ gene
therapy for β-thalassemia, including results up to 61 months from
the long-term follow-up study (LTF-303) and updated results from
the completed Phase 1/2 Northstar (HGB-204) study, will be
presented at ASH. The company will also present new data from the
ongoing Phase 3 Northstar-2 (HGB-207) study in pediatric,
adolescent and adult patients who do not have a β0/β0 genotype and
from the ongoing Phase 3 Northstar-3 (HGB-212) study in pediatric,
adolescent and adult patients who have β0/β0 genotype or an
IVS-I-110 mutation at both alleles of the β-globin gene.
New data from the company’s Phase 1/2 HGB-206 study of
LentiGlobin gene therapy for SCD will include additional patients
treated in the study and updated data for those previously
reported. The company will also present data from exploratory
assays designed to assess the relationship between drug product
characteristics and red blood cell physiology in patients treated
with LentiGlobin for SCD.
Updated Data from Ongoing Phase 1
Clinical Study (CRB-402) of bb21217
Updated Results from an Ongoing Phase 1 Clinical Study of
bb21217 Anti-BCMA CAR T Cell Therapy Presenting Author: Jesus
G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville,
Tenn. Date & Time: Oral #927, Monday, December 9, 2019,
6:45 p.m. ET
bb21217, an investigational BCMA-targeted CAR T cell therapy
being developed in partnership with Celgene, is one of bluebird
bio's lead oncology programs. bb21217 uses the idecabtagene
vicleucel CAR molecule (formerly referred to as bb2121) and is
manufactured with a process intended to increase the in vivo
persistence of CAR T cells.
This presentation will include updated data from the Phase 1
CRB-402 study, the first-in-human study of bb21217 in patients with
RRMM, designed to assess the primary endpoint of safety as well as
other pre-defined endpoints including efficacy and pharmacokinetics
measurements. CRB-402 is a two-part (dose escalation and dose
expansion), open-label, multi-site Phase 1 study of bb21217 in
adults with RRMM with a projected final enrollment of 74
patients.
Data in the abstract include results as of the data cutoff date
of April 20, 2019 for 22 patients who have received bb21217 at
three dose levels (12 at 150 x 106 CAR+ T cells; six at 300 x 106
CAR+ T cells; and four at 450 x 106 CAR+ T cells). These patients
had a median of seven prior lines of therapy (min-max: 4 – 17
lines), 18 patients had a prior autologous stem cell transplant, 19
patients received daratumumab and 13 patients received prior
treatment with bortezomib, lenalidomide, carfilzomib, pomalidomide
and daratumumab.
As of the data cutoff, the adverse events observed were
consistent with known toxicities of CAR T therapies. Thirteen of 22
patients developed cytokine release syndrome (CRS); five Grade 1,
seven Grade 2, and one Grade 3 case. All 13 patients responded to
supportive care, tocilizumab and/or corticosteroids. Five of 22
patients developed neurotoxicity; one Grade 1, two Grade 2, one
Grade 3 (vertigo/dizziness), and one Grade 4 (encephalopathy,
previously reported). For the one patient previously reported with
Grade 4 neurotoxicity, Grade 3 CRS was also reported, and both have
resolved.
Eighteen patients were evaluable for clinical response with
> two months of follow-up or
progressive disease within two months. Eighty-three percent
(n=15/18) of evaluable patients demonstrated clinical response per
the International Myeloma Working Group Uniform Response Criteria
for multiple myeloma. As of the data cutoff, with the median
follow-up after bb21217 infusion of five months (min-max: <1 –
18 months), nine patients remained in response, including two
patients with ongoing response at 15 and 18 months.
Evidence of myeloma in the bone marrow, known as minimal
residual disease, was undetectable by next-generation sequencing at
a sensitivity level of 10-5 or better in all responders who had
evaluable bone marrow samples (n=10) at Month 1. CAR T cell
persistence was observed in six of eight patients evaluable at six
months and in two of two patients evaluable at 12 months.
This study is ongoing to evaluate the potential safety and
efficacy of treatment with bb21217, and updated results, including
early clinical and CAR T cell persistence data, will be shared at
the ASH conference.
Multiple Myeloma Presentations at
ASH
Markers of Initial and Long-Term Responses to Idecabtagene
Vicleucel (Ide-Cel; bb2121) in the CRB-401 Study in
Relapsed/Refractory Multiple Myeloma Presenting Author: Ethan
G. Thompson, Ph.D., Celgene, Seattle, Wash. Date & Time:
Poster #4328, Monday, December 9, 2019, 6:00 – 8:00 p.m. ET
Updated Results from an Ongoing Phase 1 Clinical Study of
bb21217 anti-BCMA CAR T Cell Therapy Presenting Author: Jesus
G. Berdeja, M.D., Sarah Cannon Center for Blood Cancers, Nashville,
Tenn. Date & Time: Oral #927, Monday, December 9, 2019,
6:45 p.m. ET
SCD Presentations at ASH
The Relationships Between Target Gene Transduction,
Engraftment of HSCs and RBC Physiology in Sickle Cell Disease Gene
Therapy Presenting Author: Melissa Bonner, Ph.D., bluebird bio,
Cambridge, Mass. Date & Time: Oral #206, Saturday,
December 7, 2019, 2:15 p.m.
Exploring the Drivers of Clinical Benefit in Initial Patients
Treated in the HGB-206 Study of LentiGlobin for Sickle Cell Disease
(SCD) Gene Therapy Presenting Author: Mark Walters, M.D.,
Benioff Children’s Hospital, Oakland, Calif. Date &
Time: Poster #2061, Saturday, December 7, 2019, 5:30 – 7:30
p.m.
Resolution of Sickle Cell Disease Manifestations in Patients
Treated with LentiGlobin Gene Therapy: Updated Results from the
Phase 1/2 HGB-206 Group C Study Presenting Author: Julie
Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala.
Date & Time: Poster #990, Saturday, December 7, 2019,
5:30 – 7:30 p.m.
TDT Presentations at ASH
Clinical Outcomes after Allogeneic Hematopoietic Stem Cell
Transplantation in Patients with Transfusion-Dependent
β-Thalassemia Treated at the Bambino Gesù Children’s Hospital,
Rome, Italy Presenting Author: Pietro Merli, M.D., IRCCS
Ospedale Pediatrico Bambino Gesù, Rome, Italy Date &
Time: Poster #969, Saturday, December 7, 2019, 5:30 – 7:30
p.m.
Northstar-3: Interim Results from a Phase 3 Study Evaluating
LentiGlobin Gene Therapy in Patients with Transfusion-Dependent
β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles
of the HBB Gene Presenting Author: Ashutosh Lal, M.D., UCSF
Benioff Children’s Hospital, Oakland, Calif. Date &
Time: Oral #815, Monday, December 9, 2019, 5:30 p.m.
Northstar-2: Updated Safety and Efficacy Analysis of
LentiGlobin Gene Therapy in Patients with Transfusion-Dependent
β-Thalassemia and Non-β0/β0 Genotypes Presenting Author:
Alexis Thompson, M.D., MPH, Ann & Robert H. Lurie Children’s
Hospital of Chicago, Chicago, Ill. Date & Time: Poster
#3543, Monday, December 9, 2019, 6:00 – 8:00 p.m.
Long-Term Clinical Outcomes of LentiGlobin Gene Therapy for
Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204)
Study Presenting Author: Janet Kwiatkowski, M.D., MSCE,
Children's Hospital of Philadelphia, Philadelphia, Pa. Date
& Time: Poster #4628, Monday, December 9, 2019, 6:00 – 8:00
p.m.
Routine Management, Healthcare Resource Use and
Patient/Caregiver-Reported Outcomes of Patients with
Transfusion-Dependent β-Thalassaemia in the United Kingdom: A Mixed
Methods Observational Study Presenting Author: Farrukh Shah,
MBBS, FRCP, FRCPath, M.D., Whittington Hospital, London, U.K.
Date & Time: Poster #3550, Monday, December 9, 2019,
6:00 – 8:00 p.m.
SCD and TDT Presentation at
ASH
Results from the Completed HGB-205 Trial of LentiGlobin for
β-Thalassemia and LentiGlobin for Sickle Cell Disease Gene Therapy
Presenting Author: Elisa Magrin, Ph.D., Necker Children’s
Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
Date & Time: Poster #3358, Sunday, December 8, 2019,
6:00 – 8:00 p.m.
Abstracts outlining bluebird bio’s accepted data at ASH will be
available on the ASH conference website at 9 a.m. EST today.
About ide-cel and bb21217 for Multiple Myeloma
bluebird bio's lead oncology programs, idecabtagene vicleucel
(ide-cel, formerly referred to as bb2121) and bb21217, are
investigational BCMA-targeted chimeric antigen receptor (CAR) T
cell therapies being studied in a broad clinical development
program for patients with multiple myeloma. ide-cel and bb21217 are
being developed in partnership with Celgene.
KarMMa is a registration-enabling, open-label, single-arm,
multi-center Phase 2 study evaluating the efficacy and safety of
ide-cel in patients with relapsed/refractory multiple myeloma. In
November 2018, bluebird bio announced completion of enrollment in
the trial. ide-cel was granted Breakthrough Therapy Designation by
the U.S. Food and Drug Administration and Priority Medicines
(PRIME) eligibility by the European Medicines Agency in November
2017 based on preliminary clinical data from the Phase 1 CRB-401
study.
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 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 with a
projected final enrollment of 74 patients. For more information
visit: clinicaltrials.gov using identifier NCT03274219.
ide-cel and bb21217 are not approved for any indication in any
geography.
About LentiGlobin for Sickle Cell Disease
LentiGlobin for sickle cell disease 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.
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 received Orphan Medicinal Product
designation from the European Commission for the treatment of
SCD.
The U.S. Food and Drug Administration granted Orphan Drug status
and Regenerative Medicine Advanced Therapy designation for
LentiGlobin for the treatment of SCD.
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/medical-professionals/our-clinical-trials/
or clinicaltrials.gov and use identifier NCT02633943 for
LTF-303.
About LentiGlobin for β-Thalassemia
The European Commission granted conditional marketing
authorization for LentiGlobin for TDT, to be marketed as ZYNTEGLO™
(autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy,
for patients 12 years and older with TDT who do not have a β0/β0
genotype, for whom hematopoietic stem cell (HSC) transplantation is
appropriate, but a human leukocyte antigen (HLA)-matched related
HSC donor is not available.
TDT is a severe genetic disease caused by mutations in the
β-globin gene that result in reduced or absent hemoglobin (Hb). In
order to survive, people with TDT maintain Hb levels through
lifelong chronic blood transfusions. These transfusions carry the
risk of progressive multi-organ damage due to unavoidable iron
overload.
LentiGlobin 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 HbAT87Q,
which is gene therapy-derived-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 LentiGlobin for TDT were hot flush,
dyspnoea, abdominal pain, pain in extremities and non-cardiac chest
pain. One serious adverse event (SAE) of thrombocytopenia was
considered possibly related to LentiGlobin for TDT.
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.
The conditional marketing authorization for ZYNTEGLO is only
valid in the 28 member states of the EU as well as Iceland,
Liechtenstein and Norway. For details, please see the Summary of
Product Characteristics (SmPC).
The U.S. Food and Drug Administration granted LentiGlobin for
β-thalassemia Orphan Drug status and Breakthrough Therapy
designation for the treatment of TDT.
LentiGlobin for β-thalassemia continues to be evaluated in the
ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more
information about the ongoing clinical studies, visit
www.northstarclinicalstudies.com or
clinicaltrials.gov and use identifier NCT02906202 for
Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).
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 TDT. For
more information visit:
https://www.bluebirdbio.com/medical-professionals/our-clinical-trials/
or clinicaltrials.gov and use identifier NCT02633943 for
LTF-303.
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.
ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of
bluebird bio, Inc.
The full common name for ZYNTEGLO: A genetically modified
autologous CD34+ cell enriched population that contains
hematopoietic stem cells transduced with lentiviral vector encoding
the βA-T87Q-globin gene.
Forward-Looking Statements
This release contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995,
including statements regarding the Company’s views with respect to
the potential for LentiGlobin to treat transfusion-dependent
β-thalassemia and sickle cell disease, the potential for the
bb21217 product candidate to treat relapsed/ refractory multiple
myeloma. 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, the risks that the preliminary
positive efficacy and safety results from our prior and ongoing
clinical trials of our product candidates will not continue or be
repeated in our ongoing or planned clinical trials or in the
commercial context, risks that the current or planned clinical
trials of our product candidates will be insufficient to support
future regulatory submissions or to support marketing approval in
the US and EU, and the risk that any one or more of our product
candidates, will not be successfully developed, approved or
commercialized. 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 Securities and Exchange Commission. All
information in this press release is as of the date of the release,
and bluebird bio undertakes no duty to update this information
unless required by law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191106005511/en/
Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com or Media: Catherine Falcetti,
339-499-9436 cfalcetti@bluebirdbio.com
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