bluebird bio’s Analyst Day Highlights Commercial Path to Patients & Research Engine Focused on Next-Generation Gene & Cell ...
May 09 2019 - 6:30AM
Business Wire
New Collaboration with Seattle Children’s
Research Institute Targets New Immunotherapy Approaches to Acute
Myeloid Leukemia
Phase 1/2 Study for Merkel Cell Carcinoma
Planned with Fred Hutchinson Cancer Research Center
Additional Preclinical Programs Across Both the
Severe Genetic Disease and Oncology Therapeutic Areas Support IND
Goals
Company Will Webcast Live from the Event
Beginning at 8:30 a.m. EDT
bluebird bio, Inc. (Nasdaq: BLUE) today will host an Analyst Day
in New York that will highlight significant progress in the
company’s emerging immuno-oncology and severe genetic disease
pipeline, provide updates on launch expectations for its first gene
therapy product and share key aspects of its long-term growth
strategy. The company also is announcing a new research
collaboration with Seattle Children’s Research Institute in Acute
Myeloid Leukemia (AML), a Phase 1/2 study planned in Merkel Cell
Carcinoma (MCC) with the Fred Hutchinson Cancer Research Center and
programs in Diffuse Large B-cell Lymphoma (DLBCL) and MAGE-A4
positive solid tumors.
“bluebird is at a significant inflection point, with the
potential approval and launch of our first gene therapy product
this year and submissions for regulatory approval for potentially
three additional products through 2022,” said Nick Leschly,
chief bluebird. “We have the opportunity to leverage our gene and
cell therapy expertise across our platform and enable a steep
innovation curve for next-generation products. We are fueled by
what is just the beginning of our efforts to recode the science,
systems and status quo to reach new innovation frontiers and make a
significant impact on patients’ lives.”
Further strengthening its leadership position in developing
transformative first-in-class and best-in-class gene and cell
therapies, bluebird bio will discuss several key milestones and
collaboration updates across its research pipeline, which is
focused on next-generation, disruptive solutions for devastating
diseases. In addition to the two clinic-ready programs planned for
2019, the company is on track to submit 1-2 investigational new
drug applications in 2020 and beyond.
“Relentless innovation is in our DNA at bluebird. Our 1-to-Many
research strategy rapidly integrates and iterates our tools and
technologies across our core platforms of gene editing, gene
addition and cellular immunotherapy, to develop the next generation
of gene and cell therapies with the potential to improve patients’
lives,” said Philip Gregory, D. Phil., chief scientific
officer, bluebird bio. “Our research engine, in partnership with
our network of academic and industry collaborations, is designed to
take on big problems that, if successful, will disrupt our
field.”
Research highlights include:
- AML Research Collaboration with
Seattle Children’s Research Institute: The research
collaboration is intended to address two challenges of tackling
AML, specifically the heterogeneity of the disease as well as the
salvage of normal tissues with the potential for
on-target/off-tumor targeting. Our T cell immunotherapy approach is
expected to leverage technology that enables T cells to target
multiple antigens on the surface of cancer cells as well as
bluebird’s proprietary Dimerizing Agent Regulated Immunoreceptor
Complex (DARIC) platform. By utilizing the DARIC platform in
potential product candidates, we expect to be able to exert
pharmacologic control of CAR T cell activity in vivo, allowing the
investigator to switch on and switch off the activity of the
engineered T cells in the patient as needed by administering a
small molecule drug. Combined with Seattle Children’s world-class
bench-to-bedside expertise in the arena of oncology cell therapies,
the research collaboration’s goal is to rapidly accelerate
development of potential new therapies for patients with AML.
- Phase 1/2 Trial for Merkel Cell
Carcinoma: An academic, proof-of-concept phase 1/2
single-arm study evaluating Merkel Cell Polyomavirus (MCPyV)
TCR-engineered autologous T cells in combination with avelumab
(anti-PDL1) is FDA-approved and in the final initiation stages
of trial approval at the Fred Hutchinson Cancer Research Center for
the treatment of MCC, a rare neuroendocrine
cancer. Exploratory clinical data generated by researchers at
the Fred Hutchinson Cancer Research Center exploring patient
derived MCPyV reactive T cells in combination with PD1 axis
blockade has shown promising depth and durability of
response. Results from the academic phase 1/2 single-arm study
are expected to inform next-generation T cell approaches including
TCR engineering and checkpoint inhibition.
- MAGE-A4: Through its
collaboration with Medigene, bluebird has developed a
next-generation MAGE-A4 TCR expected to enter the clinic for solid
tumors in 2020. This co-receptor-independent TCR candidate has
shown robust anti-tumor activity controlling tumors in a
subcutaneous melanoma xenograft model as a single agent. Moreover,
this highly active TCR can be combined with bluebird’s chimeric
TGF-β receptor signal converter technology to “flip” the
immunosuppressive signals present in the tumor microenvironment
toward T cell stimulation and proliferation. This is the first
collaboration target with Medigene of a potential six TCR products
that the companies have agreed to work on together.
- Diffuse Large B-cell Lymphoma
Candidate: Our DLBCL preclinical program builds on the
knowledge gained from the current generation of CD19-targeting cell
therapies by incorporating multiple next-generation technologies to
potentially address both the depth and durability of response.
Specifically, our potential DLBCL product candidate combines (i)
dual-targeting directed to two novel antigens; (ii) within a unique
CAR construction that is designed to enhance T cell activation; and
(iii) gene editing for potential potency and durability
enhancements, all in a single product candidate.
- Mucopolysaccharidosis (MPSI):
Our MPSI program is focused on the severe form of MPSI, an
ultra-rare metabolic condition that causes severe neurologic
impairment and organ damage, also referred to as Hurler Syndrome.
In our academic collaboration with the University of Minnesota, we
expect to leverage key learnings from our hematopoietic stem cell
lentiviral vector (HSC LVV) platform technology to deliver
gene-modified cells that can potentially cross the blood-brain
barrier and express high and sustained levels of therapeutic
enzyme. Preclinical data in the MPS1 mouse model demonstrates full
molecular correction of the disease across all critical organs
impacted by the disease, including the brain, following
administration of an HSC LVV-gene-modified stem cell product. These
robust preclinical data support the potential clinical application
of this product candidate.
Webcast
To access the live webcast of bluebird bio’s Analyst Day
presentation, please visit the “Events & Presentations” page
within the Investors & Media section of the bluebird bio
website at http://investor.bluebirdbio.com. A replay of the
webcast will be available on the bluebird bio website for 90 days
following the meeting.
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 by researching cerebral adrenoleukodystrophy, sickle cell
disease, transfusion-dependent 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.
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 plans for the potential
commercial launch of the Company’s product candidates, as well as
the advancement of the Company’s research and development plans.
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 risk that the 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; 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 U.S. and EU; the risk that our collaborations with
third parties will not be successful; the risk that our planned
clinical studies may be delayed or not be initiated at all; and the
risk that 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190509005311/en/
Investors & Mediabluebird bioInvestors:Elizabeth
Pingpank, (617) 914-8736epingpank@bluebirdbio.comMedia:Jenn Snyder,
(617) 448-0281jsnyder@bluebirdbio.com
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