BlueSphere Bio To Partner with NMDP BioTherapies℠ for the Company’s First-In-Human TCR-based Product Candidate in Combination with Allogeneic Hematopoietic Cell Transplantation Trial
26 June 2024 - 12:00AM
BlueSphere Bio, a drug development company focused on novel T-cell
receptor (TCR) based therapies for oncology and non-oncology
indications, today announced a collaboration with NMDP
BioTherapies℠ (formerly Be The Match BioTherapies), an organization
offering solutions for companies developing and commercializing
cell and gene therapies, to advance BlueSphere’s first clinical
trial evaluating a novel cell therapy for patients with high-risk
leukemias. Under the partnership, NMDP will provide HLA-matched
healthy donor hematopoietic stem cells that meet Food and Drug
Administration (FDA) and BlueSphere’s eligibility requirements for
the company’s upcoming, FDA-cleared Phase 1/2a trial (TCX-101) of
its first-in-human (BSB-1001) product candidate for patients with
relapsed or refractory acute myeloid leukemia (AML), acute
lymphocytic leukemia (ALL), and myeloid dysplastic syndrome (MDS),
in conjunction with allogeneic hematopoietic stem cell
transplantation (alloHSCT). BSB-1001 is a TCR T-Cell therapy
product candidate that will be delivered simultaneously with an
allogeneic stem cell transplant in patients with active disease or
a high risk of relapse.
“We are excited to partner with BlueSphere Bio
to help bring the BSB-1001 therapy to physicians and patients,”
said Dr. Steve Devine, Chief Medical Officer at NMDP and Associate
Scientific Director, CIBMTR®. “BlueSphere Bio’s BSB-1001 therapy
could provide a novel and effective way to treat patients with AML,
ALL, and MDS in combination with allogeneic stem cell transplant
and prevent relapse, furthering our mutual goal to save more
lives.”
In standard of care alloHSCT, T cells from the
donor can attack and kill not only the patient’s malignant cells
but also healthy cells. This graft versus host disease (GvHD)
phenomena can be life threatening, even when the tumor has been
eliminated or is under control as a result of the alloHSCT.
BSB-1001 is an engineered TCR T cell therapy that targets the
blood-restricted antigen, HA-1. BlueSphere will provide high-risk
leukemia patients with a T cell depleted stem cell transplant
alongside the engineered BSB-1001 T cells. Because the patient will
not receive any non-HA-1 targeting T cells during the therapy, this
allows the HA-1 specific T cells to kill any remaining leukemia
cells without targeting healthy tissues in the patient (GvHD). This
should reduce the risk of GvHD while potentially enhancing the
graft versus leukemia effect. Notably, because of the unique
product and trial design, the TCX-101 trial will enroll patients
with active morphologic disease or features placing them at high
risk of relapse. Additionally, patients will receive simultaneous
administration of BSB-1001 with alloHSCT to optimally target
residual leukemia, without the use of immunosuppressive drugs.
NMDP BioTherapies will provide allogeneic stem
cells from healthy donors who are HLA matched to patients enrolled
in TCX-101. This includes bioinformatics consulting to identify
available donors from the NMDP donor registry who meet specific HLA
and other criteria. NMDP BioTherapies will also oversee the consent
and workup of donors and will manage the supply chain from
apheresis collection through delivery to BlueSphere Bio’s
manufacturing and clinical sites under a rigorous quality
system.
“NMDP BioTherapies offers everything we need to
provide our team, and ultimately patients, with critical donor
material required for making the different components of our
therapy. Its large, diverse registry of volunteer donors, robust
supply chain, quality infrastructure, and critical bioinformatics
consulting are the ideal solution for meeting our specific donor
requirements,” said Keir Loiacono, Esq., Chief Executive Officer,
BlueSphere Bio. “We are confident our partnership with NMDP
BioTherapies will help deliver this potentially life-saving therapy
to leukemia patients who have run out of meaningful treatment
options.”
BlueSphere Bio anticipates enrolling its first
patient for this trial in 4Q2024.
For more information visit NMDP BioTherapies and
BlueSphere Bio.
About NMDP
BioTherapies
NMDP BioTherapies is
the only cell and gene therapy solutions provider with customizable
services to support the end-to-end cell therapy supply chain.
Backed by the industry-leading experience of NMDP℠ and a
research partnership with the CIBMTR® (Center for
International Blood and Marrow Transplant Research®), the
organization designs solutions that advance the development of cell
and gene therapies across the globe.
NMDP BioTherapies is
dedicated to accelerating patient access to life-saving cell and
gene therapies by providing high-quality cellular source material
from the NMDP Registry, the world’s most diverse registry of more
than 7 million potential blood stem cell donors. Through
established relationships with apheresis, marrow collection, and
transplant centers worldwide, the organization develops, onboards,
trains, and manages expansive collection networks to advance cell
therapies. NMDP BioTherapies uses a proven infrastructure
consisting of regulatory compliance and managed logistics experts
and cell therapy supply chain case managers to successfully
transport and deliver regulatory-compliant life-saving therapies
across the globe. Through the CIBMTR, NMDP extends services beyond
the cell therapy supply chain to include long-term follow-up
tracking for FDA-approved CAR-T therapies.
For more information, follow NMDP
BioTherapies on LinkedIn or X.
About BlueSphere
BlueSphere Bio is the first translational
sciences stand-alone company formed by UPMC Enterprises, the
innovation, commercialization, and venture capital arm of the
Pittsburgh-based health system. BlueSphere Bio was founded upon the
proprietary TCR discovery platform – TCXpress™, designed to isolate
and functionally characterize TCRs with speed and efficiency. This
platform can be used in various clinical settings to discover TCRs
for use in either TCR-based cell therapy or in antibody-like
molecules, such as bi-specific T cell engagers. While the Company’s
initial focus is oncology, the platform could be deployed in other
therapeutic areas.
BlueSphere is leveraging the breadth and depth
of TCXpress™ to develop a portfolio of TCR-based assets with an
initial clinical focus on acute myeloid leukemia (AML), acute
lymphocytic leukemia (ALL) and myeloid dysplastic syndrome (MDS).
BSB-1001 is a TCR T cell therapy targeting the minor
histocompatibility antigen-1 (HA-1) and is the first clinical
candidate in the Company’s TCX-101 clinical program. Under the
TCX-101 clinical program, BSB-1001 will be dosed simultaneously
with allogeneic hematopoietic stem cell transplant. BlueSphere
anticipates enrolling its first patient in the TCX-101 program in
4Q2024. Using TCXpress™, the company has also discovered and
nominated three additional miHA targeting TCRs for clinical
development, all for use in the same clinical setting as BSB-1001.
These additional miHA candidates will become part of the TCX-101
Program as a panel that positions the Company with best-in-class
HLA coverage in these hematologic indications.
In addition to TCX-101, BlueSphere has further
broadened its AML therapy pipeline to address an additional subset
of patients by discovering and nominating a lead TCR reactive
against mutant NPM-1 for the TCX-102 program. The TCX-102 program
will be autologous and not given in combination with stem cell
transplant. An IND is expected in the second quarter of 2025.
Finally, in addition to a strategic
collaboration with the National Cancer Institute to advance a novel
TCR T-cell therapy targeting the rare orphan disease, recurrent
respiratory papillomatosis (RRP), BlueSphere is identifying a broad
panel of TCRs to deploy in its own first solid tumor program, which
will be announced later this year.
Media Contacts for NMDP BioTherapies:
Matt
MayrInfo@nmdpbiotherapies.com(800) 471-4431
BlueSphere Bio Contacts
Company Contact: Kim JaffeSenior Vice President, Head of
Business Development &
Strategy+1- 609-306-7042kjaffe@bluespherebio.com
Media Contact:Andrew MielachLifeSci
Communications+1-646-876-5868amielach@lifescicomms.com