BSB-1001

Through this partnership, BlueSphere will treat patients with acute myeloid and lymphocytic leukemia, and myeloid dysplastic syndrome, who have active disease or are at high risk for relapse, with the goal of reducing the rate of relapse and graft versus host disease NMDP BioTherapies will provide bioinformatics consulting to help identify donors and will manage supply chain logistics to deliver healthy, consented, and compliant donor hematopoietic stem cells from HLA-matched donors that meet Food and Drug Administration and BlueSphere eligibility requirements June 25, 2024 -- 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.” ueSphere 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. 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. 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. The content above comes from the network. if any infringement, please contact us to modify.

PITTSBURGH, June 04, 2024 (GLOBE NEWSWIRE) -- BlueSphere Bio, a T-cell receptor (TCR) T-cell therapy company utilizing a powerful TCR discovery platform to identify novel therapeutic candidates for patients with hematologic malignancies and solid tumors, today announced a strategic collaboration with the National Cancer Institute (NCI), part of the National Institutes of Health. The collaboration will seek to advance a novel TCR T-cell therapy targeting recurrent respiratory papillomatosis (RRP), a rare orphan disease. Under the terms of a Cooperative Research and Development Agreement (CRADA), BlueSphere will collaborate with the Center for Cancer Research (CCR) at the NCI. The clinical studies contemplated under the CRADA will be conducted under the leadership of Clint Allen, M.D. and Scott Norberg, D.O. at the CCR, NCI. An earlier Material Transfer Agreement had granted BlueSphere access to HPV+ tumor samples collected by the NCI, with which BlueSphere leveraged its proprietary high-throughput TCR discovery platform, TCXpress™, to identify multiple TCRs with a high affinity for human papilloma virus (HPV) 6 and 11, strains of the virus intricately linked to RRP. "This strategic collaboration with the National Cancer Institute represents a significant milestone for BlueSphere that allows us to expand the potential of the TCRs identified with our TCXpress™ platform beyond our initial oncology programs in leukemia," said Keir Loiacono, Esq., Chief Executive Officer of BlueSphere. "With the combined expertise of Drs. Allen and Norberg and the extensive resources of the NCI, we are well-positioned to explore the vast potential of the TCRs identified with our TCXpress™ platform as an innovative treatment pathway for RRP. This collaboration provides us with a unique opportunity to demonstrate proof-of-concept efficacy of the TCRs identified with our TCXpress™ platform in a novel therapeutic domain. Notably, unlike solid tumors, RRP presents distinctive challenges without the constraints of the tumor microenvironment, presenting a promising avenue for therapeutic intervention." Dr. Norberg added, "This collaboration aligns with our mission of developing novel therapies for patients suffering with RRP. This collaboration seeks to determine whether TCR-T cell therapy has the potential to eliminate the underlying cause of the disease, rather than simply treating symptoms that result from this condition. This novel approach requires a shared commitment to innovation, underscoring the potential transformative impact this collaboration aims to achieve." RRP is a disease most commonly caused by two strains of HPV: HPV 6 and HPV 11, whereby benign papillomas grow in a patient’s air passages leading from the nose and mouth into the lungs1. In certain patients, these papillomas can be debilitating and difficult to treat, particularly when found within the lungs. The focus of the collaboration will be to prepare the identified TCRs for clinical studies and to investigate the efficacy of the resulting clinical-grade TCRs in a future early-stage clinical trial targeting RRP. Per the terms of the CRADA, the contemplated clinical trial may be sponsored by the NCI or BlueSphere. BlueSphere will provide support and transfer the TCR sequences to the NCI, while the NCI will conduct all testing, sequence modifications, process development, manufacturing, and clinical trial activities. About BlueSphere Bio 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). Under its TCX-101 program, BlueSphere anticipates enrolling its first patient in 2024 for treatment with its first-in-human candidate, BSB-1001. BSB-1001 is a TCR T cell therapy targeting the minor histocompatibility antigen-1 (HA-1) to be used in conjunction with allogeneic hematopoietic stem cell transplantation (alloSCT). 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. 1 https://www.nidcd.nih.gov/health/recurrent-respiratory-papillomatosis BlueSphere ContactKim JaffeSenior Vice President, Head of Business Development & Strategy+1-609-306-7042kjaffe@bluespherebio.com BlueSphere Media ContactAndrew MielachLifeSci Communications+1-646-876-5868amielach@lifescicomms.com