BSB-1001 / BlueSphere Bio - LARVOL DELTA

BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS (clinicaltrials.gov) - P1/2 | N=2 | Terminated | Sponsor: BlueSphere Bio, Inc | N=38 ➔ 2 | Trial completion date: Mar 2029 ➔ Sep 2026 | Active, not recruiting ➔ Terminated | Trial primary completion date: Feb 2029 ➔ Sep 2026; Company Decision

Enrollment change • First-in-human • Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Myelodysplastic Syndrome • Transplantation • TP53

BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS (clinicaltrials.gov) - P1/2 | N=38 | Active, not recruiting | Sponsor: BlueSphere Bio, Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • First-in-human • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Myelodysplastic Syndrome • Transplantation • TP53

BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS (clinicaltrials.gov) - P1/2 | N=38 | Recruiting | Sponsor: BlueSphere Bio, Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Myelodysplastic Syndrome • Transplantation

Phase 1/2a Ascending Dose Study to Evaluate the Safety BSB-1001 in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS (clinicaltrials.gov) - P1/2 | N=38 | Not yet recruiting | Sponsor: BlueSphere Bio, Inc

New P1/2 trial • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Myelodysplastic Syndrome • Transplantation

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 (GlobeNewswire) - "BlueSphere Bio...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)."

Licensing / partnership • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

BlueSphere Bio Announces IND Clearance of its First in Human Candidate and New Cell Therapy Portfolio for High-Risk Leukemia Patients (GlobeNewswire) - "Today, BlueSphere Bio...announced that the Food and Drug Administration (FDA) has cleared BlueSphere’s Investigational New Drug application (IND) for a 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). The Company anticipates enrolling the first patient in this multi-center, open-label study in 4Q2024. Notably, because of the unique product and trial design, the TCX-101 trial will enroll patients with active morphologic disease or cytogenetic 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."

IND • New P1/2 trial • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Myelodysplastic Syndrome

BlueSphere Bio to Present Corporate Update and Participate in a Panel Discussion at the 2024 BIO International Convention (GlobeNewswire) - "The corporate update will highlight BlueSphere’s progress towards its first clinical program in high-risk leukemia and clinical development efforts of its first-in-human candidate, BSB-1001."

Clinical • Hematological Malignancies • Leukemia • Oncology

BlueSphere Bio to Present at the Next Generation CAR & T Cell Therapies Conference (GlobeNewswire) - "BlueSphere Bio...today announced Robert Keefe, Ph.D., Chief Development Officer of BlueSphere Bio, will present an overview of the Company’s anticipated first in human clinical candidate, BSB-1001, and its discovery using the TCXpress™ platform at the Next Generation CAR & T Cell Therapies Conference held in San Diego, CA from February 27 – March 3, 2023....BSB-1001...is the first TCR-based clinical candidate generated using TCXpress and is in development for the treatment of acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myeloid dysplastic syndrome (MDS) in conjunction with allogeneic hematopoietic stem cell transplantation (alloSCT). BlueSphere anticipates filing its first Investigational New Drug Application (IND) for BSB-1001 in 2023."

Clinical • IND • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

A Phase 1/1b Multicenter Ascending Dose Study to Evaluate the Safety of HA-1 Minor Histocompatibility Antigen-Reactive TCR-Modified T Cells (BSB-1001) in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant (alloSCT) for MRD+ AML or ALL or High/Very High Risk MDS (TCT-ASTCT-CIBMTR 2023) - "Enrollment is anticipated to begin in 2Q2023. BSB is developing a panel of TCRs targeting additional hematopoietically-restricted miHAs."

Clinical • IO biomarker • P1 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD34 • CD8 • HLA-A

A Phase 1/1b Multicenter Ascending Dose Study to Evaluate the Safety of HA-1 Minor Histocompatibility Antigen-Reactive TCR-Modified T Cells (BSB-1001) in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant (alloSCT) for MRD+ AML or ALL or Poor/Very Poor Risk MDS (ASH 2022) - "The anti-HA-1 T cell product (BSB-1001) is composed of donor CD8+ T cells transduced with a lentivirus vector encoding the anti-HA-1 TCR and the RQR8 tag, which expresses an epitope from CD20, thereby enabling the in vivo killing of BSB-1001 cells with rituximab. Enrollment is anticipated to begin in 1Q2023. BSB is also developing panels of TCRs targeting additional relatively hematopoietically-restricted miHAs with prioritization based on the frequencies of the restricting HLAs and the immunogenic/nonimmunogenic miHA alleles."

Clinical • IO biomarker • P1 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Inflammation • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD20 • CD34 • CD8 • CSF3 • HLA-A

Manufacture of allogeneic, HLA-matched, TCR-edited T-cell therapy reactive against minor histocompatibility antigen 1 to treat Acute Myeloid Leukemia in combination with CD34 HSCT with the potential for high potency and durability (SITC 2022) - "Conclusions Taken together, the results indicate that BSB-1001 is an active, highly potent drug product candidate, with a potential to be effective in treating HA-1+ HLA-A*02:01 AML patients in the setting of alloSCT. The phenotype and high cytolytic bioactivity indicate that the clinical response has a potential to be very potent and durable."

Combination therapy • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • CD34 • CD69 • CD8 • HLA-A

BlueSphere Bio Presents Data Demonstrating the Potential of Lead Product Candidate at 37th Society for Immunotherapy of Cancer (SITC) Annual Meeting (GlobeNewswire) - "BSB-1001 is a highly potent cell therapy with the potential to be an effective in treatment for patients with AML undergoing alloSCT. BSB-1001 demonstrated robust cancer cell killing in vitro, even at low doses relative to target cells. Genetic manipulations to remove the endogenous TCR had no impact on BSB-1001, supporting the manufacturing process’ ability to generate a high-quality product."

Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

BlueSphere Bio Data to be Presented at the 37th Society for Immunotherapy of Cancer (SITC) Annual Meeting (GlobeNewswire) - "BlueSphere Bio...announced that Robert Keefe, Ph.D., Chief Development Officer of BlueSphere Bio, will present data in a poster at the 37th Society for Immunotherapy of Cancer (SITC) Annual Meeting, being held virtually and in Boston, MA from November 8-12, 2022."

Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology