ARHGAP45

BlueSphere accepted as a virtual member of Johnson & Johnson Innovation – JLABS, a premier global network of health incubatorsPITTSBURGH, Aug. 14, 2024 (GLOBE NEWSWIRE) -- BlueSphere Bio, a drug development company focused on the discovery of novel T-cell receptor (TCR) based therapies, announced it has been accepted as a virtual member of Johnson & Johnson Innovation - JLABS. JLABS is a global network of open innovation ecosystems, enabling and empowering innovators to create and accelerate the delivery of life-enhancing healthcare solutions to patients around the world. “We are very excited to join JLABS,” said Chief Executive Officer Keir Loiacono, Esq. “At BlueSphere, we have a staged development strategy to bring the next generation of TCR-based therapies to the clinic as quickly and efficiently as possible. Being part of JLABS will help us achieve that goal.” BlueSphere uses their proprietary TCXpressTM platform to identify novel TCRs for known and unknown antigens relevant to hematologic malignancies, solid tumors, and rare disease. Using TCXpress, BlueSphere has built a differentiated and stepwise clinical development strategy for TCR-based therapeutics, starting in hematologic malignancies. For their first-in-human study, the company has recently received a letter to proceed from the FDA to treat high-risk AML, ALL, and MDS patients in conjunction with CD34+ stem cell transplant. This TCR T-cell therapy candidate (BSB-1001) targets the minor histocompatibility antigen HA-1. Expanding upon this lead program, the company has three additional TCR candidates ready for product development that can be used to expand HLA population coverage and the addressable market size in this high-risk leukemia space. In addition, BlueSphere recently announced their TCX-102 program, an autologous TCR-T cell therapy that will treat AML patients with mutated NPM-1, a foundational mutation commonly found in this indication. An IND is expected in Q2 of 2025 for the TCX-102 program. Together, these five TCR-T candidates give BlueSphere a uniquely broad portfolio of cell therapies to address significant unmet medical needs in hematologic oncology. Looking beyond these hematologic programs, BlueSphere has recently announced a strategic collaboration with the NCI in recurrent respiratory papillomatosis (RRP), a rare orphan disease. BlueSphere will provide validated HPV-specific TCRs to the experts at NCI, who will then manufacture the TCR-T cell product and administer it under an NCI-sponsored clinical trial. 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. Company ContactKim JaffeSenior Vice President, Head of Business Development & Strategy+1- 609-306-7042kjaffe@bluespherebio.com Media ContactAndrew MielachLifeSci Communications+1-646-876-5868amielach@lifescicomms.com

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

TCX-101 Clinical Program: TCR T-Cell therapy product candidate (BSB-1001) to be delivered simultaneously with allogeneic stem cell transplant in patients with active disease or high risk of relapse First patient to be enrolled in 4Q2024 Nomination of Three additional novel TCR candidates expand HLA population coverage and addressable market size TCX 102 Development Program: Autologous TCR-based cell therapy program will target AML patients with mutated NPM-1 IND anticipated 2Q2025 PITTSBURGH, June 03, 2024 (GLOBE NEWSWIRE) -- Today, BlueSphere Bio, a drug development company focused on the discovery of novel T-Cell receptor (TCR) based therapies, 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. BSB-1001 targets HA-1, a blood-restricted minor histocompatibility antigen (miHA) and is the first TCR-T cell therapy candidate generated using the Company’s TCXpress™ platform. TCXpress™ is an integrated, high-throughput TCR identification and screening platform that enables efficient TCR discovery with a competitive timeline. While BlueSphere is also developing assets for solid tumors and non-oncology indications, it has chosen to prioritize its high-risk leukemia programs in the clinic. Simultaneously, BlueSphere also announced three additional blood-restricted miHA TCR product candidates that are ready for clinical development – all discovered utilizing the TCXpress™ platform. Together, these four TCR T-cell therapy candidates comprising the TCX-101 program positions the Company’s miHA portfolio with best-in-class HLA coverage in these hematologic indications. “We are eager to begin treating patients in our TCX-101 trial,” said Keir Loiacono, Esq., Chief Executive Officer of BlueSphere. “Our team is focused on accelerated identification, development and translation of novel products to the clinic. While alloHSCT provides a chance of a cure for patients, a large fraction of patients will still relapse or do not have it as an option because of active leukemia. Deploying a TCR-based approach to blood restricted antigens - like miHAs combined with alloHSCT - could provide new options for these patients. We believe that BlueSphere’s unique approach to product and trial design will, among other things, enable us to treat patients who previously were unable to receive alloHSCT because of the presence of active leukemia.” To further expand the reach of our TCR-based portfolio, BlueSphere has broadened its pipeline to address an additional subset of AML patients having mutated NPM1. Utilizing TCXpress™, the Company identified and subsequently nominated a single lead TCR reactive against mutant NPM-1 for the TCX-102 program. As a testament to the power of our TCXpress™ platform and ability to accelerate translation to the clinic, the Company screened close to 700 million T cells and was able to select three (3) lead candidates in under six months, ultimately nominating a single TCR for clinical development. IND enabling work is underway with an IND filing anticipated in 2Q2025. The TCX-102 trial will be an autologous program and will not be given in combination with alloHSCT. “The NPM1 mutation is a founder mutation present in approximately 30% of AML patients and is present throughout the duration of the disease, making it an ideal target for TCR-based therapy,” said Erkut Bahceci, MD, Chief Medical Officer of BlueSphere. “The advancement of the TCX-102 program in patients with mutant NPM1 demonstrates our commitment to this disease space and underscores the power of our TCR discovery platform to discover rare and potent TCR candidates.” 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. Company Contact Kim Jaffe Senior Vice President, Head of Business Development & Strategy +1- 609-306-7042 kjaffe@bluespherebio.com Media Contact Andrew Mielach LifeSci Communications +1-646-876-5868 amielach@lifescicomms.com