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 Contact
Kim Jaffe
Senior Vice President, Head of Business Development & Strategy
+1-609-306-7042
kjaffe@bluespherebio.com

BlueSphere Media Contact
Andrew Mielach
LifeSci Communications
+1-646-876-5868
amielach@lifescicomms.com

Source: BlueSphere Bio