AURORA, Colo., and ZAGREB, Croatia, Nov. 10, 2021 /PRNewswire/ -- Taiga Biotechnologies, a cell-based immunotherapy company, announced today that the Company has completed the site initiation session for the first site to participate in the TBX-2400-01 clinical trial, a Phase 1/2 study to evaluate the safety and early efficacy of TBX-2400 in acute myeloid leukemia (AML) and myelofibrosis patients undergoing hematopoietic stem cell transplant (HSCT), in Croatia.
Taiga's platform technology, TBX-4000 uses the MYC protein to regulate cellular function. When exposed to immune cells, TBX-4000 delivers MYC across the cell membrane directly to the nucleus where MYC drives the activation and proliferation of the cell. Hematopoietic stem cells (HSCs) derived from either bone marrow or GCSF-mobilized apheresis material treated with TBX-4000 form a new product designated as TBX-2400. TBX-2400 is an allogeneic stem cell therapy that improves the rate of engraftment and reconstitution of the immune system for patients receiving a bone marrow transplant.
Taiga has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), Rare Pediatric Disease Designation from the FDA and Advanced Therapy Medicinal Product Designation from the EMA.
"The initiation of this study, under the aegis of the Croatian Ministry of Health and the EMA, represents a significant milestone for our TBX-2400 clinical development program," said Dr. Yosef Refaeli, Chief Executive Officer of Taiga Biotechnologies. "We believe TBX-2400 has the potential to dramatically change the HSCT market by reducing the risk and increasing the success rate of HSCT, reducing time and cost by as much as 50%, thereby, turning it into a commonplace procedure. In addition, TBX-2400 requires 1000x fewer cells than current procedures, which is expected to eliminate the supply-demand issues that currently limit the number of procedures performed and should increase the number of eligible procedures for acute cases, such as leukemia and lymphoma. Importantly, it has the potential to treat other immune-based diseases, such as diabetes and multiple sclerosis." This clinical study will be managed by Optimapharm, a full service European CRO headquartered in Zagreb, Croatia.
"We hope to positively impact difficult to perform HSCT transplants with the TBX-2400 program. We are encouraged by earlier pre-clinical data that support TBX-2400's ability to enhance homing to the specialized HSC niches in the bone marrow as well as to improve engraftment," said Brian C. Turner, P.D., Co-founder, President and Chief Scientific Officer of Taiga Biotechnologies.
"We are keen to test the ability of HSCTs treated with TBX-2400 in the context of adult bone marrow failure syndrome as well as in hematologic oncology indications in this study as it holds the promise to significantly enhance and improve outcomes for these very sick patients who are faced with limited treatment options," said Nadira Durakovic, M.D., Associate Professor at the University of Zagreb School of Medicine and University Hospital in Croatia and a principal investigator of the study.
TBX-2400-01 is a Phase I study to assess the safety and early efficacy of TBX-2400 in enhancing engraftment in patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) for the treatment of acute myelogenous leukemia or myelofibrosis. This is an interventional clinical trial that seeks to recruit 10-20 patients in 3 clinical sites (The University Hospital in Zagreb, The Hadassah Medical Center in Jerusalem, Israel, and the Rambam Medical Center in Haifa, Israel). The primary endpoint relates to the incidence of HSCT related severe adverse events as well as the frequency of successful transplant engraftment. In addition, the secondary endpoints include specific assessments of immune cell function following transplantation and engraftment.
More information on this clinical trial can be found at https://clinicaltrials.gov/ct2/show/NCT04709458?term=taiga+biotechnologies&draw=2&rank=1.
Taiga has developed a proprietary recombinant protein that can passively enter cells without the need for binding and uptake by a cell surface receptor, which can transiently alter intracellular levels of a critical protein involved in survival and proliferation called "MYC." Hematopoietic stem cells (HSCs) derived from either bone marrow or GCSF-mobilized apheresis material treated with TBX-4000 form a new product designated as TBX-2400. The treatment of HSCs with TBX-4000 leads to improved homing to the specialized HSC stem cell niches in the bone marrow as well as improved engraftment. The TBX-2400-01 clinical trial aims to test the safety and early efficacy of TBX-2400 cells in adults who suffer from myelofibrosis or relapsed/refractory acute myeloid leukemia. TBX-2400 has been awarded Orphan Disease Designation by the U.S. FDA and EMA. In addition, this program has also been awarded Rare Pediatric Disease designation by the U.S. FDA.
About Taiga Biotechnologies, Inc.
Taiga Biotechnologies, Inc. is a clinical stage biotechnology company focused on harnessing the power of the immune system to fight solid tumors and infectious disease. Taiga has an additional program related to bone marrow stem cell transplantation as well as a broad pre-clinical research and development pipeline. For more information, please visit the company's website (www.taigabiotech.com).
Forward looking statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of Taiga Biotechnologies' product candidates, including TBX-3400; its plans and timelines for the clinical development of TBX-3400; and the benefit of its strategic plans and focus. The words "expect," "intend," "may," "plan," "potential," "hope," "could," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Taiga Biotechnologies' current expectations and beliefs. For example, there can be no guarantee that TBX-3400 or any other product candidate Taiga Biotechnologies is developing will successfully complete necessary clinical development phases, or that development of such product candidates will successfully continue. There can be no guarantee that any positive developments in Taiga Biotechnologies' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including: Taiga Biotechnologies' results of clinical trials and pre-clinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Taiga Biotechnologies' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; and Taiga Biotechnologies' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing. Any forward-looking statements contained in this press release speak only as of the date hereof, and Taiga Biotechnologies expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
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SOURCE Taiga Biotechnologies, Inc.