Tr1X Announces FDA Clearance Of First Investigational New Drug Application For TRX103, An Allogeneic Regulatory T-Cell Therapy To Treat Autoimmune Diseases

• Company to initiate Phase 1 study of Graft versus Host Disease (GvHD) prevention in patients undergoing hematopoietic stem cell transplantation in Q2 2024
• Second IND application for refractory Crohn's disease expected in Q3 2024
• Highly scalable platform enables efficient pipeline expansion and opportunity beyond first two indications

San Diego, CA /PRNewswire/ - Tr1X, Inc., an autoimmune and inflammatory disease cell therapy company focused on the development of novel allogeneic regulatory T cell therapies (Allo-Tregs) and allogeneic regulatory T cells expressing Chimeric Antigen Receptors (Allo-CAR Tregs), today announced the U.S. Food and Drug Administration (FDA) accepted the company's Investigational New Drug (IND) application for TRX103 for the prevention of Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched hematopoietic stem cell transplantation (HSCT). The company plans to initiate a Phase 1 study of TRX103, an investigational allogeneic off-the-shelf Tr1 Treg therapy, for this indication in the second quarter of 2024. Additionally, the company is on track to submit an IND for TRX103 for patients with refractory Crohn's disease in the third quarter of 2024.

"The FDA's clearance of our IND for TRX103, the first ever allogeneic engineered Tr1 regulatory T cell product, is an important milestone that could quickly provide us with proof-of-concept data while we continue to develop TRX103 for multiple autoimmune and inflammatory diseases, including Crohn's disease," said Maria Grazia Roncarolo M.D., Co-Founder, President and Head of R&D at Tr1X. "Donor-derived autologous Tr1 cells have shown clinical promise in improving immune reconstitution and reducing GvHD but have limited potential due to lack of feasibility and high cost. TRX103, an off-the-shelf product with unique biological properties compared to other Treg and CAR-T cell therapies, has the potential to reduce inflammation, suppress pathogenic cells, and reset the immune system. TRX103 is currently produced cost effectively at scale in a fully closed end-to-end system using a process that yields billions of cells in a single campaign. This should enable Tr1X to develop further pipeline candidates that address even larger patient populations with equally unmet medical needs."

"Allogeneic stem cell transplantation is the only curative treatment for many advanced blood cancers and genetic and acquired diseases. However, there remains a burden of morbidity and mortality related to GvHD and its complications, including severe infections," said Monzr M. Al Malki, M.D., lead investigator of the Phase 1 study, Associate Professor in the Department of Hematology & Hematopoietic Cell Transplantation, and Director of the Unrelated Donor, Haploidentical and Cord Blood Transplant Programs at City of Hope National Medical Center. "As a result, innovative treatments are urgently needed. We look forward to starting this first-in-human trial to evaluate the safety, tolerability and clinical activity of these allogeneic Tr1 Treg cells and their potential to benefit patients in this setting."

About TRX103
TRX103 is an investigational allogeneic off-the-shelf engineered T cell product generated from CD4+ cells sourced from healthy donors. These donor-derived CD4+ cells are engineered to produce cells that mimic the function of Tr1 regulatory T cells. Tr1X is developing TRX103 for the treatment of several immune and inflammatory disorders. Multiple preclinical models of disease have shown TRX103 to be tolerable and effective and to have the potential to reset immune systems to a healthy state. TRX103 has the potential to overcome major limitations of current cell therapies for autoimmune diseases, which include limited persistence and side effects including cytokine release syndrome (CRS) and neurotoxicity.

About Tr1X
Tr1X is a privately held biotechnology company focused on engineering cures for immune and inflammatory diseases. Founded by industry veterans, including the scientists behind the discovery of Tr1 cells, the company's pipeline of off-the-shelf allogeneic cell therapies is being developed for the treatment of and potential cure of autoimmune diseases with high unmet medical need. The company is backed by leading investors, including The Column Group, NEVA SGR and Alexandria Venture Investments, and has received additional grant support from the California Institute for Regenerative Medicine (CIRM). For more information visit www.tr1x.bio.