FDA green light for trial of allogeneic regulatory T cell therapy

Stem cells

The US Food and Drug Administration (FDA) has accepted an Investigational New Drug (IND) application for Tr1X’s TRX103 for the prevention of Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched haematopoietic stem cell transplantation (HSCT).

The company plans to initiate a Phase I study of the investigational allogeneic off-the-shelf Tr1 Treg therapy for this indication in the second quarter of 2024. Tr1X has also revealed plans 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,” said Maria Grazia Roncarolo, Co-Founder, President and Head of R&D, 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.”

“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 Al Malki, lead investigator of the Phase I 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.”

Diana Spencer, Senior Digital Content Editor, DDW

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