FDA Clears Tr1X's IND for TRX103, a Tr1 Treg Therapy for Refractory Crohn’s Disease

SAN DIEGO, Aug. 07, 2024 – Tr1X, a biopharmaceutical company focusing on allogeneic engineered Treg and CAR-Treg cell therapies to potentially cure autoimmune and inflammatory diseases, revealed that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for TRX103. This engineered Tr1 Treg cell therapy is aimed at addressing treatment-refractory Crohn’s Disease. Currently, TRX103 is being evaluated in a Phase 1/2a clinical trial on patients who are undergoing hematopoietic stem cell transplant (HSCT) for hematological disorders.

TRX103 represents a pioneering Type 1 Treg (Tr1) cell therapy, explicitly intended to target the gut, mitigate inflammation, promote tissue repair, and restore immune balance in individuals with inflammatory bowel diseases like Crohn’s Disease. This innovative therapy is designed to fulfill the significant unmet needs of patients who have not responded to multiple targeted treatments and have limited therapeutic options left.

Dr. Florian Rieder, Vice Department Chair and Co-Director of the Inflammatory Bowel Diseases (IBD) Section at the Cleveland Clinic, commented on the breakthrough. “Despite significant progress in biological and targeted therapies for Crohn’s disease, a substantial number of our patients do not achieve remission or experience relapses. This indicates a therapeutic ceiling for existing anti-inflammatory treatments, necessitating new approaches,” he said. “TRX103 is a unique and promising Tr1 Treg cell therapy combining anti-inflammatory and immunomodulatory properties. It has the potential to reset the immune system and enhance long-term outcomes for these patients.”

Dr. Maria Grazia Roncarolo, President and Head of R&D at Tr1X, emphasized the importance of Tr1 Tregs. “Tr1 Tregs were initially validated in preclinical studies involving animal models of inflammatory bowel disease, demonstrating crucial anti-inflammatory and immunomodulatory effects,” she noted. “Testing these allogeneic engineered Tr1 cells for Crohn’s disease is a significant milestone, underlining their critical role in restoring and maintaining gut homeostasis for patients globally.”

The forthcoming Phase 1/2a study for TRX103 will be a dose-escalation and confirmation trial designed to assess the safety, tolerability, and clinical efficacy of TRX103. This multi-center trial, conducted in the US, will enroll patients with moderate to severe Crohn’s Disease who have not responded to two or more advanced therapies.

TRX103 capitalizes on the unique capabilities of Tr1 cells, such as their ability to migrate to inflamed tissues and lymph nodes, reduce inflammatory cytokines, regulate harmful T cell responses, and foster immune tolerance. Unlike other cell therapies like effector CAR-T cells, TRX103 Treg cells aim to treat autoimmune diseases with fewer side effects and potentially without requiring lymphodepletion. These cells are naturally protected from the immune system, enhancing their durability and minimizing dangerous immune responses.

About TRX103

TRX103 is an investigational allogeneic off-the-shelf engineered T cell product derived from CD4+ cells from healthy donors. These cells are engineered to act like Tr1 regulatory T cells, known as TRX cells. Tr1X is developing TRX103 for treating various immune and inflammatory disorders. Preclinical studies have shown TRX103 to be both tolerable and effective, with the potential to reset the immune system to a healthy state. TRX103 aims to surpass the significant limitations of current cell therapies for autoimmune diseases, which often include limited persistence and side effects such as cytokine release syndrome (CRS) and neurotoxicity.

About Tr1X

Tr1X is a private biotechnology company dedicated to curing immune-mediated and inflammatory diseases. Founded by industry pioneers, including the scientists who discovered Tr1 cells, the company is developing a range of off-the-shelf allogeneic cell therapies to treat and potentially cure autoimmune diseases with high unmet medical needs. Tr1X is backed by prominent investors and has received additional grant support from the California Institute for Regenerative Medicine (CIRM).

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