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Tr1X Plans to Initiate a Phase 1/2a study and Dose the First Patient by the End of 2024SAN DIEGO, Aug. 07, 2024 (GLOBE NEWSWIRE) -- Tr1X, Inc. (pronounced “Trix”), a biopharmaceutical company specializing in allogeneic engineered Treg and CAR-Treg cell therapies with the potential to cure autoimmune and inflammatory diseases, announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for TRX103, an engineered Tr1 Treg cell therapy for treatment-refractory Crohn’s Disease. TRX103 is currently being tested in a Phase 1/2a trial in patients undergoing hematopoietic stem cell transplant (HSCT) for hematological malignancies. TRX103, a first-in-class Type 1 Treg (Tr1) cell therapy, is designed to home to the gut, reduce inflammation, promote tissue healing and repair, and reset immune homeostasis in patients with inflammatory bowel diseases, including Crohn’s. This therapy aims to address the high unmet need in patients who have failed multiple targeted therapies and have limited treatment options. “Despite the advances in biological and targeted therapies in Crohn’s disease, many of our patients do not achieve remission or relapse on treatment. This suggests a therapeutic ceiling for current anti-inflammatory therapies and novel approaches are needed,” said Florian Rieder, MD, Vice Department Chair and Co-Director of the Inflammatory Bowel Diseases (IBD) Section at the Cleveland Clinic. “TRX103 is a novel and exciting Tr1 Treg cell therapeutic that combines anti-inflammatory and immunomodulating activities. It has the potential to reset the immune system to improve long-term outcomes for these patients.” “Tr1 Tregs were first validated preclinically in animal models of inflammatory bowel disease, where they demonstrated their important anti-inflammatory and immunomodulatory effects,” said Maria Grazia Roncarolo, MD, President and Head of R&D of Tr1X. “It is a full-circle moment for us to be able to test these allogeneic engineered Tr1 cells for the first time in the setting of Crohn’s disease, and to prove their importance in restoring and maintaining gut homeostasis for the benefit of patients worldwide.” The Phase 1/2a study for TRX103 will be a dose-escalation and confirmation study designed to evaluate the safety, tolerability, and clinical activity of TRX103. This multi-center, US-based trial will enroll subjects with moderate to severe Crohn’s Disease who have failed two or more advanced therapies. TRX103 leverages the unique properties of Tr1 cells, including their ability to traffic to inflamed tissues and lymph nodes, lower inflammatory cytokines, control harmful T cell responses, and create immune tolerance. Unlike other cell therapies such as effector CAR-T cells, TRX103 Treg cells aim to treat autoimmune diseases with fewer side effects and potentially without the need for lymphodepletion. These cells are naturally shielded from the immune system, enhancing their persistence and minimizing potentially dangerous immune responses. About TRX103 TRX103 is an investigational allogeneic off-the-shelf engineered T cell product generated from CD4+ cells sourced from healthy donors. These CD4+ cells are engineered to become cells that mimic the function of Tr1 regulatory T cells, called TRX cells. Tr1X is developing TRX103 for the treatment of several immune and inflammatory disorders. Multiple preclinical studies have shown TRX103 to be tolerable and effective and to have the potential to reset the immune system 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 private biotechnology company focused on curing immune mediated and inflammatory diseases. Founded by industry experts, including the scientists who discovered Tr1 cells, the company is developing a pipeline of off-the-shelf allogeneic cell therapies to treat and potentially cure autoimmune diseases with high unmet medical need. The company is supported 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. Investor/Media Contacts:Investors@tr1x.bio LifeSci AdvisorsChuck Padalachuck@lifesciadvisors.com646-627-8390

This marks the first use of an allogeneic engineered Type 1 Treg (Tr1) cell therapy in clinical trials. The first patient has successfully cleared the safety period with no serious adverse events. Additional preliminary safety and effectiveness data readouts expected by Q4 2024. July 10, 2024 -- Tr1X, Inc. (pronounced “Trix”), a biopharmaceutical company developing engineered Treg and CAR-Treg cell therapies, announced the successful dosing of the first patient in the company’s TRX103-01 proof of concept trial for prevention of Graft versus Host Disease (GvHD) and improved outcomes in patients with hematologic malignancies undergoing mismatched hematopoietic stem cell transplantation (HSCT). The study’s primary goal is to determine the safety, pharmacokinetics and pharmacodynamics of TRX103. The secondary goal of the study is to create immune tolerance to prevent GvHD and improve HSCT outcomes. The first patient had no serious adverse events, and the multi-center dose escalation trial continues to enroll. TRX103, a first-in-class Type 1 Treg (Tr1) cell therapy, is specifically designed for patients with autoimmune and inflammatory disorders, including those undergoing HSCT. TRX103 is an off-the-shelf, engineered Type 1 Treg (Tr1) cell therapy designed to reduce inflammation, prevent harmful T cell proliferation, and reset the immune system. This therapy harnesses unique properties of Tr1 cells that include trafficking to inflamed tissues and lymph nodes, lowering of inflammatory cytokines, control of harmful T cell responses, and creation of immune tolerance. Compared to other cell therapies, such as effector CAR-T cells, TRX103 Treg cells aim to treat autoimmune diseases with fewer side effects, potentially without the need for lymphodepletion. These cells are naturally shielded from the immune system, enhancing their persistence and minimizing potentially dangerous immune response. “This first patient to receive an allogeneic engineered Tr1 cell therapy, TRX103, is a significant achievement for our company, our scientists, and the field of cell and gene therapy,” said Maria Grazia Roncarolo, Co-founder, President and Head of Research and Development at Tr1X. “This is a key step in advancing our pipeline. We believe our allogeneic Tr1 Treg and CAR-Treg therapies can overcome the limitations of current treatments by controlling harmful T cells and B cells and creating immune tolerance, potentially curing a wide range of autoimmune and inflammatory disorders.” The TRX103-01 study builds on academic research that has shown the safety and efficacy of Tr1 Treg cell therapies for various autoimmune and inflammatory conditions, including prevention of GvHD and treatment of severe inflammatory bowel disease. The company has produced enough clinical doses from its first manufacturing runs using its proprietary closed process to complete the trial, which is designed to treat up to 36 patients with a one-time infusion of TRX103. Tr1X also plans to file an IND for TRX103 for treatment-refractory Crohn’s Disease in the second half of 2024. TRX103 is an investigational allogeneic off-the-shelf engineered T cell product generated from CD4+ cells sourced from healthy donors. These CD4+ cells are engineered to become cells that mimic the function of Tr1 regulatory T cells, called TRX cells. Tr1X is developing TRX103 for the treatment of several immune and inflammatory disorders. Multiple preclinical studies have shown TRX103 to be tolerable and effective and to have the potential to reset the immune system 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. Tr1X is a private biotechnology company focused on curing immune mediated and inflammatory diseases. Founded by industry experts, including the scientists who discovered Tr1 cells, the company is developing a pipeline of off-the-shelf allogeneic cell therapies to treat and potentially cure autoimmune diseases with high unmet medical need. The company is supported 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. The content above comes from the network. if any infringement, please contact us to modify.

Pictured: Cell therapy concept/iStock, Meletios Verras Tr1X came out of stealth mode on Wednesday with $75 million in Series A funding as it looks to bring allogeneic engineered Treg and CAR-Treg cell therapies to treat major autoimmune diseases into the clinic this year. The funding round was led by The Column Group and had participation from Alexandria Ventures and NEVA SGR. Tr1X will be led by CEO William Lis, who has been in biotech for over 25 years and was the interim CEO of Jasper Therapeutics from 2019 to 2022. Before that, he was CEO of Portola Therapeutics from 2008 to 2018. The San Diego-based biotech contends that its products are the first engineered Type 1 regulatory T (Tr1) therapeutics to be derived from universal donors to recapitulate function of naturally occurring Tr1 cells, which have features that can benefit patients with autoimmune and inflammatory diseases. Tr1 cells are “crucial” for maintaining homeostasis and tolerance in healthy patients, which include clamping down on inflammation and suppressing “pathogenic effector T cell response.” Long-term tolerance has also been observed in preclinical models, according to the company. Tr1X also claims it has proprietary tech that allows for converting CD4+T cells from healthy donors and then engineered to “target-specific” tissues or organs. The company’s first investigational cell therapy, TRX103, is slated to go into a Phase I trial this year to prevent Graft versus Host Disease in patients undergoing mismatched hematopoietic stem cell transplant. The company is also planning programs in Type 1 diabetes, inflammatory bowel disease as well as multiple B-cell mediated autoimmune diseases, with the expectation of being in Phase I clinical trials in multiple indications in 2024. Tr1X’s science is based on the work of its scientific founder, Maria Grazia Roncarolo, who will serve as the president and head of R&D as well as a director at the company. “The ability to develop a pipeline of medicines based on our work on regulatory T cells represents the culmination of decades of discovery and research into the underpinnings of immunological tolerance and autoimmunity,” Roncarolo said in a statement. “Tr1 cells have unique properties and represent the ideal therapeutic platform to develop ‘immune reset’ products. Our two platforms have the required attributes to address a broad set of indications, including dual targeting of pathogenic T and B cells.” So far in 2024, a steady stream of investment has been coming in for smaller biotechs emerging from stealth. This week, European-based biotech Disco Therapeutics emerged with over $20 million. Earlier this month, psychedelic-focused biotech MAPS Public Benefit Corporation rebranded to Lykos Therapeutics and secured a $100 million Series A round. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.