Talaris' Cell Therapy for Organ Rejection

By Erin Harris, Editor-In-Chief, Cell & Gene
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Talaris Therapeutics is a late-clinical stage cell therapy company, and the company’s lead candidate, FCR001, is a cell therapy designed to combat organ rejection. The company is currently evaluating FCR001 in FREEDOM-1, a randomized, controlled, open-label, multi-center Phase 3 trial in the U.S. This registration trial is designed to assess the safety and efficacy of FCR001 in first-time living donor kidney transplant (LDKT) recipients, where FCR001 is administered the day after the kidney transplant. The goal of the FREEDOM-1 trial is to assess the potential of FCR001 to induce durable immune tolerance to the transplanted kidney without the need for chronic immunosuppression to prevent graft rejection. Based on the data generated from the Phase 2 trial, FCR001 has been granted Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations by the FDA in this indication.

FREEDOM-2 is a Phase 2 trial to evaluate the potential for FCR001 to induce durable immune tolerance in patients who have previously received a kidney from a living donor, a process called delayed tolerance. In this trial, FCR001 will be administered between three to twelve months after the initial kidney transplant.

FREEDOM-3 is a Phase 2 trial exploring the safety and clinical activity of FCR001 in patients with a severe form of scleroderma, a group of rare autoimmune diseases of the joints and connective tissue. The treatment, an allogeneic stem cell therapy, is made of stem and immune cells from a healthy donor. If the data are positive, this could open the use of Talaris’ therapy for a variety of severe, systemic autoimmune diseases for which there is currently no cure. Their proprietary therapeutic approach, Facilitated Allo-HSCT (allogeneic hematopoietic stem cell transplantation) Therapy, is designed to induce immune tolerance. It does so by creating chimerism. According to NIH, recent exciting discoveries have demonstrated that HSCT could be expanded to solid tissue cancers with profound effects on the effectiveness of adoptive immunotherapy.

Across these conditions, immunosuppression dramatically affects not only people’s health but also their quality of life. Indeed, immunosuppressive therapy may be used to keep a person from rejecting a bone marrow or organ transplant. It may also be used to treat conditions in which the immune system is overactive, such as autoimmune diseases and allergies. Some types of immunosuppressive therapy may increase a person’s risk of cancer by lowering the body’s ability to kill cancer cells.

Talaris is now treating living donor kidney transplant patients in its Phase 3 trial of FCR001, gathering the final data needed for FDA submission. “Today, organ transplant recipients must take lifelong immunosuppression to avoid rejecting their transplanted organ. These immunosuppressive regimens have significant morbidities, risks, and quality of life challenges,” explains Scott Requadt, CEO, Talaris. “A treatment alternative for these patients is greatly needed. The interim update presented today from our FREEDOM-1 study continues to support our prior data and belief that patients who achieve >50% T cell chimerism at 3, 6 and 12 months after the administration of FCR001 may be durably weaned from chronic immunosuppression without rejecting their transplanted organ. Overall, we are pleased with the continued progress of our FREEDOM-1 trial, with seven study patients now successfully dosed and 100% engraftment across all dosed patients, at multiple trial sites. These interim results continue to give us confidence that FCR001 has the potential to transform the standard of care in solid organ transplantation.”