22 April 2019 - - US-based biotechnology company Talaris Therapeutics, Inc., formerly Regenerex, has received full results of its Phase 2 trial of FCR001 in living donor kidney transplant recipients, the company said.

The therapy's strong durability of benefit and safety profile support the initiation of a Phase 3 clinical trial in LDKT later this year.

The company also closed a USD 100m series A financing from a syndicate of leading life sciences investors.
FCR001, an investigational, allogeneic cell therapy that was patented and incubated at the University of Louisville, is being developed to induce or restore patients' immune tolerance by establishing a stable, chimeric immune system comprised of both donor and recipient cells.

Clinical data have shown the potential for FCR001 to durably free a significant proportion of living donor kidney transplant recipients from all chronic immunosuppression by 12 months after their transplant, without rejection of their transplanted organ.

In a groundbreaking Phase 2 study of FCR001 conducted at two leading transplant centers in the United States, 26 of 37 of LDKT recipients of FCR001 were able to be weaned off all of their immunosuppression treatments.

The degree of HLA mismatch (spanning 0/6 to 6/6) between the donor and the recipient did not affect either the safety or the efficacy of the therapy.

Every tolerized patient has subsequently remained off all chronic immunosuppression, with median follow-up since transplant of approximately five years and longest follow-up of almost 10 years.

In addition, of the seven LDKT patients with a prior auto-immune disease that led to their kidney failure and who were successfully tolerized with FCR001, none has to date had a recurrence of their underlying auto-immune disease.

FCR001 has received a Regenerative Medicine Advanced Therapy designation from the US Food and Drug Administration.

The company plans to launch additional Phase 2 studies to evaluate the potential of FCR001 to treat certain severe auto-immune or immune-mediated disorders, as well as to induce durable immune tolerance in other organ transplant settings.

Kidney transplants are the most frequent organ transplant procedure in the world. Over 6,400 LDKTs were performed in the US alone in 2018.

Currently, organ transplant recipients require lifelong immunosuppression to prevent rejection of their transplanted organ. Chronic immunosuppression brings significant side effects, an increased risk of infection and malignancy, and reduced quality of life.

Additionally, the toxicity of these drugs to the kidney leads to declining kidney function, typically resulting in kidney failure and the need for a new transplant within 15 years.

FCR001 is an investigational, allogeneic cell therapy developed by Talaris to induce or restore patients' immune tolerance. FCR001 builds on over 30 years of research by the company's founder, Dr. Suzanne Ildstad, into the means by which durable immune tolerance can be induced in a patient who receives a transplanted organ or can be restored in patients with certain immune-mediated or blood disorders.

It was patented and incubated at the University of Louisville. The lead indication for FCR001 is to induce durable immune tolerance in living donor kidney transplant recipients. FCR001 has received Orphan Drug Designation and a Regenerative Medicine Advanced Therapy designation from the US Food and Drug Administration.

Talaris Therapeutics, Inc., formerly Regenerex, is a late-clinical stage biotechnology company that is developing transformative cell therapies with the potential to eliminate the burden of chronic immunosuppression for organ transplant recipients as well as induce durable remissions in patients with severe auto-immune and immune-mediated disorders.

Talaris was founded on technology discovered and developed by Dr. Suzanne Ildstad and operates its own cell processing facility in Louisville, KY. Talaris is backed by leading life sciences investors Blackstone Life Sciences, Longitude Capital and Qiming Venture Partners USA and maintains corporate offices in Boston, MA and Louisville, KY.