Photo courtesy of Talaris.

Talaris Chief Executive Officer Scott Requadt sees FCR001, the companys investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications).

In fact, it was that versatility of the products potential that caused Requadt to join the company as CEO in 2018 after the company, known then as Regenerex, secured $100 million in financing. At the time, Requadt helmed the venture capital group Claris that backed the Series A.

I was really enthusiastic about the company and FCR001, Requadt told BioSpace in an interview.

FCR001, an investigational, allogeneic cell therapy, was previously part of Novartis gene and cell therapy unit, until it was dissolved in 2016. When that unit dissolved, rights to FCR001 reverted to Regenerex. With the return of those rights came multiple opportunities in organ transplant and severe autoimmune disorders. Cell therapies can address complex, multi-pathway diseases and Talaris has big plans for the future of FCR001 to help patients acquire or restore immune tolerance.

Our goal is to basically do for immune tolerance what CAR-T has done for oncology, Requadt said. The same product and the same basic biology will be used. We can treat organ transplant and autoimmune diseases in the same manner.

Through FCR001, Talaris will be able to change the underlying pathology of the disease so the immune system no longer sees it as a threat, Requadt said. The companys lead program is in kidney donor transplant, but FCR001, a one-time stem cell therapy, is also being explored as a treatment for scleroderma, a multi-system autoimmune disease. If FCR001 is effective in these areas, Requadt said those successes will pave the way for use in other indications.

This is a pipeline from a single product, Requadt said of FCR001, which has received a Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration (FDA).

In July, Kentucky-based Talaris dosed the first patient in its Phase III FREEDOM-1 study of FCR001 in living donor kidney transplant (LDKT) recipients. The trial is expected to enroll 120 adult patients who will receive kidney donations from living donors. The primary endpoint of the study will be the proportion of FCR001 recipients who are free from necessary drugs to maintain immunosuppression without biopsy proven acute rejection at 24 months post-transplant.

Organ transplant patients are required to continue taking drugs to suppress their immune systems to protect the new organ from immune system responses. However, those immunosuppressant treatments drugs are toxic to the kidney and can ultimately kill the transplanted organ in 10 to 15 years. The drugs can also lead to metabolic disorders and cardiovascular issues, Requadt said.

In 2018, Talaris posted positive Phase II data in LDKT recipients, with 70% of patients who received the treatment able to discontinue the use of immunosuppressant drugs. The Phase II data showed that every tolerized patient has been able to remain free of the use of chronic immunosuppressants for up to 10 years. The median follow-up following transplant was five years, with the longest case being a decade. Additionally, Requadt said the company has also seen better kidney functions in recipients who received FCR100 due to the lack of toxicity issues.

While there are other companies exploring similar approaches, Requadt said to his knowledge, no other group has a 10-year data set that demonstrates the safety and efficacy of a treatment like FRC001.

Not only has Talaris seen the impressive results in removing patients from the anchor of immunosuppressant drugs, Requadt said the use of FCR001 decreased the risk of rejection in patients whose biomarkers did not have as high a match with the kidney donors.

In addition to the Phase III study in LDKT patients, Talaris also has plans to conduct a Phase II study in LDKT Delayed Tolerance Induction and will begin research in Deceased Donor Kidney Transplant patients.

Talaris will also use FCR001 in the treatment of diffuse systemic sclerosis (SSc), a severe form of the rare autoimmune disease scleroderma, a rare and potentially fatal chronic autoimmune disease which causes progressive scarring, or fibrosis, of the bodys connective tissues. Autologous hematopoietic stem cell transplant has been shown to halt organ damage and induce remission of the disease. However, with the use of a patients own stem cells, there is a risk of disease recurrence. Also, some patients must undergo full myeloablative conditioning with or without total body irradiation, which is associated with direct organ toxicity and increased risk of future cancers. Talaris aims to harness the power of FCR001 and use stem cells from donors to lower those risks and provide an opportunity for these patients, Requadt said.

For all of these indications were using the same product and were using the same protocols with patients and were treating the patients in the same way. This has the potential to be paradigm shifting, Requadt said.

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Does Talaris' Investigational Cell Therapy Have Potential to Be a 'Pipeline in a Product'? - BioSpace