All three patients treated with FT819 following fludarabine-free conditioning regimen for severe lupus nephritis achieve Primary Efficacy Renal Response (PERR); first patient to reach 1-year follow-up continues in drug-free Definition of Remission in SLE (DORIS)
First extrarenal SLE patient on maintenance therapy treated with FT819 in the absence of conditioning achieves Low Lupus Disease Activity State (LLDAS) at 3-month follow-up and maintained at 6 months
Cumulative clinical experience in nearly 60 patients treated with off-the-shelf program across autoimmunity and oncology continues to support therapeutic differentiation with favorable safety profile and short duration of hospitalization
SAN DIEGO, CA, USA I June 11, 2025 I Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today announced new and updated clinical data from the first five patients dosed with FT819 for the treatment of moderate-to-severe systemic lupus erythematosus (SLE) at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain.
“The initial clinical profile of our FT819 off-the-shelf CAR T-cell program in moderate-to-severe SLE has shown clear therapeutic differentiation as all five patients achieved significant disease improvement with either less-intensive or no conditioning chemotherapy, with the first patient to reach 12-month follow-up showing durable drug-free remission,” said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. “We remain focused and have made good progress on growing our clinical footprint in the U.S. and Europe to accelerate the pace of patient enrollment in our FT819 program in lupus, as well as in myositis, systemic sclerosis and ANCA-associated vasculitis. With promising preliminary clinical data, a favorable safety profile, shortened hospitalization requirements, reduced or no conditioning chemotherapy regimens, and unique on-demand product availability with low cost of goods, we believe FT819 is not only well-suited for administration in academic settings but also an ideal drug product to support treatment in the community setting, facilitating broad patient access and geographical reach. Notably, the treatment response of the first patient exhibiting remission in SLE without conditioning chemotherapy is supportive of our pursuit to create drug products that bring together the treatment feasibility of biologics and the efficacy of CAR T cells. We look forward to discussing these initial clinical data and the unique attributes of FT819 with the FDA this summer under our RMAT designation to align on a registrational pathway for the benefit of patients with lupus.”
New and Updated Clinical Data Summary
The Company is currently conducting a multi-center, Phase 1 clinical trial of FT819, its off-the-shelf, CD19-targeted, 1XX CAR T-cell product candidate, for the treatment of patients with moderate-to-severe SLE, including lupus nephritis and extrarenal lupus (NCT06308978). The study is designed to evaluate the safety, pharmacokinetics, and activity of a single dose of FT819 administered to relapsed / refractory patients either (i) with a fludarabine (flu)-free conditioning regimen, consisting of either cyclophosphamide alone or bendamustine alone (Flu-free Conditioning Regimen) or (ii) in the absence of conditioning to patients on standard-of-care maintenance therapy (Conditioning-free Regimen). As of the data cut-off date of May 15, 2025, five patients have been treated with FT819 with sufficient follow-up to be evaluable, providing the following key insights:
Flu-free Conditioning Regimen
- Active Lupus Nephritis. Three relapsed / refractory patients with active lupus nephritis (median prior therapies = 8 [7-8], with all patients having received prior B-cell targeted therapy; median baseline SLEDAI-2K = 20 [14-20]) were treated with a single dose of FT819 at 360 million cells in the less-intensive Flu-free Conditioning Regimen. All three patients achieved primary efficacy renal response (PERR), with a 10-point or greater reduction in SLE disease activity index (SLEDAI-2K) from baseline (SLEDAI-2K best response; Patient 1: 20 to 4 at 12 months; Patient 2: 20 to 8 at 6.5 months; Patient 3: 14 to 4 at 1 month). The first patient to reach 12-month follow-up continues on-study in drug-free DORIS (definition of remission in SLE).
- Extrarenal Lupus. One relapsed / refractory patient with extrarenal lupus (prior therapies = 6, including cyclophosphamide; SLEDAI-2K = 18) was treated with a single dose of FT819 at 900 million cells in the less-intensive Flu-free Conditioning Regimen consisting of cyclophosphamide alone. The patient was evaluable for 1-month follow-up, demonstrating improvement across multiple disease-specific scores including an 8-point reduction in SLEDAI-2K from baseline and a 1-point reduction in PGA. The initial kinetics of improvement observed at 1-month following administration of FT819 appear to be generally consistent with those patients treated for active lupus nephritis.
Conditioning-free Regimen
- Add-on to Maintenance Therapy. One patient with extrarenal lupus (prior therapies = 5, including mycophenolate mofetil), who was on a stable dose of mycophenolate mofetil (1500mg twice daily) and 5 mg / day of steroids as maintenance therapy, was treated with a single dose of FT819 at 360 million cells in the Conditioning-free Regimen as an add-on to maintenance therapy. At 3-months following administration of FT819 in the absence of conditioning, the patient achieved low lupus disease activity state (LLDAS), which was maintained at the most recent follow-up at 6 months. The SLEDAI-2K reduced to 2 from 8 at baseline, Physician’s Global Assessment to 0.5 from 2 at baseline, and tapering of steroid dose to less than 5 mg / day.
B-cell Remodeling
Patients in the Flu-free Conditioning Regimen showed rapid and deep B-cell depletion in the periphery within the first month of treatment, with the repopulating B-cell compartment demonstrating a shift toward a non-switched, naïve repertoire with reduction of pathogenic double-negative B cell subset within the first 60 days following administration of FT819. The first patient in the Conditioning-free Regimen also showed a reduction of B cell population in the periphery, with remodeling of the B-cell compartment towards a non-switched, naïve repertoire also within the first 60 days following administration of FT819.
In 59 patients treated with FT819 across autoimmunity and oncology, a favorable safety profile continues to be observed with low incidence of low-grade cytokine release syndrome (CRS), no events of immune effector cell-associated neurotoxicity (ICANS), and no events of graft-versus-host disease (GvHD). Specifically, in the five evaluable patients treated with FT819 for SLE as of the data cut-off date:
- there have been no events of ICANS, no events of GvHD, and one event of low-grade cytokine release syndrome (Grade 2);
- no dose-limiting toxicities were observed in any patient; and
- with no FT819 related severe adverse events (SAEs) observed, all patients were discharged following an initial short-duration hospitalization stay of three days (as mandated per the clinical protocol), supporting the potential for outpatient administration
Program Expansion
The Company has reached agreement with the U.S. Food and Drug Administration (FDA) to allow for clinical investigation of multiple B cell-mediated autoimmune diseases under its current Phase 1 clinical trial of FT819, establishing the basis for conduct of a single Phase 1 basket study across autoimmune indications. The Company plans to initiate independent dose-expansion cohorts in each of anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc) in the second half of 2025. In addition, the Company plans to discuss novel registrational strategies with the FDA for FT819 to treat moderate-to-severe SLE under its Regenerative Medicine Advanced Therapy (RMAT) designation at its upcoming meeting in August. The Company currently has approximately 450 cryopreserved drug product bags of FT819 in inventory available for treatment of patients.
About Fate Therapeutics’ iPSC Product Platform
Human induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be administered in combination with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with patient- and donor-sourced cell therapies. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications.
About Fate Therapeutics, Inc.
Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived T-cell and natural killer (NK) cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com.
SOURCE: Fate Therapeutics
Post Views: 1,247
All three patients treated with FT819 following fludarabine-free conditioning regimen for severe lupus nephritis achieve Primary Efficacy Renal Response (PERR); first patient to reach 1-year follow-up continues in drug-free Definition of Remission in SLE (DORIS)
First extrarenal SLE patient on maintenance therapy treated with FT819 in the absence of conditioning achieves Low Lupus Disease Activity State (LLDAS) at 3-month follow-up and maintained at 6 months
Cumulative clinical experience in nearly 60 patients treated with off-the-shelf program across autoimmunity and oncology continues to support therapeutic differentiation with favorable safety profile and short duration of hospitalization
SAN DIEGO, CA, USA I June 11, 2025 I Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today announced new and updated clinical data from the first five patients dosed with FT819 for the treatment of moderate-to-severe systemic lupus erythematosus (SLE) at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain.
“The initial clinical profile of our FT819 off-the-shelf CAR T-cell program in moderate-to-severe SLE has shown clear therapeutic differentiation as all five patients achieved significant disease improvement with either less-intensive or no conditioning chemotherapy, with the first patient to reach 12-month follow-up showing durable drug-free remission,” said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. “We remain focused and have made good progress on growing our clinical footprint in the U.S. and Europe to accelerate the pace of patient enrollment in our FT819 program in lupus, as well as in myositis, systemic sclerosis and ANCA-associated vasculitis. With promising preliminary clinical data, a favorable safety profile, shortened hospitalization requirements, reduced or no conditioning chemotherapy regimens, and unique on-demand product availability with low cost of goods, we believe FT819 is not only well-suited for administration in academic settings but also an ideal drug product to support treatment in the community setting, facilitating broad patient access and geographical reach. Notably, the treatment response of the first patient exhibiting remission in SLE without conditioning chemotherapy is supportive of our pursuit to create drug products that bring together the treatment feasibility of biologics and the efficacy of CAR T cells. We look forward to discussing these initial clinical data and the unique attributes of FT819 with the FDA this summer under our RMAT designation to align on a registrational pathway for the benefit of patients with lupus.”
New and Updated Clinical Data Summary
The Company is currently conducting a multi-center, Phase 1 clinical trial of FT819, its off-the-shelf, CD19-targeted, 1XX CAR T-cell product candidate, for the treatment of patients with moderate-to-severe SLE, including lupus nephritis and extrarenal lupus (NCT06308978). The study is designed to evaluate the safety, pharmacokinetics, and activity of a single dose of FT819 administered to relapsed / refractory patients either (i) with a fludarabine (flu)-free conditioning regimen, consisting of either cyclophosphamide alone or bendamustine alone (Flu-free Conditioning Regimen) or (ii) in the absence of conditioning to patients on standard-of-care maintenance therapy (Conditioning-free Regimen). As of the data cut-off date of May 15, 2025, five patients have been treated with FT819 with sufficient follow-up to be evaluable, providing the following key insights:
Flu-free Conditioning Regimen
- Active Lupus Nephritis. Three relapsed / refractory patients with active lupus nephritis (median prior therapies = 8 [7-8], with all patients having received prior B-cell targeted therapy; median baseline SLEDAI-2K = 20 [14-20]) were treated with a single dose of FT819 at 360 million cells in the less-intensive Flu-free Conditioning Regimen. All three patients achieved primary efficacy renal response (PERR), with a 10-point or greater reduction in SLE disease activity index (SLEDAI-2K) from baseline (SLEDAI-2K best response; Patient 1: 20 to 4 at 12 months; Patient 2: 20 to 8 at 6.5 months; Patient 3: 14 to 4 at 1 month). The first patient to reach 12-month follow-up continues on-study in drug-free DORIS (definition of remission in SLE).
- Extrarenal Lupus. One relapsed / refractory patient with extrarenal lupus (prior therapies = 6, including cyclophosphamide; SLEDAI-2K = 18) was treated with a single dose of FT819 at 900 million cells in the less-intensive Flu-free Conditioning Regimen consisting of cyclophosphamide alone. The patient was evaluable for 1-month follow-up, demonstrating improvement across multiple disease-specific scores including an 8-point reduction in SLEDAI-2K from baseline and a 1-point reduction in PGA. The initial kinetics of improvement observed at 1-month following administration of FT819 appear to be generally consistent with those patients treated for active lupus nephritis.
Conditioning-free Regimen
- Add-on to Maintenance Therapy. One patient with extrarenal lupus (prior therapies = 5, including mycophenolate mofetil), who was on a stable dose of mycophenolate mofetil (1500mg twice daily) and 5 mg / day of steroids as maintenance therapy, was treated with a single dose of FT819 at 360 million cells in the Conditioning-free Regimen as an add-on to maintenance therapy. At 3-months following administration of FT819 in the absence of conditioning, the patient achieved low lupus disease activity state (LLDAS), which was maintained at the most recent follow-up at 6 months. The SLEDAI-2K reduced to 2 from 8 at baseline, Physician’s Global Assessment to 0.5 from 2 at baseline, and tapering of steroid dose to less than 5 mg / day.
B-cell Remodeling
Patients in the Flu-free Conditioning Regimen showed rapid and deep B-cell depletion in the periphery within the first month of treatment, with the repopulating B-cell compartment demonstrating a shift toward a non-switched, naïve repertoire with reduction of pathogenic double-negative B cell subset within the first 60 days following administration of FT819. The first patient in the Conditioning-free Regimen also showed a reduction of B cell population in the periphery, with remodeling of the B-cell compartment towards a non-switched, naïve repertoire also within the first 60 days following administration of FT819.
In 59 patients treated with FT819 across autoimmunity and oncology, a favorable safety profile continues to be observed with low incidence of low-grade cytokine release syndrome (CRS), no events of immune effector cell-associated neurotoxicity (ICANS), and no events of graft-versus-host disease (GvHD). Specifically, in the five evaluable patients treated with FT819 for SLE as of the data cut-off date:
- there have been no events of ICANS, no events of GvHD, and one event of low-grade cytokine release syndrome (Grade 2);
- no dose-limiting toxicities were observed in any patient; and
- with no FT819 related severe adverse events (SAEs) observed, all patients were discharged following an initial short-duration hospitalization stay of three days (as mandated per the clinical protocol), supporting the potential for outpatient administration
Program Expansion
The Company has reached agreement with the U.S. Food and Drug Administration (FDA) to allow for clinical investigation of multiple B cell-mediated autoimmune diseases under its current Phase 1 clinical trial of FT819, establishing the basis for conduct of a single Phase 1 basket study across autoimmune indications. The Company plans to initiate independent dose-expansion cohorts in each of anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc) in the second half of 2025. In addition, the Company plans to discuss novel registrational strategies with the FDA for FT819 to treat moderate-to-severe SLE under its Regenerative Medicine Advanced Therapy (RMAT) designation at its upcoming meeting in August. The Company currently has approximately 450 cryopreserved drug product bags of FT819 in inventory available for treatment of patients.
About Fate Therapeutics’ iPSC Product Platform
Human induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be administered in combination with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with patient- and donor-sourced cell therapies. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications.
About Fate Therapeutics, Inc.
Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived T-cell and natural killer (NK) cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com.
SOURCE: Fate Therapeutics
Post Views: 1,247
