FT819 / Fate Therap, Memorial Sloan-Kettering Cancer Center - LARVOL DELTA

FT819 in Subjects With B-cell Malignancies (clinicaltrials.gov) - P1 | N=54 | Active, not recruiting | Sponsor: Fate Therapeutics | Trial primary completion date: Sep 2024 ➔ Jun 2025

Trial primary completion date • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • B Cell Lymphoma • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Oncology • CD19

Fate Therapeutics Presents Updated Phase 1 Clinical Data of FT819 Off-the-shelf CAR T-cell Product Candidate for Systemic Lupus Erythematosus… (GlobeNewswire) - "With the strength of this clinical data, the Company continues to advance preparations for a pivotal study and is engaged in discussions with the United States Food and Drug Administration (FDA) under its Regenerative Medicine Advanced Therapy (RMAT) designation regarding plans to initiate registrational trial of FT819 in 2026...Preliminary data in Regimen A (with patients receiving a single FT819 dose after pretreatment with either a single dose of cyclophosphamide or two doses of bendamustine) show mean SLEDAI-2K score across both dose levels (DL1, 360 million cells; DL2, 900 million cells) decreased progressively from baseline: DL1 SLEDAI-2K score decreased from a mean of 15.2 (n=5) at baseline to a mean of 10 at month 3 (n=2), and to a mean of 6 at month 6 (n=2), representing mean percent drops of 50% and 70%, respectively; and; DL2 SLEDAI-2K score decreased from a mean of 14.3 (n=3) at baseline to a mean of 6 at month 3 (n=2) and to 4 at month 6 (n=1)..."

New trial • P1 data • Lupus Nephritis • Systemic Lupus Erythematosus

Authorization received from Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA) to initiate clinical trials of FT819. (Fate Therapeutics Press Release) - "Fate Therapeutics has now received regulatory clearance from the United Kingdom (UK) MHRA to proceed with clinical evaluation of FT819 in autoimmune diseases. This authorization represents a significant step in the Company’s international expansion and highlights global interest in the unique ability of FT819 to enable broad patient accessibility of CAR T cells without the requirement of intensive conditioning. The first of several planned UK clinical sites is now active and open to patient enrollment. The Company also received regulatory authorization from the European Union (EU) EMA to initiate the FT819 clinical trial across multiple EU countries."

Trial status • ANCA Vasculitis • Immunology • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

UCI Health clinical trial tests promising CAR T-cell therapy for lupus (Newswise) - "One of the first California participants treated, a 41-year-old Irvine woman, experienced a striking decrease in symptoms and improved quality of life within several weeks of treatment, said UCI Health rheumatologist Dr. Sheetal Desai, the clinical trial’s principal investigator at the UCI Alpha Clinic, one of nine U.S. clinical trial sites...She said the CAR T-cell therapy has the potential to help patients with moderate to severe systemic lupus erythematosus who do not respond to current treatments. Desai is a professor and chief of the UC Irvine School of Medicine's Division of Rheumatology and executive vice chair of the Department of Medicine."

Trial status • Systemic Lupus Erythematosus

Longitudinal Analysis of B cell Remodeling in Systemic Lupus Erythematosus Following iPSC-Derived CAR T-cell Therapy (ACR Convergence 2025) - P1 | "The CAR transgene is integrated into the T cell receptor (TCR) alpha constant locus to promote uniform CAR expression and enhance potency, with bi-allelic disruption of TCR expression to completely prevent graft-versus-host disease. All patients enrolled to date were treated with FT819 for moderate to severe systemic lupus erythematosus (SLE) and either received fludarabine-free CCT (bendamustine or cyclophosphamide) or received no CCT and continued their standard-of-care therapy. Collectively, these findings demonstrate that FT819 can remodel the B-cell compartment toward a naïve and more diverse repertoire, even without CCT, supporting immune reset as a mechanism of clinical remission. This trial is actively enrolling, and updated patient data will be included at the time of presentation."

CAR T-Cell Therapy • IO biomarker • Graft versus Host Disease • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

iPSC-Derived Off-the-Shelf anti-CD19 CAR T cells Deliver Improved Clinical Outcomes in Lupus with Reduced or No Conditioning Chemotherapy (ACR Convergence 2025) - P1 | "FT819 was administered as a single dose under 2 regimens (Reg): Reg A, fludarabine-free conditioning chemotherapy (CCT) with single-dose cyclophosphamide or bendamustine daily for 2 days prior to FT819; and Reg B, no CCT with continued stable dose of maintenance therapy, including mycophenolate (MMF). As of the data cutoff date, pts treated in Reg A and Reg B had follow-up ranging from 1-12 months (mo). Preliminary patient data indicates a favorable safety profile, effective B cell depletion, and promising initial efficacy. These findings support the continued evaluation of FT819 in SLE as well as other B-cell mediated autoimmune diseases (ANCA-associated vasculitis, idiopathic inflammatory myositis, and systemic sclerosis) included in the FT819 protocol."

CAR T-Cell Therapy • Clinical • Clinical data • IO biomarker • ANCA Vasculitis • Fatigue • Glomerulonephritis • Graft versus Host Disease • Hematological Disorders • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Myositis • Nephrology • Scleroderma • Systemic Sclerosis • Vasculitis

Program Expansion and Outlook (The Manila Times) - "In addition, the Company is actively engaged under its Regenerative Medicine Advanced Therapy (RMAT) designation with the U.S. Food and Drug Administration (FDA) to align on a registrational study design with the goal to initiate a pivotal study in 2026."

New trial • Systemic Lupus Erythematosus

Increasing Participation in the FT819 Cell Therapy Trial Amongst People Living with Lupus: A Focus Group Study (ACR Convergence 2025) - "Clinical trials in the U.S. have often failed to include representative samples, leading to a lack of generalizability. This study identified barriers and facilitators to cell therapy trial participation among a purposive sample. The findings can guide activities such as patient engagement, recruitment, and site selection."

Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Fate Therapeutics Presents New Clinical Data at ACR Convergence 2025 Demonstrating Immune Remodeling and Durable Responses in Patients with Systemic Lupus Erythematosus Treated with FT819 Off-the-Shelf CAR T-Cell Therapy (The Manila Times) - "All patients surpassing a 3-month post-treatment time point (n=5) showed significant reductions in the SLE Disease Activity Index (SLEDAI-2K) score and Physician’s Global Assessment (PGA); SLE patients with lupus nephritis (LN) surpassing a 3-month post-treatment time point (n=2) achieved complete renal response (CRR) at 6 months; first patient continues in drug-free Definition of Remission in SLE (DORIS) at 15 months follow-up."

P1 data • Systemic Lupus Erythematosus

In discussion with the FDA on potential registrational study design in moderate-to-severe SLE and refractory LN (GlobeNewswire) - "In August, the Company met with the U.S. Food and Drug Administration (FDA) under its Regenerative Medicine Advanced Therapy (RMAT) designation for FT819 to seek preliminary feedback on a proposed registrational study design to support regulatory approval in moderate-to-severe SLE and refractory LN."

FDA event • Lupus Nephritis • Systemic Lupus Erythematosus

Phase 1 SLE study amended to include additional B cell-mediated autoimmune diseases (GlobeNewswire) - "The Company has expanded its current Phase 1 clinical trial of FT819 to include clinical investigation of multiple B cell-mediated autoimmune diseases, with plans to initiate independent dose-expansion cohorts in the second half of 2025 for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc)."

Trial status • ANCA Vasculitis • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

Treatment of Refractory Systemic Lupus Erythematosus with Off-the-Shelf iPSC-derived Anti-CD19 CAR T-cell Therapy (EULAR 2025) - P1 | "Regimen A is a single dose of FT819 following treatment with fludarabine-free preconditioning chemotherapy consisting of either cyclophosphamide (CY) or bendamustine...All patients reported a median of 8 prior therapies (range 7-8) to treat their SLE, including rituximab...At the time of study entry, all patients continued baseline hydroxychloroquine therapy, and two patients continued low dose corticosteroids... Preliminary data from the first three patients in this clinical trial indicate favorable safety profile, effective B cell depletion with reconstitution of more naïve B cells, and promising initial efficacy. These findings support the continued evaluation of FT819 in SLE and expansion to other B-cell mediated autoimmune diseases, including ANCA-associated vasculitis, idiopathic inflammatory myositis, and systemic sclerosis. This initial report highlights the entry of off-the-shelf anti-CD19 CAR T-cell therapy into the clinical trial landscape, with..."

CAR T-Cell Therapy • IO biomarker • ANCA Vasculitis • Fatigue • Glomerulonephritis • Graft versus Host Disease • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Myositis • Nephrology • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis • Vasculitis

Fate Therapeutics Announces Updated Clinical Data for FT819 Off-the-shelf CAR T-cell Product Candidate Demonstrating Durability of Drug-free Remission for Severe Lupus Nephritis at EULAR 2025 Congress (GlobeNewswire) - P1 | N=244 | NCT06308978 | Sponsor: Fate Therapeutics | "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."

P1 data • Lupus Nephritis • Systemic Lupus Erythematosus

Fate Therapeutics Announces Phase 1 Data Presentation of FT819 Off-the-Shelf CAR T-cell Product Candidate for SLE at EULAR 2025 Congress (GlobeNewswire) - "The Company has been selected to provide an oral presentation featuring clinical data from its ongoing Phase 1 clinical trial of FT819, its off-the-shelf, CD19-targeted, 1XX CAR T-cell product candidate, in patients with moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978). The Phase 1 study is designed to evaluate the safety and activity of FT819 in patients receiving either a fludarabine (flu)-free conditioning regimen or maintenance therapy without conditioning."

P1 data • Systemic Lupus Erythematosus

Fate Therapeutics Reports First Quarter 2025 Financial Results and Business Updates (GlobeNewswire) - "The Company’s ongoing multi-center, Phase 1 clinical trial of FT819 for moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978) is designed to evaluate the safety, pharmacokinetics, and efficacy of a single dose of FT819 following a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone. The Company is currently enrolling patients at two dose levels....The Company plans to present new clinical data from its FT819 Phase 1 study during an oral session at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in Barcelona, Spain on June 11."

P1 data • Trial status • Systemic Lupus Erythematosus

Fate Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA for FT819 to Treat Moderate to Severe Systemic Lupus Erythematosus (SLE) (GlobeNewswire) - "Fate Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819, an investigational, off-the-shelf, iPSC-derived CAR T-cell therapy in Phase 1 clinical development for the treatment of active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN)....The Company’s RMAT application included initial clinical safety and activity data from patients treated with FT819 in its ongoing multi-center Phase 1 clinical trial....The Company will report additional clinical data from the Phase 1 study of FT819 at scientific meetings later in 2025."

FDA event • P1 data • Lupus Nephritis • Systemic Lupus Erythematosus

FT819-102: A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease (clinicaltrials.gov) - P1 | N=244 | Recruiting | Sponsor: Fate Therapeutics | N=64 ➔ 244

Enrollment change • Immunology • Inflammatory Arthritis • Lupus • Myositis • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis • Vasculitis

Completed Type D Meeting with FDA for Inclusion of Additional Diseases in FT819 Phase 1 Study (GlobeNewswire) - "In December 2024, the Company reached an agreement with the U.S. Food and Drug Administration (FDA) to allow for the clinical investigation of additional B cell-mediated autoimmune diseases under our current Phase 1 clinical trial of FT819. As a follow-up to the meeting, the Company has submitted an amended clinical protocol to the FDA that enables the conduct of independent dose-expansion cohorts for SLE as well as anti-neutrophilic cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The Company plans to initiate dose-expansion cohorts in one or more of AAV, IIM, and SSc in 2025. Additionally, the FDA agreed to allow for investigation of a multi-dose treatment cycle as well as for re-treatment upon disease progression, making the treatment dosing paradigm more aligned with traditional biological therapies. The FDA also permitted the expansion of study eligibility criteria..."

Clinical protocol • FDA event • Trial status • ANCA Vasculitis • Myositis • Systemic Lupus Erythematosus • Systemic Sclerosis

Fate Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Business Updates (GlobeNewswire) - "Phase 1 Dose Expansion Initiated for SLE using Flu-free Conditioning Regimen: Based on clinical data from the first three patients treated with FT819 in its ongoing multi-center, Phase 1 clinical trial for moderate-to-severe systemic lupus erythematosus (SLE) (NCT06308978), the Company has initiated dose expansion in up to 10 patients at 360 million cells....First Patient Treated with FT819 as Add-on to Maintenance Therapy: The Company amended the clinical protocol of its FT819 Phase 1 study to include a new treatment arm to assess the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 as an add-on to maintenance therapy without conditioning chemotherapy in patients with SLE. The first patient in the new arm was on a stable dose of oral mycophenolate mofetil (MMF) and was treated with a single dose of FT819 at 360 million cells without administration of any conditioning chemotherapy."

Trial status • Systemic Lupus Erythematosus

FT819 Patient 1 Case Study (GlobeNewswire) - P1 | N=64 | NCT06308978 | Sponsor: Fate Therapeutics | "Rapid elimination of CD19+ B cells in the periphery was observed following treatment, and B-cell recovery by Month 3 was predominantly comprised of naïve, non-class switched B cells with near-complete elimination of switched memory B cells and deep depletion of plasmablasts, indicative of an immune reset. The patient reported that her debilitating fatigue had entirely resolved without further treatment, and treatment with methylprednisolone was discontinued at Month 3. The patient achieved DORIS (definition of remission in SLE) clinical remission, including with resolution of arthritis and active urinary sediment and with a substantial reduction in proteinuria, as of Month 6 follow-up. The patient continues on-study, in DORIS clinical remission, and remains free of all immunosuppressive therapy."

P1 data • Immunology • Lupus • Lupus Nephritis • Systemic Lupus Erythematosus

Fate Therapeutics Presents New Phase 1 Clinical Data of FT819 Off-the-shelf, CAR T-cell Product Candidate for Systemic Lupus Erythematosus (GlobeNewswire) - P1 | N=64 | NCT06308978 | Sponsor: Fate Therapeutics | "Fate Therapeutics, Inc...today presented new clinical and translational data from the Company’s FT819 Phase 1 Autoimmunity study for moderate-to-severe systemic lupus erythematosus (SLE) at the American Society of Hematology (ASH) Annual Meeting being held in San Diego, CA. The first three study patients, each of whom presented with active lupus nephritis (LN) despite having been treated with multiple standard-of-care therapies, received fludarabine-free conditioning followed by a single dose of FT819 at 360 million cells."

P1 data • Immunology • Lupus Nephritis • Systemic Lupus Erythematosus

Fate Therapeutics Reports Third Quarter 2024 Financial Results and Business Updates (GlobeNewswire) - "Initial Clinical Data from Phase 1 Autoimmunity Study of FT819 1XX CAR T-cell Product Candidate to be Presented in 4Q24; Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning Chemotherapy Opened for Enrollment; FT522 Multi-indication IND Application for Conditioning-free Treatment of B Cell-mediated Autoimmune Diseases Allowed by FDA; Initial Phase 1 Clinical Data in B-cell Lymphoma to be Presented at ACR Convergence."

IND • P1 data • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Immunology • Lupus • Lymphoma • Oncology • Systemic Lupus Erythematosus

Fate Therapeutics Presents 6-Month Follow-up Data on First Patient Treated in Phase 1 Autoimmunity Study with Fludarabine-free Conditioning and FT819 Off-the-shelf, 1XX CAR T-cell Product Candidate at ACR Convergence (GlobeNewswire) - P1 | N=64 | NCT06308978 | Sponsor: Fate Therapeutics | "Fate Therapeutics...today presented initial clinical and translational data from the first patient treated in its FT819 Phase 1 Autoimmunity study for moderate-to-severe systemic lupus erythematosus (SLE) at the American College of Rheumatology (ACR) Convergence being held in Washington, D.C....The patient achieved DORIS (definition of remission in SLE) clinical remission and LLDAS (low lupus disease activity state) as of Month 6 follow-up. The patient continues on-study, in clinical remission, and free of all immunosuppressive therapies as of a data cutoff date of November 11, 2024.... The Company plans to present clinical and translational data from the first three patients at the American Society of Hematology (ASH) Annual Meeting being held in San Diego, CA on December 7-10."

P1 data • Immunology • Systemic Lupus Erythematosus

Fate Therapeutics Reports Second Quarter 2024 Financial Results and Business Updates (GlobeNewswire) - P1 | N=64 | NCT06308978 | Sponsor: Fate Therapeutics | "The first patient treated in the study, a 27 year-old woman diagnosed with lupus nephritis over ten years ago who has refractory disease despite having been treated with multiple standard-of-care therapies, received conditioning chemotherapy followed by a single dose of FT819 at 360 million cells. The patient remains on-study, and there have been no Grade ≥3 adverse events and no events of any grade of cytokine release syndrome (CRS), immune effector-cell associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD). The Company plans to present clinical and translational data from the Phase 1 study at a medical conference in the second half of 2024....The Company intends to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the third quarter of 2024 for the treatment of a basket of B cell-mediated autoimmune diseases with FT522..."

IND • P1 data • Immunology • Lupus • Systemic Lupus Erythematosus

FT819 in Moderate to Severe Active Systemic Lupus Erythematosus (clinicaltrials.gov) - P1 | N=64 | Recruiting | Sponsor: Fate Therapeutics | N=32 ➔ 64

Enrollment change • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus