First patient treated with FT819 off-the-shelf CAR T-cell product candidate following fludarabine-free conditioning for severe lupus nephritis demonstrated durability of response with drug-free definition of remission in systemic lupus erythematosus (DORIS) at 12-month follow-up Held initial discussion with FDA under FT819 RMAT designation to seek feedback on registrational pathway in moderate-to-severe Systemic Lupus Erythematosus (SLE) and refractory Lupus Nephritis (LN) First extrarenal SLE patient treat ...

Core Insights - Fate Therapeutics announced promising clinical data for FT819, an off-the-shelf CAR T-cell therapy for moderate-to-severe systemic lupus erythematosus (SLE), showing all five patients treated achieved significant disease improvement [2][3] - The first patient reached a 12-month follow-up and continues in drug-free remission, indicating the potential for durable treatment effects [2][4] - The company is expanding its clinical trials to include multiple B cell-mediated autoimmune diseases, with plans for independent dose-expansion cohorts in various conditions [8] Clinical Data Summary - A multi-center, Phase 1 clinical trial is evaluating FT819's safety and efficacy in patients with moderate-to-severe SLE, including lupus nephritis and extrarenal lupus [3] - As of May 15, 2025, five patients have been treated, with all showing significant improvements in disease activity scores [3][4] - Three patients with active lupus nephritis achieved primary efficacy renal response, with reductions in SLEDAI-2K scores of 10 points or more [4][5] Treatment Regimens - FT819 was administered under two regimens: a fludarabine-free conditioning regimen and a conditioning-free regimen [3][6] - In the fludarabine-free regimen, three patients with active lupus nephritis showed significant reductions in SLEDAI-2K scores, with one patient achieving a score reduction from 20 to 4 at 12 months [4] - One patient with extrarenal lupus on maintenance therapy achieved low lupus disease activity state (LLDAS) at 3 months, maintained at 6 months, with a reduction in SLEDAI-2K from 8 to 2 [6] B-cell Remodeling - Patients treated with FT819 exhibited rapid B-cell depletion and a shift towards a non-switched, naïve B-cell repertoire within the first 60 days [7] - This remodeling was observed in both treatment regimens, indicating a potential mechanism of action for FT819 [7] Safety Profile - The treatment demonstrated a favorable safety profile, with low incidence of cytokine release syndrome and no events of neurotoxicity or graft-versus-host disease [8][10] - All patients were discharged after a short hospitalization, supporting the potential for outpatient administration [10] Regulatory and Development Plans - Fate Therapeutics has reached an agreement with the FDA to investigate multiple autoimmune diseases under its Phase 1 trial for FT819 [8] - The company plans to discuss registrational strategies with the FDA for FT819 in SLE under its RMAT designation [8]

Core Insights - Fate Therapeutics is presenting clinical data from its Phase 1 study of FT819, an off-the-shelf CAR T-cell therapy for moderate-to-severe systemic lupus erythematosus (SLE) at the EULAR 2025 conference [1][2] - The study evaluates the safety and efficacy of FT819 with a fludarabine-free conditioning regimen, aiming to enhance patient access and therapeutic outcomes [2][4] Company Overview - Fate Therapeutics is a clinical-stage biopharmaceutical company focused on developing induced pluripotent stem cell (iPSC)-derived cellular immunotherapies [5][6] - The company has established a leadership position in creating multiplexed-engineered master iPSC lines and manufacturing off-the-shelf iPSC-derived cell products [6] Product Platform - The iPSC product platform allows for unlimited self-renewal and differentiation into all cell types, enabling the creation of well-defined and uniform engineered cell products [4][6] - The platform is supported by over 500 issued patents and 500 pending patent applications, highlighting its innovative approach and potential for broad patient application [4]

Financial Performance - Fate Therapeutics reported a loss of 32 cents per share for Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 39 cents, and an improvement from a loss of 47 cents per share in the same period last year [1] - The company earned collaboration revenues of $1.6 million, exceeding the Zacks Consensus Estimate of $1 million, although revenues declined by 15.8% year-over-year [1] - Research and development (R&D) expenses decreased by 9.3% to $29.1 million, while general and administrative (G&A) expenses decreased by approximately 34% to $13.8 million [4] - As of March 31, 2025, cash, cash equivalents, and investments totaled $272.7 million, which is expected to sustain operations through the first half of 2027 [4] Stock Performance - Shares of Fate Therapeutics increased by 5% in after-hours trading on May 13, following the better-than-expected earnings announcement [2] - Year-to-date, the stock has declined by 39.4%, compared to the industry's decline of 6.4% [3] Pipeline Development - Fate Therapeutics is focused on developing universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform, which includes iPSC-derived natural killer (NK) cells and T-cell product candidates [5] - A phase I study of FT819, a T-cell product candidate for systemic lupus erythematosus (SLE), is ongoing, with dose expansion initiated in up to 10 patients at 360 million cells [6][8] - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819 for treating active moderate to severe SLE, including lupus nephritis [9] - Fate Therapeutics is co-developing FT825/ONO-8250, a multiplexed-engineered, iPSC-derived CAR T-cell product candidate, with ONO Pharmaceutical, and is conducting a phase I study for advanced solid tumors [9][10]

Core Insights - Fate Therapeutics is advancing its FT819 off-the-shelf CAR T-cell product candidate for moderate-to-severe systemic lupus erythematosus (SLE), with new Phase 1 data to be presented at the EULAR 2025 Congress in June [1][2] - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for FT819, which will expedite its development and review process [5] - The company has $273 million in cash and investments, providing a projected operating runway through the first half of 2027 [1][11] Company Developments - The ongoing Phase 1 study of FT819 is designed to evaluate safety, pharmacokinetics, and efficacy, with patient enrollment at two dose levels: 360 million cells and 900 million cells [5] - The company plans to expand the Phase 1 study to include additional B cell-mediated autoimmune diseases, such as anti-neutrophilic cytoplasmic antibody-associated vasculitis, idiopathic inflammatory myositis, and systemic sclerosis [5] - Fate Therapeutics is also conducting a Phase 1 study for FT825, a CAR T-cell product candidate targeting HER2 in advanced solid tumors, which has shown a favorable safety profile [6] Financial Performance - For the first quarter of 2025, the company reported total revenue of $1.6 million, primarily from preclinical development activities [11] - Total operating expenses for the same period were $42.9 million, with research and development expenses accounting for $29.1 million [11][17] - The net loss for the first quarter was $37.6 million, compared to a net loss of $48.0 million in the same quarter of the previous year [11][17] Cash Position - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $272.7 million, which supports its ongoing operations and clinical development programs [11][19]

Core Viewpoint - Fate Therapeutics has received RMAT designation from the FDA for its investigational iPSC-derived CAR T-cell therapy, FT819, aimed at treating moderate to severe systemic lupus erythematosus (SLE) [1][2][3] Group 1: RMAT Designation and Its Implications - The RMAT designation acknowledges the potential of FT819 to meet the significant unmet needs of lupus patients and facilitates increased communication with the FDA during development [2][6] - The designation allows for early interactions with the FDA, including discussions on potential surrogate endpoints for accelerated approval [3][6] Group 2: Clinical Development of FT819 - FT819 is currently in Phase 1 clinical trials, focusing on safety and efficacy with a fludarabine-free conditioning regimen [2][6] - The ongoing study includes dose expansion to 10 patients at 360 million cells and safety assessments at 900 million cells [2] - Additional clinical data from the Phase 1 study is expected to be presented at scientific meetings later in 2025 [2][6] Group 3: Company Overview and iPSC Product Platform - Fate Therapeutics specializes in developing iPSC-derived cellular immunotherapies and has established a leadership position in creating multiplexed-engineered master iPSC lines [4][5] - The company's iPSC product platform is designed to produce uniform, off-the-shelf cell products that can be stored and administered in combination with other therapies [4][5] - Fate Therapeutics holds an extensive intellectual property portfolio with over 500 issued patents and 500 pending applications [4]