On March 5, 2025 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2024 (Press release, Fate Therapeutics, MAR 5, 2025, View Source [SID1234650907]).
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"We begin 2025 with resolve and focus to advance our lead clinical programs in autoimmunity and oncology," said Bob Valamehr, Ph.D. MBA, President and Chief Executive Officer of Fate Therapeutics. "The team continues to make great progress in our pursuit of achieving therapeutic differentiation for patients with B cell-mediated autoimmune diseases, and we look forward to providing clinical and regulatory updates as we advance our FT819 off-the-shelf CAR T-cell product candidate in SLE. We remain focused on driving patient enrollment and engaging with the FDA to further discuss novel development pathways for CAR T-cell therapy in autoimmune disease, including the use of fludarabine-free conditioning as well as add-on to maintenance therapy without conditioning. We also plan to explore FT819 clinical trial expansion in additional autoimmune diseases. In addition, our FT825 off-the-shelf CAR T-cell program for advanced solid tumors is advancing into higher-dose cohorts as monotherapy and in combination with monoclonal antibody therapy under our collaboration with Ono Pharmaceutical. We look forward to sharing clinical data from these two high-priority programs throughout the year."
FT819 iPSC-derived 1XX CAR T-cell Program
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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. The dose expansion stage is designed to evaluate the safety and efficacy of a fludarabine (flu)-free conditioning regimen, consisting of either bendamustine alone or cyclophosphamide alone, followed by a single dose of FT819. The Company is also assessing the safety, pharmacokinetics, and anti-B cell activity of FT819 at 900 million cells in dose escalation. FT819 is the Company’s off-the-shelf CD8αβ+ T-cell product candidate that incorporates a CD19-targeted chimeric antigen receptor (CAR) with a novel 1XX costimulatory domain into the T-cell receptor alpha constant (TRAC) locus.
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Clinical Data from First Three SLE Patients Presented at ASH (Free ASH Whitepaper). In December 2024, the Company highlighted clinical and translational data from the first three patients treated with FT819 for SLE at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Each patient presented with active lupus nephritis (LN) despite having been treated with multiple standard-of-care therapies and received flu-free conditioning followed by a single dose of FT819 at 360 million cells. As of a data cutoff date of December 4, 2024, there were no dose-limiting toxicities (DLTs), and no events of any grade of cytokine release syndrome (CRS), immune effector-cell associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD). All three patients showed rapid, deep, and sustained elimination of CD19+ B cells in the periphery during the first month of treatment. The first and only patient eligible for disease assessment at six-month follow-up as of the data cutoff date achieved DORIS (Definition Of Remission In SLE) clinical remission and was free of all immunosuppressive therapy.
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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. There were no DLTs and no events of CRS, ICANS, or GvHD. The patient remains on-study.
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Completed Type D Meeting with FDA for Inclusion of Additional Diseases in FT819 Phase 1 Study. 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, including the inclusion of patients between the ages of 12 and 17.
FT825 / ONO-8250 iPSC-derived CAR T-cell Program
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First Patient Treated with FT825 / ONO-8250 in Combination with Monoclonal Antibody Therapy. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). Enrollment is currently ongoing at the third dose level of 900 million cells as monotherapy and at the second dose level of 300 million cells in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy.
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Initial Phase 1 Clinical Data Presented at 2024 SITC (Free SITC Whitepaper). At the 2024 Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in November, the Company presented initial clinical data from three heavily pre-treated patients, all of whom were previously treated with at least five prior lines of therapy including HER2-targeted therapy. Each patient was administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 as monotherapy at the first dose level of 100 million cells. As of a data cutoff date of October 25, 2024, FT825 / ONO-8250 demonstrated a favorable safety profile with no DLTs and no events of any grade of CRS, ICANS, or GvHD. In addition, at Day 8 following treatment, peak CAR T-cell expansion was observed and phenotyping of FT825 / ONO-8250 sourced from the patients’ peripheral blood was indicative of an activated state (as evidenced by high levels of Granzyme B expression and maintenance of CAR expression) with no evidence of exhaustion (as evidenced by low levels of PD-1 and TIM3 expression).
FT522 iPSC-derived CAR NK Cell Program
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Initial Translational Data of FT522 without Conditioning Chemotherapy Presented at ACR Convergence. FT522 is the Company’s off-the-shelf, CD19-targeted CAR NK cell product candidate and first product candidate to incorporate Alloimmune Defense Receptor (ADR) technology, which is designed to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In November 2024, the Company presented initial translational data from its ongoing multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334). The presentation illustrated that live FT522 cells with anti-B cell activity were detected in the patients’ peripheral blood through Day 15, demonstrating the potential of FT522 to persist and function in the presence of an unmatched, fully-intact immune system. The Company intends to assess any further clinical development of FT522 in relapsed / refractory BCL upon completion of dose escalation at the second dose level of 900 million cells.
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Unique Clinical Development Opportunities for FT522 in Autoimmunity under Evaluation. The FDA has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Phase 1 clinical protocol allows for treatment of patients with up to four weekly doses of FT522, without administration of conditioning chemotherapy, as an add-on to rituximab induction therapy (Regimen A) and as an add-on to maintenance therapy in combination with rituximab (Regimen B). The Company is currently evaluating opportunities and timelines for the clinical development of FT522 in autoimmunity.
Fourth Quarter 2024 Financial Results
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Cash & Investment Position: Cash, cash equivalents, and investments as of December 31, 2024 were $306.7 million.
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Total Revenue: Revenue was $1.9 million for the fourth quarter of 2024, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical.
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Total Operating Expenses: Total operating expenses were $63.6 million for the fourth quarter of 2024, including research and development expenses of $33.6 million and general and administrative expenses of $15.3 million. Such amount included $9.1 million of non-cash stock-based compensation expense and a one-time non-cash asset impairment charge of $14.7 million related to equipment and right-of-use assets.
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Shares Outstanding: As of December 31, 2024, common shares outstanding were 113.9 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares.