On August 13, 2024 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 cellular immunotherapies to patients with cancer and autoimmune diseases, reported business highlights and financial results for the second quarter ended June 30, 2024 (Press release, Fate Therapeutics, AUG 13, 2024, View Source [SID1234645804]).
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"We are pleased with the initial clinical and translational observations from our three ongoing Phase 1 studies and look forward to sharing data from each program in the second half of 2024," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We remain keenly focused on achieving therapeutic differentiation in autoimmunity with our off-the-shelf FT819 CAR T-cell and FT522 CAR NK cell product candidates. Our ongoing FT819 study for systemic lupus erythematosus now includes single-agent cyclophosphamide as an alternative conditioning regimen for patients, and we are working to expand clinical investigation of FT819 to treat additional B cell-mediated diseases. We are also finalizing our FT522 IND submission for the treatment of a basket of autoimmune diseases without administration of conditioning chemotherapy to patients, having now treated the first patient without conditioning chemotherapy in our ongoing FT522 study for B cell lymphoma. In addition, under our solid tumor collaboration with Ono Pharmaceutical, we have now treated the first three patients as monotherapy with our multiplexed-engineered FT825 CAR T-cell collaboration candidate, and we are poised to initiate dosing in combination with monoclonal antibody therapy to explore the potential of dual-antigen targeting in advanced solid tumors."
FT819 iPSC-derived 1XX CAR T-cell Program
Enrollment Ongoing in FT819 Phase 1 Autoimmunity Study. The multi-center, Phase 1 clinical trial for patients with systemic lupus erythematosus (SLE) is designed to evaluate the safety, pharmacokinetics, and anti-B cell activity of FT819, 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 (NCT06308978). 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.
Single-agent Cyclophosphamide Included as Alternative Conditioning Regimen. In addition to conditioning of patients with either cyclophosphamide and fludarabine (Cy / Flu) or bendamustine, the Company amended the clinical protocol of its FT819 Phase 1 autoimmunity study to include single-agent cyclophosphamide (Cy) as a third conditioning regimen. Cy is a commonly-used agent with an established safety profile for the treatment of patients with B cell-mediated autoimmune diseases.
Favorable Safety Profile and Proof-of-Concept for Autoimmune Disease Established in FT819 Phase 1 BCM Study. At the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting in May, the Company presented clinical and translational data from its FT819 Phase 1 study in relapsed / refractory B cell malignancies (BCM) (NCT04629729). 43 heavily pre-treated patients were treated with conditioning chemotherapy and a single dose of FT819 across five dose levels. The safety and tolerability profile of FT819 was favorable, with no dose-limiting toxicities, no events of any grade of ICANS or GvHD, and low incidence (14%) of low-grade CRS. In addition, a single dose of FT819 exhibited multiple mechanisms implicated in generating an immune reset in patients with B cell-mediated autoimmune diseases, including rapid, deep, and sustained CD19+ B-cell depletion in the periphery throughout the one-month treatment cycle as well as primary, secondary, and tertiary tissue trafficking, infiltration, and activity with CD19+ B cell elimination in tissue. The Company successfully completed dose escalation in its FT819 Phase 1 BCM study, and has focused further clinical development of FT819 exclusively in autoimmunity.
FT825 / ONO-8250 iPSC-derived CAR T-cell Program
Enrollment Ongoing in Phase 1 Study with HER2-targeted CAR T-cell for Advanced Solid Tumors. 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 as monotherapy and in combination with monoclonal antibody therapy in patients with advanced solid tumors (NCT06241456). Designed using the Company’s iPSC product platform, FT825 / ONO-8250 incorporates seven synthetic controls of cell function including a novel cancer-specific H2CasMab-2 CAR, which has exhibited similar potency with greater specificity for cancer cells expressing HER2 compared to trastuzumab in preclinical studies. Three patients have been treated in the Phase 1 study with a single dose of FT825 / ONO-8250 as monotherapy at the first dose level of 100 million cells and, during the third quarter of 2024, the Company plans to initiate enrollment as monotherapy at the second dose level of 300 million cells and in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy at the first dose level of 100 million cells. The Company plans to present clinical and translational data from the Phase 1 study at a medical conference in the second half of 2024.
FT522 iPSC-derived CAR NK Cell Program
First Patient Treated without Conditioning in Phase 1 BCL Study. FT522 is the Company’s off-the-shelf, CD19-targeted CAR NK cell product candidate and its first 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 its ongoing multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), the first patient has now been treated in the first three-dose cohort at 300 million cells per dose without conditioning chemotherapy (Regimen B). In addition, the first patient has now been treated in the second three-dose cohort at 900 million cells per dose with conditioning chemotherapy (Regimen A). No dose-limiting toxicities and no events of any grade of CRS, ICANS, or GvHD, have been reported in the Phase 1 study. The Company plans to present clinical and translational data from the Phase 1 study at a medical conference in the second half of 2024.
IND Application for Phase 1 Basket Study in Autoimmunity to be Submitted in 3Q24. 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, including without administration of conditioning chemotherapy to patients. At the ASGCT (Free ASGCT Whitepaper) conference, the Company presented preclinical data from a novel re-challenge assay using peripheral blood mononuclear cells (PBMCs) from unmatched SLE donors, showing that FT522 uniquely drove rapid and deep CD19+ B cell depletion, eliminated alloreactive T cells, and maintained functional persistence, indicating that FT522 can function effectively in the presence of an unmatched host immune system. The Company also shared initial clinical observations from the first two patients treated with FT522 at 100 million cells per dose in Regimen A of its ongoing Phase 1 BCL study, which showed rapid, deep, and sustained B-cell depletion in the periphery throughout the one-month treatment cycle. In addition, both patients showed enhanced persistence of FT522 in the periphery compared to clinical data observed with FT596, a prior-generation CD19-targeted CAR NK cell without ADR technology.
Second Quarter 2024 Financial Results
Cash & Investment Position: Cash, cash equivalents and investments as of June 30, 2024 were $352.0 million.
Total Revenue: Revenue was $6.8 million for the second quarter of 2024, which was derived from the achievement of a $5 million milestone in connection with the clinical development of FT825 / ONO-8250 and the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under its collaboration with Ono.
Total Operating Expenses: For the second quarter of 2024, GAAP operating expenses were $51.9 million, including research and development expenses of $34.6 million and general and administrative expenses of $17.3 million. Such amounts included $9.6 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 113.8 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million, as of June 30, 2024. Each preferred share is convertible into five common shares.