On February 26, 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 disorders, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2023 (Press release, Fate Therapeutics, FEB 26, 2024, View Source [SID1234640448]).

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"We have started the year with strong momentum across our iPSC product platform in oncology and autoimmunity, including the award of a grant by the California Institute of Regenerative Medicine to support Phase 1 clinical investigation of our off-the-shelf FT819 CAR T-cell program in systemic lupus erythematosus," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We have also treated the first patient with FT522, our off-the-shelf CAR NK cell program targeting CD19+ B cells, which is our first product candidate to incorporate our proprietary Alloimmune Defense Receptor technology that is designed to reduce or eliminate the need for administration of intense chemotherapy conditioning to patients. In addition, we have initiated the Phase 1 study of our FT825 / ONO-8250 CAR T-cell program in solid tumors, which incorporates seven synthetic controls of cell function including a novel cancer-specific binding domain targeting HER2. We are well positioned to generate initial clinical data across these off-the-shelf programs during 2024."

FT819 iPSC-derived CAR T-cell Program

CLIN2 Grant Awarded by CIRM to Fund FT819 Phase 1 Autoimmunity Study in SLE. In February, the Company was awarded $7.9 million by the California Institute for Regenerative Medicine (CIRM) to support clinical investigation of FT819 in patients with systemic lupus erythematosus (SLE). FT819 is the Company’s off-the-shelf CAR T-cell product candidate that incorporates several novel synthetic controls of cell function, including the integration of a novel CD19-targeted 1XX chimeric antigen receptor (CAR) construct into the T-cell receptor alpha constant (TRAC) locus that is intended to promote uniform CAR expression, enhance T-cell potency, and prevent graft-versus-host disease (GvHD). The multi-center, Phase 1 clinical trial for SLE is designed to evaluate the safety, pharmacokinetics, and anti-B-cell activity of a single dose of FT819 administered following a standard three-day chemotherapy conditioning regimen. The Company is currently conducting study start-up activities at multiple U.S. clinical sites.
Dose Escalation Ongoing in Phase 1 Study for B-cell Lymphoma. The Company’s landmark clinical trial of FT819 for the treatment of relapsed / refractory B-cell malignancies is the first-ever clinical investigation of a T-cell product candidate manufactured from a clonal master iPSC line. The Company is currently enrolling patients in single-dose treatment cohorts at 540 million cells and at 1.08 billion cells using a standard three-day chemotherapy conditioning regimen. Any further clinical development of FT819 for the treatment of B-cell malignancies will be determined by the Company based on safety and activity at these higher dose levels. Clinical data previously presented by the Company from the first 11 patients with relapsed / refractory B-cell lymphoma treated with a single dose of FT819 at up to 360 million cells showed anti-tumor activity including three complete responses and one partial response, CAR T-cell expansion that peaked in the peripheral blood between Days 8 and 11, and a favorable safety profile with no dose-limiting toxicities, no events of any grade of immune effector-cell associated neurotoxicity syndrome (ICANS) or GvHD, and no events of Grade 3 or greater cytokine release syndrome (CRS).
FT825 / ONO-8250 iPSC-derived CAR T-cell Program

Phase 1 Study Initiated with HER2-targeted CAR T-cell for Advanced Solid Tumors. In January, the Company initiated enrollment of a multi-center, Phase 1 clinical trial of FT825 / ONO-8250 under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono). 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 targeting human epidermal growth factor receptor 2 (HER2). Preclinical data of FT825 / ONO-8250 presented at the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting demonstrated that the profile of its novel HER2-targeted antigen binding domain is unique and differentiated from that of trastuzumab, exhibiting similar potency with greater specificity for cancer cells expressing HER2. The Phase 1 study is designed to assess the safety, pharmacokinetics, and activity of a single dose of FT825 / ONO-8250 as monotherapy and in combination with monoclonal antibody therapy in patients with advanced solid tumors.
FT522 iPSC-derived CAR NK Cell Program

First Patient Treated with ADR-armed, CD19-targeted CAR NK Cell Product Candidate. FT522 is the Company’s first product candidate incorporating its proprietary Alloimmune Defense Receptor (ADR) technology, which is designed to reduce or eliminate the need for administration of intense chemotherapy conditioning to patients receiving cell therapies. The multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma is currently enrolling patients in the first three-dose cohort at 300 million cells per dose of Regimen A, which includes administration of chemotherapy conditioning. Subject to clearance of dose-limiting toxicities at this first dose level of Regimen A, enrollment is expected to commence in the first three-dose cohort at 300 million cells per dose of Regimen B without administration of chemotherapy conditioning.
Preclinical Studies Ongoing to Support Expansion into Autoimmunity. The Company is conducting a preclinical assessment of the potential for FT522 to induce CD19+ B-cell depletion across a range of autoimmune diseases, including without administration of intense chemotherapy conditioning to patients. In a disseminated Nalm6 leukemia model comprised of CD19+ target cells resistant to T-cell killing, ADR-armed, CD19-targeted CAR NK cells exhibited robust killing in vivo of CD19+ target cells in the presence of alloreactive T cells, suggesting that FT522 has the potential to deplete CD19+ B cells without administration of intense chemotherapy conditioning to patients. Additional preclinical studies are ongoing with FT522 in combination with monoclonal antibody therapy to assess the potential depletion of both CD19+ B-cell and CD38+ plasma-cell autoantibody-producing lineages.
FT576 iPSC-derived CAR NK Cell Program

Dose Escalation Ongoing in Phase 1 Multiple Myeloma Study. The Company’s multi-center, Phase 1 clinical trial of FT576, its BCMA-targeted CAR NK cell product candidate, is currently accruing patients with relapsed / refractory multiple myeloma in three-dose treatment cohorts as monotherapy as well as in combination with CD38-targeted monoclonal antibody. Using a standard three-day chemotherapy conditioning regimen, the Company has treated six patients at 1 billion cells per dose, with no dose-limiting toxicities and no reports of any grade of CRS, ICANS or GvHD. The study is currently enrolling patients at 2.5 billion cells per dose. Any further clinical development of FT576 for the treatment of multiple myeloma will be determined by the Company based on safety and activity at these higher dose levels.
Fourth Quarter 2023 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of December 31, 2023 were $316.2 million.
Total Revenue: Revenue was $1.7 million for the fourth quarter of 2023, which was derived from the Company’s 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 fourth quarter of 2023, GAAP operating expenses were $49.8 million, including research and development expenses of $31.8 million and general and administrative expenses of $17.9 million. Such amounts included $9.5 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 98.6 million, and preferred shares outstanding were 2.8 million, as of December 31, 2023. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Monday, February 26, 2024 at 5:00 p.m. ET to review financial and operating results for the quarter and full year ended December 31, 2023. In order to participate in the conference call, please register using the conference link here. The live webcast can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.