On November 8, 2023 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 third quarter ended September 30, 2023 (Press release, Fate Therapeutics, NOV 8, 2023, View Source [SID1234637257]).
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"We achieved several key milestones for our iPSC product platform in oncology and autoimmunity, creating additional opportunities to generate new clinical data across multiple programs during 2024," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We have initiated patient enrollment in our Phase 1 study of FT522, our ADR-armed, CD19-targeted CAR NK cell program, where we intend to assess FT522 with and without conditioning chemotherapy in patients with B-cell lymphoma. In addition, our IND application was cleared by the FDA for FT825/ONO-8250 in solid tumors under our collaboration with ONO Pharmaceutical, which multiplexed-engineered CAR T-cell program incorporates seven synthetic controls of cell function including a novel cancer-specific binding domain targeting HER2. Finally, I am pleased to announce the expansion of our iPSC product platform into autoimmunity with the clearance by the FDA of our IND application for FT819, our off-the-shelf, CD19-targeted CAR T-cell program, in systemic lupus erythematosus."
FT522 iPSC-derived CAR NK Cell Program in B-cell Lymphoma
Phase 1 Study of ADR-armed, CD19-targeted CAR NK Cell Program Open for Enrollment. FT522 is the Company’s off-the-shelf, multiplexed-engineered natural killer (NK) cell product candidate that incorporates five synthetic controls of cell function. It is the Company’s first product candidate armed with its proprietary alloimmune defense receptor (ADR) technology, which is comprised of a synthetic engineered receptor targeting 4-1BB expressed on alloreactive immune cells. In preclinical studies, engagement of ADR-armed CAR NK cells with alloreactive immune cells mitigated rejection, promoted cellular proliferation, and increased anti-tumor activity, indicating that ADR-armed CAR NK cells may be effective without requiring administration of intensive conditioning chemotherapy to patients. The Phase 1 study of FT522 in combination with rituximab for relapsed / refractory B-cell lymphoma (BCL) is designed to assess safety, pharmacokinetics, and activity with and without administration of a standard three-day preconditioning regimen to patients. Enrollment into the first three-dose cohort at 300 million cells per dose has been initiated. The Company is also assessing in preclinical studies the potential of FT522 to induce benefit across a range of autoimmune diseases.
FT825/ONO-8250 iPSC-derived CAR T-cell Program in Solid Tumors
IND Application Cleared by FDA for Multiplexed-engineered, CAR T-cell Program Incorporating Seven Novel Synthetic Controls of Cell Function. Under the Company’s collaboration with ONO Pharmaceutical Co., Ltd. (ONO), the companies are co-developing FT825/ONO-8250, an iPSC-derived CAR T-cell product candidate that incorporates a novel cancer-specific H2CasMab-2 CAR targeting HER2 and is designed to overcome unique challenges in treating solid tumors. The Company’s Investigational New Drug (IND) application for FT825/ONO-8250 was cleared by the U.S. Food and Drug Administration (FDA) in October for conduct of a Phase 1 study in patients with advanced solid tumors. The dose-escalation schema includes two treatment regimens: single-dose FT825/ONO-8250 as monotherapy; and FT825/ONO-8250 in combination with cetuximab. Novel synthetic controls incorporated into the multiplexed-engineered CAR T-cell product candidate include a CXCR2 receptor to promote cell trafficking, a chimeric TGFβ receptor to redirect immunosuppressive signals in the tumor microenvironment, and a high-affinity, non-cleavable CD16a receptor to promote antibody-dependent cellular cytotoxicity. Preclinical data of FT825/ONO-8250, which was presented at the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, demonstrated that the antigen binding profile of H2CasMab-2 is unique and differentiated from that of trastuzumab, exhibiting similar potency with greater specificity for malignant HER2-expressing cells.
FT819 iPSC-derived CAR T-cell Program in Systemic Lupus Erythematosus and B-cell Malignancies
Expansion into Autoimmunity with Phase 1 Study Start-up Ongoing in SLE. In July, the Company’s IND application was cleared by the FDA for clinical investigation of FT819 in patients with systemic lupus erythematosus (SLE), including those with active lupus nephritis (LN) or active SLE without renal involvement. FT819 is the Company’s off-the-shelf, iPSC-derived CAR T-cell product candidate that incorporates several novel synthetic controls of cell function including the integration of a novel CD19-targeted 1XX CAR construct into the T-cell receptor alpha constant (TRAC) locus, which is intended to promote uniform CAR expression, enhance T-cell potency, and prevent graft-versus-host disease. The clinical trial is designed to evaluate the safety, pharmacokinetics, anti-B-cell activity of a single dose of FT819 administered following a standard three-day preconditioning regimen. The FT819 Phase 1 protocol received a favorable review by clinical experts of the Protocol Design Committee of Lupus Therapeutics, an affiliate of the Lupus Research Alliance.
Phase 1 Study Advancing in Single-dose Escalation Cohorts for B-cell Malignancies. The Company’s landmark Phase 1 clinical trial of FT819 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 in BCL and at 360 million cells in chronic lymphocytic leukemia using a standard three-day preconditioning regimen. Clinical data previously presented by the Company from the first 11 patients with relapsed / refractory BCL 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 immune effector-cell associated neurotoxicity syndrome (ICANS) and mild cytokine release syndrome (CRS).
FT576 iPSC-derived CAR NK Cell Program in Multiple Myeloma
Phase 1 Study Accruing Patients in Three-dose Treatment Cohorts. The Company’s Phase 1 study of FT576, its multiplexed-engineered BCMA-targeted CAR NK cell product candidate for relapsed / refractory multiple myeloma, is currently enrolling patients in two, three-dose treatment cohorts at 1 billion cells per dose. The Company has treated three patients as monotherapy as well as two patients in combination with CD38-targeted monoclonal antibody therapy to assess the therapeutic potential of dual-antigen targeting of myeloma cells, with no dose-limiting toxicities reported by investigators in either cohort.
Third Quarter 2023 Financial Results
Cash & Investment Position: Cash, cash equivalents and investments as of September 30, 2023 were $349.7 million. In addition, as of September 30, 2023, cash receivables from the Company’s collaboration with ONO were $1.5 million.
Total Revenue: Revenue was $1.9 million for the third 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 third quarter of 2023, GAAP operating expenses were $53.2 million, including research and development expenses of $34.3 million and general and administrative expenses of $18.9 million. Such amounts included $10.1 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 September 30, 2023. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Wednesday, November 8, 2023 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 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.