On May 3, 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 first quarter ended March 31, 2023 (Press release, Fate Therapeutics, MAY 3, 2023, View Source [SID1234630913]).
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"Over the first months of 2023, we have sharpened our clinical focus and significantly reduced our operating expenses, creating the necessary cash runway to achieve key milestones across our multiplexed-engineered CAR NK and CAR T-cell pipeline. We sincerely thank our employees whose patience and perseverance have allowed us to emerge through this transition period with a renewed sense of energy, commitment, and drive to bring first-in-class, iPSC-derived cellular immunotherapies to patients with cancer and autoimmune disorders," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We are now well-positioned to clinically assess higher therapeutic exposures for our FT576 BCMA-targeted CAR NK cell program in multiple myeloma and our FT819 CD19-targeted CAR T-cell program in B-cell malignancies. In addition, we aim to bring our FT522 CD19-targeted CAR NK cell program, which incorporates our proprietary ADR technology designed to enhance NK cell potency, extend functional persistence, and resist host immune cell rejection, into clinical development in the second half of 2023 for B-cell lymphoma, and intend to expand its clinical reach to include severe autoimmune disorders. Finally, we are excited to be jointly developing our clinical strategy with ONO Pharmaceutical for FT825/ONO-8250, our HER2-targeted CAR T-cell collaboration program for solid tumors for which we plan to submit an IND application in the second half of 2023."
NK Cell Programs
FT576 BCMA-targeted CAR NK Cell Program Accruing Patients in Multi-dose Escalation Cohorts for Multiple Myeloma. The Company’s Phase 1 study of FT576, its multiplexed-engineered, BCMA-targeted chimeric antigen receptor (CAR) NK cell product candidate for relapsed / refractory multiple myeloma, is currently enrolling two-dose treatment cohorts as monotherapy and in combination with CD38-targeted monoclonal antibody (mAb) therapy at 300 million cells per dose. Upon clearance of the current treatment cohorts, the Company plans to open and assess three-dose treatment cohorts starting at 1 billion cells per dose. At the 2022 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, the Company presented interim Phase 1 clinical data from nine heavily pre-treated patients in the single-dose cohorts, which showed encouraging clinical evidence of BCMA-targeted activity and a favorable safety profile indicating the potential for administration in the outpatient setting. Translational data from the CD38-targeted mAb combination regimen showed rapid and selective depletion of CD38-positive patient immune cells in the peripheral blood and bone marrow that extended through the first month of therapy, indicating that the regimen may uniquely serve to attenuate reconstitution of activated T cells, extend functional persistence of FT576, and enable dual-antigen targeting of myeloma cells.
Initiation of Clinical Assessment of FT522 ADR-armed, CD19-targeted CAR NK Cell Program Anticipated in 2H23. FT522 is the Company’s first product candidate to incorporate its proprietary alloimmune defense receptor (ADR) technology, which has been shown in preclinical studies to increase NK cell potency, enhance functional persistence, and confer resistance to host immune cell allo-reactivity. The Company has recently submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to investigate the safety and activity of FT522 in combination with CD20-targeted mAb therapy in patients with B-cell lymphoma, including without prior administration of intensive conditioning chemotherapy. In addition, the Company is currently conducting preclinical studies to support clinical assessment of FT522 in autoimmune disease, including in combination with CD20- and CD38-targeted mAb therapy, to selectively target and durably deplete pathogenic B cells, plasma cells, and auto-reactive T cells.
T-cell Programs
First-of-kind FT819 Program Advancing in Single-dose Escalation Cohorts for B-cell Malignancies. The Company’s landmark Phase 1 clinical trial of FT819, which is the first-ever clinical investigation of a T-cell product candidate manufactured from a clonal master iPSC line, is currently enrolling patients in single-dose escalation cohorts at 540 million cells in B-cell lymphoma and at 180 million cells in chronic lymphocytic leukemia. At the 2022 ASH (Free ASH Whitepaper) Annual Meeting, the Company presented interim Phase 1 clinical data from eight patients with relapsed / refractory aggressive large B-cell lymphoma treated with a single dose of FT819 ranging from 90 million cells to 360 million cells, which demonstrated a favorable safety profile and objective responses including in patients who were not eligible for or had previously failed autologous CD19-targeted CAR T-cell therapy. FT819 incorporates several novel features 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.
2023 IND Submission Planned for HER2-targeted CAR T-cell Program for Solid Tumors. 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 targeting human epidermal growth factor receptor 2 (HER2)-expressing solid tumors. IND-enabling activities for FT825/ONO-8250 are currently ongoing, and the Company plans to submit an IND application to the FDA in 2023 to jointly conduct with ONO a Phase 1 study for the treatment of patients with HER2-positive solid tumors. The multiplexed-engineered, iPSC-derived CAR T-cell product candidate incorporates seven novel synthetic controls designed to enhance effector cell function and overcome unique challenges in treating solid tumors, including a novel HER2-targeted binding domain with a differentiated targeting profile, a synthetic CXCR2 receptor to promote cell trafficking, a synthetic TGFβ receptor to redirect immunosuppressive signals in the tumor microenvironment, and a synthetic interleukin-7 receptor fusion protein to induce T-cell activation.
Strategic Pipeline Prioritization & Corporate Restructuring
During the first quarter of 2023, in connection with the termination of its collaboration with Janssen Biotech, Inc. (Janssen), the Company discontinued all collaboration activities, including withdrawing an IND application previously allowed by the FDA for a first collaboration product for the treatment of B-cell lymphoma. In addition, following a strategic review of its wholly-owned iPSC-derived NK cell and T-cell programs, the Company focused its operations on advancing its most innovative and differentiated programs and initiated the discontinuation of its FT516, FT596, FT538, and FT536 NK cell product candidates. As part of its corporate restructuring, the Company reduced its workforce to approximately 220 employees.
First Quarter 2023 Financial Results & 2023 Guidance
Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2023 were $412.8 million. In addition, as of March 31, 2023, cash receivables from collaborations were $13.5 million. The Company expects its cash, cash equivalents, and investments to exceed $300 million at year-end 2023.
Total Revenue: Revenue was $59.0 million for the first quarter of 2023, of which $52.3 million was associated with the termination of its collaboration with Janssen and $6.7 million was derived from its ongoing collaboration with ONO. Under the ONO collaboration, a one-time amount of $6.2 million was recorded as revenue for the first quarter of 2023 associated with the Company’s conduct of IND-enabling activities for FT825/ONO-8250, for which ONO exercised its development and commercialization option in November 2022. For each of the remaining three quarters of 2023, the Company expects to recognize approximately $0.8 million in revenue under the ONO collaboration in connection with its conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen.
Total Operating Expenses: For the first quarter of 2023, GAAP operating expenses were $87.6 million, including research and development expenses of $65.6 million and general and administrative expenses of $21.9 million. Such amounts included $11.0 million of non-cash stock-based compensation expense and a one-time charge of $12.9 million for severance and other employee termination-related costs associated with the Company’s corporate restructuring. For the full year ending December 31, 2023, the Company expects its GAAP operating expenses to be between $265 million to $285 million.
Shares Outstanding: Common shares outstanding were 98.2 million, and preferred shares outstanding were 2.8 million, as of March 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, Wednesday, May 3, 2023 at 5:00 p.m. ET to review financial and operating results for the quarter ended March 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.