On May 9, 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 first quarter ended March 31, 2024 (Press release, Fate Therapeutics, MAY 9, 2024, View Source [SID1234642989]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We made great progress across three key areas of clinical focus – we treated the first patient with our off-the-shelf FT819 CAR T-cell therapy for autoimmunity, we initiated patient enrollment without conditioning chemotherapy for our ADR-armed FT522 CAR NK cell therapy, and we treated the first patient with our multiplexed-engineered FT825 CAR T-cell therapy for solid tumors," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "As we look toward the middle of the year, we plan to expand clinical development in autoimmunity with our off-the-shelf FT819 and FT522 programs, where we believe our iPSC product platform is highly differentiated and has the potential to overcome numerous challenges that hinder treatment of patients with cell therapies, such as the requirement for apheresis and Cy / Flu conditioning, extended hospitalization, risk of secondary malignancies, and limited access. In addition, while we continue with the preclinical assessment of our next-generation, BCMA-targeted cell product candidates, we do not intend to further advance FT576 into Phase 1 dose expansion in relapsed / refractory multiple myeloma."
FT819 iPSC-derived CAR T-cell Program
First SLE Patient Treated in 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 novel CD19-targeted 1XX chimeric antigen receptor (CAR) construct into the T-cell receptor alpha constant (TRAC) locus (NCT06308978). The first patient, a 27 year-old woman diagnosed with SLE 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 was discharged after a three-day hospital stay without any notable adverse events. In a "first-of-kind" translational assessment using a sample of the patient’s blood obtained prior to administration of conditioning chemotherapy, FT819 induced rapid and potent depletion of the patient’s CD19+ B cells in an ex vivo cytotoxicity assay.
Phase 1 BCM Study Data Establish Proof-of-Concept for Autoimmune Disease. At the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting, the Company today presented preclinical and translational data from its FT819 Phase 1 study in relapsed / refractory B cell malignancies (BCM), which showed that a single dose of FT819 exhibits multiple mechanisms implicated in generating an immune reset in patients with B cell-mediated autoimmune diseases. Clinical observations included: dose-dependent pharmacokinetics, reaching Cmax at Day 8 with persistence through the first two weeks in the periphery; rapid, deep, and sustained CD19+ B cell depletion in the peripheral blood using standard Cy / Flu and alternative conditioning chemotherapy regimens; patient case studies of secondary and tertiary tissue trafficking, infiltration, and activity with CD19+ B cell elimination in tissue; and patient case studies of plasma cell depletion and B cell reconstitution, which showed recovery of naïve B cells with little to no recovery of activated memory B cells or plasmablasts.
Dose-escalation Completed in Phase 1 BCM Study with Further Clinical Development to Focus Exclusively on Autoimmunity. The Company has successfully completed dose escalation in its Phase 1 BCM study, demonstrating safety and tolerability of FT819 at a single dose up to 1.08 billion cells (NCT04629729), and intends to pursue further clinical development exclusively in autoimmunity. 43 heavily pre-treated patients (B cell lymphoma, n=25; chronic lymphocytic leukemia, n=12; and acute lymphocytic leukemia, n=6) 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 immune effector-cell associated neurotoxicity syndrome (ICANS) or graft-versus-host disease (GvHD), and low incidence (14%) of only low-grade cytokine release syndrome (CRS). There were no study discontinuations or deaths related to FT819. Clinical responses were observed across all three histologies. In 17 patients with aggressive large B cell lymphoma, 12 (71%) of whom had previously received autologous CD19-targeted CAR T-cell therapy, the overall response and complete response rates were 47% and 24%, respectively.
FT825 / ONO-8250 iPSC-derived CAR T-cell Program
First Patient Treated 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). The first patient was diagnosed with HER2-positive gastroesophageal junction (GEJ) adenocarcinoma, had progressed after receiving multiple lines of treatment including HER2-targeted therapies, and was administered standard conditioning chemotherapy followed by a single dose of FT825 / ONO-8250 as monotherapy at 100 million cells. 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.
FT522 iPSC-derived CAR NK Cell Program
Conditioning-free Treatment Arm of Phase 1 BCL Study Open for Enrollment. 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), patient enrollment has now been initiated in the first three-dose cohort at 300 million cells per dose without conditioning chemotherapy (Regimen B). Three patients have been treated with conditioning chemotherapy in the first three-dose cohort at 300 million cells per dose (Regimen A). There were no dose-limiting toxicities and no events of any grade of CRS, ICANS, or GvHD, and dose escalation is ongoing at 900 million cells per dose.
ASGCT Presentation Data Show ADR Differentiation and Proof-of-Concept for Autoimmunity. At the ASGCT (Free ASGCT Whitepaper) conference, the Company today presented preclinical data as well as early translational data from its ongoing Phase 1 BCL study. In a novel re-challenge assay using peripheral blood mononuclear cells (PBMCs) from unmatched SLE donors, 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 in Regimen A, 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. The Company intends to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the middle of 2024 for the treatment of various autoimmune diseases with FT522, including without administration of conditioning chemotherapy to patients.
FT576 iPSC-derived CAR NK Cell Program
Dose Escalation Completed in Phase 1 Multiple Myeloma Study. The Company has completed dose escalation in its multi-center, Phase 1 clinical trial of FT576, its BCMA-targeted CAR NK cell product candidate, for relapsed / refractory multiple myeloma (NCT05182073). Using a standard conditioning chemotherapy regimen, 12 patients were treated with a three-dose treatment schedule at 1 billion cells per dose (n=6) or at 2.5 billion cells per dose (n=6) as monotherapy or in combination with CD38-targeted monoclonal antibody therapy. There were no dose-limiting toxicities and no events of any grade of CRS, ICANS or GvHD. Response assessment is ongoing at 2.5 billion cells per dose. Five of six (83%) heavily pre-treated patients achieved a clinical response at 1 billion cells per dose, including two penta-exposed patients treated with FT576 as monotherapy that achieved very good partial responses. The Company is continuing to assess the FT576 Phase 1 dose-escalation dataset and to preclinically evaluate the potential of its next-generation, BCMA-targeted cell product candidates, including for the treatment of multiple myeloma and autoimmune diseases, and does not intend to further advance FT576 into Phase 1 dose expansion in relapsed / refractory multiple myeloma.
First Quarter 2024 Financial Results
Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2024 were $391.1 million, which includes net proceeds from the closing of the Company’s approximately $80 million underwritten offering of common stock at $5.50 per share and approximately $20 million concurrent private placement of pre-funded warrants at $5.499 per pre-funded warrant in the first quarter of 2024.
Total Revenue: Revenue was $1.9 million for the first quarter of 2024, 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 first quarter of 2024, GAAP operating expenses were $53.0 million, including research and development expenses of $32.1 million and general and administrative expenses of $20.9 million. Such amounts included $11.0 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 March 31, 2024. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Thursday, May 9, 2024 at 5:00 p.m. ET to review financial and operating results for the quarter and full year ended March 31, 2024. In order to participate in the conference call, please dial (833) 630-1956 (domestic) and (412) 317-1837 (international). 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.