On August 5, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the second quarter ended June 30, 2020 (Press release, Fate Therapeutics, AUG 5, 2020, View Source [SID1234562909]).
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"Early clinical data from our FT596 program are very encouraging, as we observed a partial response in a heavily-pretreated patient with refractory diffuse large B-cell lymphoma at the first dose level without any reported events of cytokine release syndrome, neurotoxicity or graft-versus-host disease. Additionally, the safety, tolerability, and immunogenicity data across our off-the-shelf NK cell programs continue to suggest that multiple doses of iPSC-derived NK cells can be administered to a patient without matching," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We continue to be pleased with our pace of innovation, where the recent clearances of our IND applications by the FDA for FT538, the first-ever CRISPR-edited iPSC-derived cell therapy, and for FT819, the first-ever iPSC-derived CAR T-cell therapy, continue to demonstrate our unique ability to rapidly bring multiplexed engineered, off-the-shelf NK cell and T-cell cancer immunotherapies to patients. In addition, we successfully launched our Janssen collaboration with strong momentum, bringing together Janssen’s proprietary tumor-targeting antigen binders and our industry-leading iPSC product platform to develop novel off-the-shelf CAR NK and CAR T-cell immunotherapies for hematologic malignancies and solid tumors."
Clinical Programs
Partial Response Reported with FT596 Monotherapy at First Dose Level. In June, the Company reported Day 29 protocol-defined response assessments for the first two patients treated with FT596, the Company’s off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-cancer modalities: a proprietary CD19-targeting chimeric antigen receptor (CAR), a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor, and an IL-15/IL-15 receptor fusion. Each patient had previously been treated with at least four lines of therapy for diffuse large B-cell lymphoma (DLBCL), and was administered a single dose of FT596 as a monotherapy at the first dose level (30 million cells). The first patient had progressive disease, having most recently relapsed following treatment with an FDA-approved CD19-targeting CAR T-cell therapy. The second patient had a partial response, having most recently been refractory to an experimental donor-derived NK cell regimen. The FT596 response assessment in this patient showed a greater than 70% reduction in metabolic activity and a greater than 50% reduction in tumor size as assessed by PET-CT scan. No dose-limiting toxicities, no FT596-related serious adverse events (SAEs), and no events of cytokine release syndrome, neurotoxicity, or graft-versus-host disease were reported by investigators in either patient.
Enrollment Initiated with FT596 in Combination with Rituximab for B-cell Lymphoma. In addition to assessing FT596 as a monotherapy, the FT596 Phase 1 clinical trial includes the administration of FT596 in combination with rituximab, which enables dual-antigen targeting of CD19 and CD20 through the product candidate’s CAR and hnCD16 receptors, respectively. Enrollment into this dose-escalating regimen has been initiated at 30 million cells for patients with relapsed / refractory B-cell lymphoma.
Second FT516 IND Application Cleared by FDA for Advanced Solid Tumors. In April, the U.S. Food & Drug Administration (FDA) cleared an investigational new drug (IND) application for FT516, the Company’s off-the-shelf NK cell cancer immunotherapy derived from a clonal master iPSC line engineered to express hnCD16 Fc receptor. The Phase 1 study, which is sponsored by investigators from the Masonic Cancer Center, University of Minnesota, is intended to assess intraperitoneal administration of three once-weekly doses of FT516, each with IL-2 cytokine support, for women with recurrent ovarian cancer. The Company is preparing to initiate a multi-dose Phase 1 clinical trial of FT516 in combination with avelumab, a checkpoint inhibitor therapy targeting PD-L1, for patients with advanced solid tumors, and is continuing to enroll a multi-dose Phase 1 clinical trial of FT516 as a monotherapy for patients with acute myeloid leukemia (AML) and in combination with CD20-directed monoclonal antibodies for patients with advanced B-cell lymphoma.
Evidence of Clinical Activity Observed with FT500 for Advanced Solid Tumors. In May 2020, the Company announced the completion of the dose-escalation stage of the FT500 Phase 1 clinical trial for advanced solid tumors, having enrolled three patients at the second dose level of FT500 in combination with checkpoint inhibitor therapy. Each of these three patients was previously treated with at least four lines of therapy, and most recently was refractory to, or had relapsed on, checkpoint inhibitor therapy. Evidence of clinical activity was observed in two patients, with one patient having a 58% reduction in the size of target lesions prior to discontinuing treatment during the second 30-day cycle due to non-target lesion formation and one patient having stable disease following completion of the second 30-day cycle. No dose-limiting toxicities, no FT500-related SAEs or Grade ≥3 adverse events, and no events of cytokine release syndrome, neurotoxicity, or graft-versus-host disease were reported by investigators in the dose-escalation stage of the study. The Company is enrolling the dose-expansion stage of the FT500 Phase 1 clinical trial and intends to treat up to 15 patients who are refractory to, or have relapsed on, checkpoint inhibitor therapy, administering three once-weekly doses of FT500 at 300 million cells per dose, each with IL-2 cytokine support, for up to two 30-day cycles in combination with the same checkpoint inhibitor on which the patient failed or relapsed.
IND Application Cleared by FDA for FT538, the First-ever CRISPR-edited, iPSC-derived Cell Therapy. In May 2020, the FDA cleared the Company’s IND application for FT538, its off-the-shelf NK cell product candidate derived from a clonal master iPSC line engineered with three functional components to enhance innate immunity: a novel hnCD16 Fc receptor; an IL-15/IL-15 receptor fusion; and the elimination of CD38 expression. The Company is preparing to initiate a multi-center Phase 1 clinical trial of three once-weekly doses of FT538 as a monotherapy for patients with relapsed / refractory AML and in combination with daratumumab for patients with relapsed / refractory multiple myeloma.
IND Application Cleared by FDA for FT819, the First-ever iPSC-derived CAR T-cell Therapy. In June 2020, the FDA cleared the Company’s IND application for FT819, an off-the-shelf allogeneic CAR T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master iPSC line, and is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy including: a novel 1XX CAR signaling domain, which has been shown to extend T-cell effector function without eliciting exhaustion; integration of the CAR transgene directly into the T-cell receptor alpha constant (TRAC) locus, which has been shown to promote uniform CAR expression and enhanced T-cell potency; and complete bi-allelic disruption of T-cell receptor expression for the prevention of graft-versus-host disease, a potentially life-threatening complication associated with allogeneic T-cell therapy. The Company is preparing to initiate a multi-center Phase 1 clinical trial of FT819 for patients with chronic lymphocytic leukemia, acute lymphoblastic leukemia, or B-cell lymphoma. Each indication will enroll independently and evaluate three dose-escalating treatment regimens: a single dose of FT819; a single dose of FT819 with IL-2 cytokine support; and three fractionated doses of FT819.
Investigator-initiated Clinical Trial of FT516 for COVID-19 Opened to Enrollment. Investigators from the Department of Medicine, Division of Infectious Diseases and International Medicine, University of Minnesota are currently enrolling a Phase 1 clinical trial of FT516 in up to 20 patients with Coronavirus Disease 2019 (COVID-19) at high risk of developing critical life-threatening illness.
Preclinical Programs
Four Anti-Tumor Modalities of FT576 for Multiple Myeloma Highlighted at ASGCT (Free ASGCT Whitepaper). The Company presented new preclinical data for FT576, its off-the-shelf, iPSC-derived, multi-antigen targeted CAR-BCMA NK cell product candidate for multiple myeloma, at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Virtual Annual Meeting in May. In a serial re-stimulation cytotoxicity assay against an MM1.R myeloma cell line, FT576 was shown to be resistant to anti-CD38 monoclonal antibody induced fratricide and to have enhanced cytotoxicity both as a monotherapy and in combination with daratumumab as compared to daratumumab alone. The presentation "Generation of a Novel Single Cell-Derived Multi-Engineered Master Pluripotent Cell Line as a Renewable Source of Off-The-Shelf Multi-Antigen-Targeting NK Cell Immunotherapy for Multiple Myeloma" was selected by ASGCT (Free ASGCT Whitepaper) as an outstanding poster award winner.
New CAR MICA/B Solid Tumor Program Announced. At ASGCT (Free ASGCT Whitepaper) in May, the Company presented initial preclinical data from its new CAR MICA/B solid tumor program, which targets the pan-tumor associated stress antigens MICA and MICB. While these stress-inducible cell-surface proteins are selectively expressed at high levels on many solid tumors, shedding of these proteins by cancer cells is a common mechanism of immune evasion. The preclinical data demonstrated that the Company’s novel CAR MICA/B construct targets a specific epitope that uniquely prevents antigen shedding to overcome tumor escape (Science, DOI: 10.1126/science.aao0505).
Corporate Highlights
Strategic Collaboration Formed with Janssen for Novel iPSC-derived Cell-based Cancer Immunotherapies. In April, the Company entered into a global collaboration and option agreement with Janssen Biotech, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop iPSC-derived CAR NK and CAR T-cell product candidates targeting up to four tumor-associated antigens. In entering into the collaboration, the Company received $100 million including $50 million in an upfront cash payment and $50 million from the purchase by Johnson & Johnson Innovation – JJDC, Inc. (JJDC) of the Company’s common stock. The Company is eligible to receive payments of up to $3.0 billion upon the achievement of certain development, regulatory and commercial milestones, plus tiered double-digit royalties on worldwide net sales of products targeting the antigens, and has the right to elect to co-commercialize each product candidate in the U.S. and share equally in profits and losses in the U.S., subject to its payment of certain clinical development costs and adjustments in milestone and royalty payments.
Completed $201 Million Common Stock Offering. In June, the Company closed an underwritten public offering of 7.1 million shares of its common stock at a public offering price of $28.31 per share. In connection with the offering, JJDC purchased an additional 1.8 million newly-issued shares of the Company’s common stock in a private placement at the offering price.
Second Quarter 2020 Financial Results
Cash & Investment Position: Cash, cash equivalents and investments as of June 30, 2020 were $533.4 million.
Total Revenue: Revenue was $5.5 million for the second quarter of 2020, which was derived from the Company’s collaborations with Janssen and Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $26.7 million for the second quarter of 2020, which includes $4.4 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $7.5 million for the second quarter of 2020, which includes $2.9 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 86.8 million, and preferred shares outstanding were 2.8 million, as of June 30, 2020. Each preferred share is convertible into five common shares.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Wednesday, August 5, 2020 at 5:30 p.m. ET to review financial and operating results for the quarter ended June 30, 2020. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 8295527. The live webcast can be accessed under "Events & Presentations" in the Investors & Media 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.