Fate Therapeutics Reports Positive Interim Data from its Phase 1 Study of FT516 in Combination with Rituximab for B-cell Lymphoma

3 of 4 Patients Evaluable for Efficacy in Dose Escalation Cohorts 2 and 3 Show Objective Response, with 2 Patients Achieving Complete Response

No Observed Events of Any Grade of Cytokine Release Syndrome, Immune Effector Cell-Associated Neurotoxicity Syndrome, or Graft-vs-Host Disease

Six Doses of FT516 were Well-tolerated with No FT516-related Grade 3 or Greater Adverse Events Reported by Investigators

Management to Host Virtual Event Entitled “The Power of hnCD16” Today at 4:30 PM Eastern Time

SAN DIEGO, Dec. 04, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today announced positive interim data from the Company’s dose escalation Phase 1 study of FT516 in combination with rituximab for patients with relapsed / refractory B-cell lymphoma. FT516 is the Company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor, which is designed to maximize antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells.

“We are highly encouraged by these Phase 1 data, which clearly demonstrate that off-the-shelf, iPSC-derived NK cells can drive complete responses for cancer patients and that our proprietary hnCD16 Fc receptor can effectively synergize with and enhance the mechanism of action of tumor-targeted antibodies,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “Importantly, the safety profile of FT516 continues to suggest multiple doses of iPSC-derived NK cells can be administered in the outpatient setting, and supports potential use across multiple lines of therapy, including as part of early-line CD20-targeted monoclonal antibody regimens, for the treatment of B-cell lymphoma.”

As of a November 16, 2020 data cutoff, three patients in the second dose cohort of 90 million cells per dose and one patient in the third dose cohort of 300 million cells per dose were available for assessment of safety and efficacy. All four patients were heavily pre-treated, having received at least two prior rituximab-containing regimens. Each patient received two 30-day treatment cycles, with each cycle consisting of fludarabine and cyclophosphamide lympho-conditioning followed by three once-weekly doses of FT516, IL-2 cytokine support, and rituximab.

Safety Data

All four relapsed / refractory patients were administered FT516 in an outpatient setting with no requirement for inpatient monitoring. No dose-limiting toxicities, and no cases of any grade of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, or graft-versus-host disease, were observed. The multi-dose, two-cycle treatment regimen was well-tolerated with no FT516-related grade 3 or greater adverse events reported by investigators. In addition, no evidence of anti-product T- or B-cell mediated host-versus-product alloreactivity was detected, supporting the potential to safely administer up to six doses of FT516 in the outpatient setting without patient matching. All grade 3 or greater treatment emergent adverse events were not related to FT516 and were consistent with lympho-conditioning chemotherapy and underlying disease.

Activity Data

Three of four relapsed / refractory patients achieved an objective response, including two complete responses (CR), following the second FT516 treatment cycle as assessed by PET-CT scan per Lugano 2014 criteria. A CR was achieved in one patient with diffuse large B-cell lymphoma (DLBCL) who was most recently refractory to a rituximab-containing treatment regimen, and a CR was achieved in one patient with follicular lymphoma (FL) who had previously been treated with four rituximab-containing treatment regimens. Notably, in one patient for which an interim tumor assessment showed a partial response following the first FT516 treatment cycle, the response deepened to a CR following administration of the second FT516 treatment cycle, suggesting that additional FT516 treatment cycles can confer clinical benefit.

M = million; CR = Complete Response; PR = Partial Response; PD = Progressive Disease

As of November 16, 2020 database entry. Data subject to cleaning and source document verification.

¹ Day 29 of the second FT516 treatment cycle as assessed per Lugano 2014 criteria

Dose escalation is continuing in the current dose cohort of 300 million cells per dose in combination with rituximab, and a fourth dose cohort of 900 million cells per dose in combination with rituximab is planned. The Company previously reported that two patients treated in the first dose cohort of 30 million cells per dose in combination with rituximab showed a protocol-defined response assessment of progressive disease. No events of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, or graft-versus-host disease were observed in either patient.

About Fate Therapeutics’ iPSC Product Platform

The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About FT516

FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Scientists from the Company have shown in a peer-reviewed publication (Blood. 2020;135(6):399-410) that hnCD16 iPSC-derived NK cells, compared to peripheral blood NK cells, elicit a more durable anti-tumor response and extend survival in combination with anti-CD20 monoclonal antibodies in an in vivo xenograft mouse model of human lymphoma. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About Fate Therapeutics, Inc.

Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Company’s immuno-regulatory product candidates include ProTmune™, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Company’s iPSC-derived NK cell product candidates, including FT516, its ongoing and planned clinical studies, and the expected clinical development plans for FT516. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in studies of its product candidates, including preclinical studies and clinical trials of any of its product candidates, will not be observed in ongoing or future studies involving these product candidates, the risk that the Company may cease or delay clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties or delays in subject enrollment in current and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:

Christina Tartaglia

Stern Investor Relations, Inc.

212.362.1200