NK100 Shows Clinical Benefit in Treating Advanced Hematologic Malignancies and Solid Tumors

Disease Control Shown in All Subjects Re-treated with Second Dose of NK100

No NK100-related Dose Limiting Toxicities Reported

Submitted Data to FDA Demonstrating FT500 Master iPSC Bank is Free of Adventitious Agents

SAN DIEGO, Nov. 11, 2018 (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, announced new clinical data for FATE-NK100, an investigational, first-in-class, allogeneic donor-derived natural killer (NK) cell cancer immunotherapy, and provided a regulatory update on the development of FT500, a universal, off-the-shelf NK cell product candidate derived from a master induced pluripotent stem cell (iPSC) line, on November 10, 2018.

Twenty heavily pre-treated subjects, each presenting with progressive disease at the time of enrollment, have been treated with FATE-NK100 in the dose-escalation phases of three Phase 1 clinical trials. As of a October 22, 2018 data cutoff, one-month follow-up data were available on fourteen subjects¹, with clinical benefit indicated in seven of these fourteen subjects:

• In the DIMENSION study for the treatment of advanced solid tumors (n=5 in the monotherapy regimen; n=1 in the monoclonal antibody combination regimen¹), one subject at the second dose level (1-3x10⁷ cells per kg) and two subjects at the third dose level (3-10x10⁷ cells per kg) treated with a single intravenous infusion of FATE-NK100 in the monotherapy regimen had stable disease at one month. These two subjects at the third dose level were each subsequently treated with a second dose of FATE-NK100 and remain on study (3.1 and 5.0 months, respectively) with ongoing disease control. The study is currently enrolling at the third dose level in the monotherapy regimen, at the second dose level in the cetuximab combination regimen (1-3x10⁷ cells per kg) and at the run-in dose level in the trastuzumab combination regimen (1x10⁶ cells per kg).
• In the APOLLO study for the treatment of recurrent ovarian cancer (n=4), one subject treated with a single intraperitoneal infusion of FATE-NK100 at the second dose level (1-3x10⁷ cells per kg) had stable disease at one month. The subject was subsequently treated with a second dose of FATE-NK100 and maintained disease control for 6.2 months. The study is currently enrolling at the third dose level (3-10x10⁷ cells per kg).
• In the VOYAGE study for the treatment of refractory or relapsed acute myelogenous leukemia (n=4), all three subjects treated with a single intravenous infusion of FATE-NK100 at the second dose level (1-3x10⁷ cells/kg) showed complete clearance of leukemic blasts in the bone marrow and achieved a morphologic leukemia-free state at Day 14 following treatment. Each of these three subjects received a single dose of FATE-NK100 only, and the anti-leukemic response in each of these subjects was transient. The study is currently enrolling at the second dose level.

No dose limiting toxicities related to FATE-NK100 were reported. One serious adverse event related to FATE-NK100 was reported (Grade 3: abdominal pain) in the APOLLO study.

“The safety and clinical benefit observed with a single infusion of FATE-NK100 as a monotherapy in heavily pre-treated cancer patients, including in refractory AML patients that have high leukemic blast burden in the marrow and in advanced solid tumor patients with progressive disease, are encouraging,” said Sarah Cooley, M.D., Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation at the University of Minnesota and the lead investigator of the VOYAGE study. “We are particularly excited that a repeat dose of FATE-NK100 was well-tolerated and showed persistence. Importantly, all three subjects re-treated with a second dose have demonstrated disease control. These data provide compelling proof-of-concept for FATE-NK100 and support earlier intervention with NK cell therapy using a multi-dose treatment cycle.”

In addition, the Company announced that adventitious agents testing of the master iPSC bank for the clinical production of FT500, a universal, off-the-shelf NK cell product candidate, has been completed. The FT500 master iPSC bank was found to be free of adventitious agents as determined by in vivo and in vitro testing. The Company has submitted these results to the U.S. Food and Drug Administration (FDA) in furtherance of the agency’s review of the Company’s FT500 Investigational New Drug (IND) application. Upon allowance by the FDA of the FT500 IND, the Company plans to initiate Phase 1 clinical testing of FT500 as a monotherapy and in combination with checkpoint inhibitor therapy for the treatment of advanced solid tumors. This first-in-human study is expected to evaluate the safety and tolerability of multiple doses of FT500 in multiple dosing cycles.

An updated presentation on the Company’s NK cell cancer immunotherapy franchise can be found under “Events & Presentations” in the Investors and Media section of the Company's website at www.fatetherapeutics.com.

¹ Excludes one subject treated at the run-in dose level in the cetuximab combination regimen (1x10⁶ cells per kg)

About FATE-NK100
FATE-NK100 is an investigational, first-in-class, allogeneic donor-derived natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells, a highly specialized and functionally distinct subset of activated NK cells expressing the maturation marker CD57. Higher frequencies of CD57⁺ NK cells in the peripheral blood or tumor microenvironment in cancer patients have been linked to better clinical outcomes. In August 2017, non-clinical data describing the unique properties and anti-tumor activity of FATE-NK100 were published by Cancer Research (doi:10.1158/0008-5472.CAN-17-0799), a peer-reviewed journal of the American Association of Cancer Research. Three clinical trials of FATE-NK100 are currently being conducted: VOYAGE for the treatment of refractory or relapsed acute myelogenous leukemia; APOLLO for the treatment of recurrent ovarian cancer; and DIMENSION for the treatment of advanced solid tumors, including in combination with monoclonal antibody therapy.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary iPSC product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of 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 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 to treat many patients. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.

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 is pioneering the development of off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology pipeline is comprised of FATE-NK100, a donor-derived natural killer (NK) cell cancer immunotherapy that is currently being evaluated in three Phase 1 clinical trials, as well as iPSC-derived NK cell and T-cell immunotherapies, with a focus on developing augmented cell products intended to synergize with checkpoint inhibitor and monoclonal antibody therapies and to target tumor-specific antigens. The Company’s immuno-regulatory pipeline includes ProTmune™, a next-generation 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 www.fatetherapeutics.com.

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 advancement of and plans related to the Company's product candidates and clinical studies, the therapeutic potential of the Company’s NK cell cancer immunotherapies, including FATE-NK100 and FT500, the Company’s regulatory strategy, and the Company’s plans for its intended clinical investigation of FATE-NK100 and FT500. 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 of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any ongoing or future clinical studies, the risk that the Company may cease or delay preclinical or 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 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), the risk that results observed in prior clinical trials or preclinical studies of FATE-NK100 or FT500 may not be replicated in ongoing or future clinical trials or studies, and the risk that FATE-NK100 or FT500 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
christina@sternir.com

Source: Fate Therapeutics, Inc.