Mar 19 2018

Fate Therapeutics Announces Additional Clinical Data from Phase 1 Stage of PROTECT Clinical Trial of ProTmune™

No Events of Cancer Relapse

No ProTmune-related Serious Adverse Events Reported by Investigators

SAN DIEGO, March 19, 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 today additional clinical data from the Phase 1 stage of its PROTECT clinical trial of ProTmune™, the Company’s next-generation hematopoietic cell graft. The data is being featured in a poster presentation at the 44th Annual Meeting of the European Society for Blood and Marrow Transplantation being held in Lisbon, Portugal, March 18-21, 2018.

“The primary objective of hematopoietic cell transplantation for cancer patients is disease-free survival. We are very encouraged that no events of cancer relapse have occurred with ProTmune in the Phase 1 stage of PROTECT,” said Chris Storgard, M.D., Chief Medical Officer of Fate Therapeutics. “Our clinical findings underscore the compelling safety profile of ProTmune and suggest that ProTmune has the unique potential to attenuate early, life-threatening events of acute GvHD and promote the curative potential of allogeneic transplant. We continue to follow Phase 1 subjects and look forward to assessing key one-year outcomes with ProTmune, including incidence and severity of chronic GvHD, cancer relapse and disease-free survival.”

Seven adult subjects with hematologic malignancies undergoing matched unrelated donor hematopoietic cell transplantation (HCT) received ProTmune as the hematopoietic cell graft in the Phase 1 stage of PROTECT. As of a February 26, 2018 data cut-off, there have been no events of cancer relapse with a median time on study of 228 days. Additionally, no serious adverse events related to ProTmune have been reported by investigators. The randomized, controlled and double-blinded Phase 2 stage of PROTECT is currently enrolling up to 60 subjects at 14 U.S. centers.

Fate Therapeutics is developing ProTmune as a preventative therapy to reduce the incidence and severity of acute graft-versus-host disease (GvHD) by Day 100 following HCT. Acute GvHD is the leading cause of early morbidity and mortality following allogeneic HCT. Extended use of immunosuppressive agents to treat acute GvHD compromises the anti-leukemia activity of the transplant procedure and significantly increases the risk of cancer relapse and mortality. Additionally, treatment of acute GvHD is ineffective in about half of patients, and these refractory patients have a dismal prognosis with mortality rates in excess of 70%. There are currently no therapies for the prevention of acute GvHD approved by the U.S. Food and Drug Administration.

Day 100 clinical data from the Phase 1 stage of PROTECT were previously presented at the 59thAmerican Society of Hematology Annual Meeting and Exposition in December 2017. All seven subjects receiving ProTmune remained alive and relapse-free during the first 100 days following HCT. Three of the seven subjects experienced acute GvHD during the first 100 days following HCT, all of whom responded to standard-of-care steroid treatment. The median time to resolution of the maximum GvHD grade was seven days [range: 5-8 days].

Three subjects with acute lymphoblastic leukemia (ALL), three subjects with acute myeloid leukemia (AML) and one subject with myelodysplastic syndrome (MDS) received ProTmune as the hematopoietic cell graft in the Phase 1 stage. All subjects are being followed for a period of up to two years post-HCT. Non-relapse mortality deemed not attributable to ProTmune occurred in two subjects (Subject 1 on Day 228 from pulmonary edema; Subject 3 on Day 151 from atrial fibrillation). The remaining five of seven Phase 1 subjects are alive and relapse-free.

PROTECT Clinical Data – Time on Study *
Subject 1 2 3 4 5 6 7
Age / Gender 66 / F 56 / F 66 / F 34 / F 48 / M 56 / M 69 / F
Hematologic Malignancy MDS AML AML ALL ALL ALL AML
CD34+ cell dose (x106/kg) 10.3 4.6 10.9 4.8 3.2 3.0 9.4
CD3+ cell dose (x108/kg) 3.1 1.8 2.6 2.8 2.0 1.2 2.8
Time on Study (Days) 228 343 151 251 243 208 195
ProTmune-related SAEs None None None None None None None
Cancer Relapse-free Yes Yes Yes Yes Yes Yes Yes
Survival No Yes No Yes Yes Yes Yes
* Data is based on a February 26, 2018 data cut-off. The database is not locked, and final data are subject to change.

PROTECT Phase 2 Clinical Trial
The ongoing Phase 2 stage of PROTECT is a randomized, controlled and double-blinded clinical trial assessing the safety and efficacy of ProTmune in up to 60 adult subjects with hematologic malignancies undergoing matched unrelated donor HCT following myeloablative conditioning. Subjects are being randomized, in a 1:1 ratio, to receive either ProTmune as the hematopoietic cell graft or a conventional matched unrelated donor mobilized peripheral blood cell graft. The primary efficacy endpoint of PROTECT is cumulative incidence of Grades 2-4 acute GvHD by Day 100 following HCT, where prospective clinical studies have shown that 40% to 80% of patients undergoing matched unrelated donor HCT experience Grades 2-4 acute GvHD. Additional key endpoints, including rates of chronic GvHD, cancer relapse, and disease-free and overall survival, are also being assessed.

About ProTmune™
ProTmune™ is an investigational next-generation hematopoietic cell graft for the prevention of acute GvHD in patients undergoing allogeneic HCT. ProTmune is manufactured by pharmacologically modulating an allogeneic donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to decrease the incidence and severity of acute GvHD while maintaining the anti-leukemia activity of the graft. ProTmune has been granted Orphan Drug and Fast Track Designations by the U.S. Food and Drug Administration, and Orphan Medicinal Product Designation by the European Commission.

About Allogeneic HCT
There are approximately 30,000 allogeneic HCT procedures performed globally each year according to the Center for International Blood and Marrow Transplant Research. The procedure is performed with curative intent most often for patients with acute leukemias and myelodysplastic syndromes. However, patients face a multitude of life-threatening complications during the initial weeks and months following allogeneic HCT, and only about 50% of patients remain alive and are relapse-free at one year following HCT. The two leading causes of morbidity and mortality are cancer relapse, which occurs in 25% to 30% of patients, and GvHD.

About Acute GvHD
Acute GvHD is a severe immunological disease that commonly arises in patients during the first weeks following allogeneic HCT when newly-transplanted donor immune cells attack the patient’s tissues and organs, resulting in a potentially fatal immune system reaction. Prospective clinical studies have shown that 40% to 80% of patients undergoing matched unrelated donor HCT experience Grades 2-4 acute GvHD, with most incidents occurring by Day 60 following HCT despite the use of standard prophylaxis regimens. Mortality directly attributable to acute GvHD or its treatment occurs in 10% to 20% of patients.

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 therapies using its proprietary induced pluripotent stem cell (iPSC) product platform. This platform uniquely enables the single-cell selection of a precisely engineered iPSC clone and the subsequent creation and maintenance of 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 consistently and repeatedly manufacturing homogeneous cell products in quantities that support the treatment of many thousands of patients in an off-the-shelf manner. 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

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 therapeutic and market potential of ProTmune™, the Company’s advancement of and plans for its clinical investigation of ProTmune, including the Company’s ability to assess key one-year outcomes from the Phase 1 stage of PROTECT and continue the ongoing Phase 2 stage of PROTECT, the ability of ProTmune to prevent, or reduce the occurrence of, graft-versus-host disease, disease relapse or mortality, the potential safety of ProTmune in the treatment of diseases, the timing for receipt of clinical data and success of the Company’s PROTECT clinical trial, and the Company’s development and product registration strategy for ProTmune, including its ability to pursue accelerated registration. 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 cessation or delay of planned development and clinical activities for a variety of reasons (including any difficulties or delay in enrolling subjects in clinical trials or in manufacturing or supplying ProTmune for clinical testing, the occurrence of any adverse events or other results that may be observed during development, or any requirements that may be imposed by regulatory authorities on the manufacture or conduct of clinical trials of ProTmune including those necessary to support product registration), the risk that results observed in prior preclinical studies and early-stage clinical trials of ProTmune may not be observed in ongoing or future studies or clinical trials, the risk that ProTmune may not produce therapeutic benefits or may cause other unanticipated adverse effects, the risk that the Company’s expenditures may exceed current expectations for a variety of reasons, and the risk that the Company may allocate its financial and other resources to programs or product candidates that ultimately have less therapeutic or commercial potential than other product opportunities. 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.

Christina Tartaglia
Stern Investor Relations, Inc.

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Source: Fate Therapeutics, Inc.