SAN DIEGO, July 20, 2016

Targazyme, Inc. a clinical-stage biopharmaceutical company developing novel enzyme technologies and products to improve clinical efficacy outcomes for cancer immunotherapy and stem cell and solid organ transplantation, announced today that it has enrolled the first patient in a Phase 1/2 TZ 101-treated regulatory T cell clinical study. This Phase 1/2 clinical study will evaluate the efficacy and safety of TZ101-treated regulatory T cells in the prevention and treatment of acute and chronic Graft-Versus-Host-Disease (“GVHD”) in stem cell transplants. This clinical milestone will hopefully translate into the clinic the recent preclinical findings published in the journal Blood demonstrating the effectiveness of ex vivo treatment of regulatory T-cells with TZ101 in the prevention of GVHD.

GVHD can be a life-threatening complication of hematopoietic stem cell and solid organ transplantation that affects approximately 188,000 patients a year annually in the United States (source: Statista, 2015). Regulatory T-cells are an important component of immune surveillance and mounting response to GVHD and other autoimmune processes.

“Based on the findings published in the journal Blood, we believe that TZ101 can potentially reduce patient morbidity and mortality from GVHD, which remains a difficult to treat complication in approximately 50% of patients, as well as its potential application in solid organ transplants.” said Lynne A. Bui, MD, Chief Medical officer of Targazyme. ‘The application of TZ101-treated regulatory T-cells extends beyond GVHD into the treatment of autoimmune diseases such as lupus nephritis and Crohn’s disease as well.”

“The initiation of the Phase 1/2 study with TZ 101-treated regulatory T-cells in the stem cell transplant setting is an important clinical milestone for our evaluation of TZ101 as a potential treatment for patients suffering from GVHD and life-threatening autoimmune diseases,” said Lynnet Koh, Chief Executive Officer of Targazyme. “This clinical study builds on available clinical data that TZ101 improves time to absolute neutrophil count and platelet recovery in patients undergoing hematopoietic stem cell transplants, and together, with our second product TZ102, provides proof of concept that our pipeline of products are enabling technologies for improving efficacy outcomes for various cells such as T cells, natural killer cells, and hematopoietic, cardiac and neural stem cells, that are used to prevent and treat a variety of different diseases for which there is a high unmet medical need.”

About Targazyme, Inc.

Targazyme Inc. is a San Diego-based, clinical-stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy outcomes for cell therapy, immunotherapies for autoimmune diseases and cancer, stem cell transplantation, gene therapy and regenerative medicine.
The Company’s clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf biologic products used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice. The Company has received worldwide patents, multiple FDA orphan drug designations, has an open investigational new drug application (IND) with multiple ongoing clinical studies and a Phase 3 Special Protocol Assessment (SPA) with the FDA, and numerous major medical/scientific awards and grants.

Targazyme has partnerships and collaborations with Kyowa Hakko Kirin and Florida Biologix, as well as various medical research institutions including the University of Texas MD Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, UCLA Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center. Learn more at

Contact Information:
Chloe Le