SOURCE: Targazyme, Inc.

March 17, 2015 15:24 ET

Targazyme, Inc. and M.D. Anderson Cancer Center Receive FDA IND Clearance for Phase I/II Study of TZ101-Treated Regulatory T Cells to Prevent Graft vs Host Disease in Patients Eligible for Hematologic Stem Cell Transplantation

SAN DIEGO, CA--(Marketwired - March 17, 2015) - Targazyme, Inc., a clinical stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for a variety of cell therapies including stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted IND Clearance to M.D. Anderson Cancer Center to start enrolling patients in a Phase I/II clinical study to evaluate the safety and efficacy of TZ101-fucosylated regulatory T cells (Tregs) in preventing and reducing the severity and incidence of graft vs. host disease (GVHD) in patients eligible for hematologic stem cell transplant.

GVHD is a serious, life-threating complication of stem cell transplantation, affecting over 60% of all patients undergoing allogeneic stem cell transplantation. This clinical study hopefully will affirm Targazyme's novel treatment approach. TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a high percentage of these patients and is very difficult to manage clinically. If the efficacy of TZ101-treated Tregs is demonstrated in GVHD, then the company expects the treatment to be useful in a number of autoimmune diseases such as rheumatoid arthritis, multiple sclerosis and others.

"This Phase I/II study will hopefully translate into the clinic and give our patients the impressive increases in survival seen in animal models and also the persistence/activity of TZ101-treated Tregs in the preclinical models of GVHD," said Dr. Lynne A. Bui, Senior Vice President of Development at Targazyme. 

"This FDA IND clearance for TZ101-treated regulatory T cells for the prevention and treatment of GVHD provides additional validation of our innovative products to potentially improve efficacy outcomes for patients undergoing stem cell transplantation and immunotherapy," said Lynnet Koh, CEO and Chairman of the Board of Targazyme. "We are excited that the study is open for enrollment at M.D. Anderson Cancer Center to start treating patients with this promising modality for preventing GVHD in patients undergoing stem cell transplantation."

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 stem cell medicine, immunotherapy for cancer and autoimmune diseases, gene therapy and regenerative medicine.

In addition to conducting immunotherapy proof of concept trials, Targazyme is also gearing up for its global product registration trial in hematopoietic stem cell transplantation. The company's clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf kits 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 a number of world-wide patents, multiple FDA orphan drug designations and 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 M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, University of California Los Angeles Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego Medical Center, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center. Learn more at http://www.targazyme.com.

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