SAN DIEGO, April 23, 2015:

Targazyme, Inc., announced today that the U.S. Food and Drug Administration (FDA) and Targazyme have reached concurrence on the company’s Special Protocol Assessment (SPA) application for its Phase 3 registration trial. This pivotal trial is designed to evaluate the efficacy of TZ101-treated cells in patients with hematologic malignancies undergoing hematopoietic stem cell (cord blood) transplantation with the primary outcome measure being time to neutrophil engraftment. The trial is expected to begin in 2016.

“This pivotal Phase 3 multicenter study complements and builds upon the encouraging MD Anderson Cancer Center Phase 2 trial efficacy data for leukemia and lymphoma patients undergoing dual cord blood transplantation” said Dr. Lynne A. Bui, Senior Vice President of Development at Targazyme.  “The TZ 101 Phase 2 data reported at the 2014 American Society of Hematology Meeting and our recent 2015 Blood publication showed a good safety profile and promising efficacy outcomes in terms of time to neutrophil and platelet recovery.  Successful replication of these results in this Phase 3 trial will allow for reduced morbidity and mortality in patients undergoing this life-saving procedure through the use of TZ101.”

“This concurrence from FDA on our SPA enables us to initiate our Phase 3 trial with prior agreement with FDA on the full approval pathway for TZ101 in hematopoietic stem cell transplantation”, said Lynnet Koh, CEO and Chairman of the Board of Targazyme. “This milestone is an important step forward for Targazyme as we drive towards our goal of first FDA product approval followed by potential product label expansion for TZ101, an enabling platform technology for improving efficacy outcomes for stem cell transplantation, immunotherapy, gene therapy and regenerative medicine.

About Special Protocol Assessments

A Special Protocol Assessment is a written agreement with the FDA on the trial design, endpoints and statistical analysis needed to support FDA product approval. It is intended to form the basis for a marketing application and may only be changed through a written agreement between the Sponsor and FDA.

About TZ101

TZ101 is a kit composed of an enzyme (fucosyltransferase VI) and its substrate,(GDP-fucose).  TZ101 is added to cells at point of care in the clinical centers using a quick and easy 30-minute incubation at room temperature.  During this time, the enzyme attaches a sugar (fucose) to the blood stem cells, a process known as fucosylation.  The ‘fucosylated’ cells are then infused into the patient’s blood stream. Fucosylation of the donor cells increases their ability to home and engraft in the patient’s bone marrow, where they will produce ample blood cells for recovery.

About the Phase 3 Trial The study is a randomized, multi-center, Phase 3 trial involving approximately 216 patients with leukemia and lymphoma and will compare engraftment rates in patients receiving TZ101-treated versus untreated cord blood cells in a dual cord blood transplantation. The primary outcome measure is the median time to neutrophil engraftment and secondary outcome measures are median time to platelet recovery, non-relapse mortality and relapse-free survival.

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.

Targazyme is initiating its product registration trial in hematopoietic stem cell transplantation as well as conducting proof of concept trials in immunotherapy for autoimmune disease and for cancer.  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 worldwide 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

Contact Information: Chloe Le