Targazyme, Inc. Receives FDA Clearance For a Phase 2 Study of Pediatric Cancer Patients Undergoing Stem Cell Transplantation For Treatment of Hematologic Malignancies

SAN DIEGO,CA--(Marketwired - March 06, 2017) - Targazyme Inc., a clinical stage biopharmaceutical company developing novel enzyme products to improve clinical efficacy, safety and cost of care outcomes for a variety of cell therapies including stem cell transplantation, cancer immunotherapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted IND Clearance to begin enrolling patients in a Phase 2 clinical study to evaluate the efficacy of TZ101-fucosylated hematopoietic stem cell transplants in pediatric cancer patients.

"This Phase 2 single cord blood pediatric cancer patient study complements and builds upon the encouraging MD Anderson Cancer Center Phase 2 trial efficacy data for adult leukemia, lymphoma and MDS patients'. said Dr. Rosenthal, M.D., M.H.C.M.; Chair of the City of Hope's Department of Pediatrics at City of Hope Cancer Center, Duarte, CA. 'The TZ 101 Phase 2 data reported in the 2015 Blood publication showed a good safety profile and promising efficacy outcomes in terms of time to neutrophil and platelet recovery with additional positive signals in stem cell engraftment rate, reduction in chronic GVHD and overall survival in adult cancer patients. Successful replication of these results in the single cord blood transplant setting in pediatric cancer patients may lower the rate of opportunistic infections, reduce the incidence of chronic GVHD and reduce morbidity, mortality and hospitalization for kids undergoing this life-saving procedure through the use of TZ 101."

"This FDA IND clearance for a phase 2 study of TZ 101 in pediatric cancer patients is an important clinical milestone to help us drive towards our initial target product label of 'reduction in opportunistic infections and GVHD post hematopoietic stem cell transplantation' for both pediatric and adult cancer patients", said Lynnet Koh, Chief Executive Officer of Targazyme. 'This clinical study builds on available data that TZ 101 and TZ 102 improves efficacy outcomes for cell therapy with various cells such as T cells, natural killer cells, hematopoietic stem cells, mesenchymal stem cells that are used to prevent and treat different diseases for which there are high unmet medical needs.'

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, safety and cost of care outcomes for stem cell transplantation, immunotherapies for cancer and autoimmune diseases, 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. Targazyme's fucosylation technology has received numerous medical and scientific awards from institutions such as NIH, NCI, US Treasury, CPRIT, ETF, OCAST, JDRF, Leukemia, Lymphoma Society.

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.