SOURCE: Targazyme, Inc.

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October 24, 2016 19:00 ET

Targazyme, Inc. Receives FDA Clearance for a Phase II Haplo-Identical Transplant Study of Patients Undergoing Bone Marrow Transplantation for Treatment of Hematologic Malignancies

SAN DIEGO, CA--(Marketwired - October 24, 2016) - Targazyme Inc., a clinical stage biopharmaceutical company developing novel enzyme technologies and products to improve clinical efficacy and cost of care 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 begin enrolling patients in a Phase II clinical study to evaluate the safety and efficacy of TZ101-fucosylated bone marrow stem cells from haplo-identical donors in cancer patients.

The transplantation of blood-forming stem cells from bone marrow is an accepted treatment to restore the body's ability to make blood and immune cells and is a treatment for various cancers like leukemia, lymphoma and some types of anemia. Recent medical advances have made possible the use of a haploidentical donor who is usually a 50% match to the recipient. Targazyme's product (TZ101) has the potential to improve the outcomes of this lifesaving treatment by improving the ability of TZ101 treated stem cells to home, adhere and engraft into the bone marrow, accelerating hematopoietic recovery, reducing both opportunistic infections and ICU/hospital stay for cancer patients undergoing the haplo-identical transplants.

"Enhancing bone marrow stem cell engraftment with a product like TZ101 will improve clinical outcomes for patients with serious, life-threatening cancers and other disorders for which hematopoietic stem cell transplant is prescribed," said Dr. Gheath Alatrash, Assistant Professor, Department of Stem Cell Transplantation, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX.

"This FDA IND clearance for a phase 2 TZ101 haplo-identical transplant study is an important clinical milestone to help us drive towards our initial target product label of accelerating hematopoietic recovery post hematopoietic stem cell transplantation" 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

Targazyme Inc. is a San Diego-based, clinical-stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy and cost of care 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. 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.

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