Targazyme Inc. Receives National Cancer Institute SBIR Award for Phase 2 Clinical Study of TZ101-Treated Regulatory T Cell Therapy for Prevention/Treatment of GVHD

SAN DIEGO, CA--(Marketwired - July 27, 2015) - Targazyme, Inc., a clinical-stage biopharmaceutical company developing novel enzyme technologies and products to improve efficacy outcomes for stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today the award of a $2MM SBIR PHASE II grant from the National Cancer Institute (NCI). This award will fund a Phase 2 clinical study at The University of Texas MD Anderson Cancer Center and mechanism studies at the University of Minnesota with TZ101-treated regulatory T cells (Tregs) for the prevention and reduction of graft versus host disease (GVHD) in patients eligible for a hematologic stem cell transplantation.

GVHD is a medical, life-threatening complication of stem cell transplantation. This significant award confirms the importance of Targazyme's novel enzyme treatment approach to prevent and reduce the incidence and severity of GVHD in patients with blood cancers where stem cell transplantation is prescribed. TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a large percentage of these patients and is very difficult to manage clinically.

"This SBIR award will allow us to now translate this novel therapy to the clinic. The impressive increases in survival of animals and the increased persistence and activity of TZ101-treated Tregs in preclinical models of GVHD will hopefully result in reduced morbidity and mortality for our patients," said Dr. Lynne Bui, SVP of Clinical Development at Targazyme.

"The granting of this medical award, FDA IND Clearance and FDA Orphan Drug status for TZ101 for prevention/treatment of GVHD in stem cell transplant patients provides additional validation of our novel platform technologies," said Lynnet Koh, Founder, CEO and Chairman of the Board of Targazyme. "This human proof-of-concept trial with TZ101-treated regulatory T cells will set us on a course towards our goal of preventing and treating graft-versus-host disease for our transplant patients as well as improving immunotherapy efficacy outcomes for patients with a variety of auto-immune diseases."

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 disease and for cancer, gene therapy and regenerative medicine.

The company's clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf 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 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 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, Sloan Memorial Kettering, and New York Blood Center. Learn more at http://www.targazyme.com.