Reports Interim Phase I/IIa Trial Results for TZ101, a Novel Enzyme Treatment to Enhance Stem Cell Targeting/Engraftment for Improved Cancer Patient Clinical Outcomes
SAN ANTONIO, TX DEC 18 2013.
America Stem Cell, Inc. (ASC) today announces that it has changed its name to Targazyme, Inc. (www.old.targazyme.com). The new name reflects the company’s family of enzyme products aimed at improving cell targeting and engraftment for better efficacy outcomes in stem cell and regenerative medicine.
Phase I/IIa results of Targazyme’s lead product, TZ101, were presented at the American Society of Hematology meeting in New Orleans (Popat et al. Abstract #691, Dec 9). The Phase I/IIa clinical trial evaluating TZ101 (formerly ASC-101), entitled “Cord Blood Fucosylation” (NCT01471067), is being led by Dr. Elizabeth Shpall, M.D., Deputy Chair, Department of Stem Cell Transplantation, University of Texas MD Anderson Cancer Center. This study involves a dual umbilical cord blood transplantation evaluating safety and efficacy following treatment of one of the cords with TZ101 prior to infusion in patients with hematologic malignancies and myelodysplastic syndrome.
Results from 16 patients (20 treated to date) show that TZ101 has a good safety profile. There were no observed infusion-related adverse events nor were there adverse events considered related to TZ101. Furthermore, the rate of acute graft versus host disease was similar to historical controls.
For the primary efficacy endpoints, treatment of cord blood with TZ101 led to (1) a reduction in the time to neutrophil recovery with a median of 15 days compared to historical matched control value of 24 days, (2) a reduction in the time to platelet recovery with a median of 35 days compared to historical matched control value of 49 days, and (3) a cumulative incidence of neutrophil and platelet engraftment of 94%.
“Enhancing umbilical cord stem cell engraftment into bone marrow in the dual cord transplant setting will lead to improved clinical outcomes for patients with serious, life-threatening cancers and other disorders for which hematopoietic stem cell transplant is prescribed,” said Dr. Shpall.
“Targazyme was founded with the vision to make stem cell transplants safer and more efficacious for patients undergoing cell therapy, starting with cancer patients. This interim Phase I/IIa human proof of concept patient data demonstrates the potential of Targazyme’s enzyme therapies to transform outcomes for cancer patients undergoing hematopoietic stem cell transplantation. This may translate to additional applications in the fields of immunotherapy and regenerative medicine,” said Lynnet Koh, Founder and Chief Executive Officer of Targazyme.
Targazyme’s lead product, TZ101 (formerly ASC-101), is a kit consisting of an enzyme (fucosyltransferase VI) and its substrate (GDP-fucose), which has received orphan drug designation from the FDA for use in cord blood transplantation. At the point-of-care, cells are incubated with TZ101 and GDP-fucose for 30 minutes at room temperature, and then washed prior to infusion into the patient. TZ101 attaches the sugar fucose to trisaccharide acceptor molecules on the cell surface, completing the synthesis of the tetrasacharride sialyl Lewis X (sLeX). Increasing the amount of sLeX on the surface of cells increases their ability to adhere to selectins, which are cell adhesion molecules that are upregulated at sites of inflammation, ischemia or tissue damage. Therefore, TZ101 treatment enhances the targeting of therapeutic cells to the tissue sites where they are needed most.
About Targazyme, Inc.
Targazyme, Inc. is a privately held biotechnology company based in San Antonio, TX with additional offices in San Diego, CA and Basel, Switzerland. Its key technology platforms (TZ101 and TZ102) are designed to improve the homing and engraftment of a wide variety of therapeutic cells to target tissues. Applications in development include hematopoietic stem cell transplants, immunotherapies for solid tumors, cell therapies to ameliorate graft-versus-host and autoimmune diseases, and cell therapies for ischemic diseases such as myocardial infarction and stroke. 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, University of California San Diego, Sanford-Burnham Medical Research Institute and Indiana University.