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Phase 1b PROVIDE Study in Pediatric Patients with Inherited Metabolic Disorders Expected to Begin in 4Q14
Fate Therapeutics, Inc. (Nasdaq:FATE), a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators to treat orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug Application (IND) for the clinical development of PROHEMA in pediatric patients undergoing hematopoietic stem cell (HSC) transplantation for the treatment of inherited metabolic disorders (IMDs).
The FDA’s clearance of the IND allows the Company to begin expanding its clinical investigation of PROHEMA into rare, non-malignant disorders. The Company plans to initiate enrollment of the “PROVIDE” trial (PROHEMA ealuation for the treatment of inherited metabolic disorders) in pediatric patients with IMDs in the fourth quarter of 2014.
“For many severe inherited metabolic disorders, including those with central nervous system involvement, cellular enzyme replacement through unrelated donor cord blood transplantation has emerged as a potentially transformative therapeutic intervention,” commented Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. “We believe pharmacologically-optimized HSC therapeutics, such as PROHEMA, hold significant promise for improving outcomes in patients with inherited metabolic and other rare genetic disorders. FDA clearance of the PROVIDE study marks an important step towards our goal of clinically evaluating this novel treatment paradigm in the non-malignant disease setting.”
The PROVIDE trial is designed to enroll up to 12 patients with various forms of IMDs, between the ages of 1 and 18, at up to three leading U.S. pediatric HSC transplant centers. The trial’s inclusion criteria allows for the enrollment of patients with different types of lysosomal and peroxisomal storage diseases such as Hurler and Hunter syndromes, Krabbe disease and various other leukodystrophies, among others. The primary endpoint of the PROVIDE trial is safety as assessed by neutrophil engraftment.
PROHEMA (16, 16-dimethyl prostaglandin E2, or dmPGE2, modulated cord blood), the Company’s lead pharmacologically-modulated HSC therapeutic, is currently in Phase 2 clinical development for the treatment of adult patients with hematologic malignancies undergoing allogeneic HSC transplantation, and the Company intends to initiate enrollment of a Phase 1b clinical trial in pediatric patients with hematologic malignancy in mid-2014.
In 2010, the FDA granted PROHEMA orphan designation for the enhancement of stem cell engraftment in patients undergoing allogeneic cord blood HSC transplantation. In in vivo murine models of allogeneic HSC transplantation, Fate Therapeutics demonstrated that the use of PROHEMA, as compared to unmodulated HSCs, led to a significant increase both in the engraftment of donor HSCs and in the donor-derived expression of enzyme in the brain.
