Fate Therapeutics Inc., a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators to treat orphan diseases, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug Application (IND) amendment to evaluate Prohema in pediatric patients undergoing hematopoietic stem cell (HSC) transplantation for the treatment of hematologic malignancies. The FDA’s clearance of the IND amendment allows the company to expand its clinical investigation of Prohema, which to date has only been administered to adults, in pediatric patients as young as one year of age. The company plans to initiate enrollment of the PROMPT (Prohema for the treatment of hematologic Malignancies in PediaTric patients undergoing single umbilical cord blood transplantation) study, which is designed to enroll up to 18 patients, between the ages of 1 and 18, at three leading U.S. pediatric transplant centers in mid-2014.
“Each year, over 3,500 children in the U.S. are diagnosed with leukemia, many of whom may ultimately require HSC transplantation,” commented Pratik Multani, chief medical officer of Fate Therapeutics. “Building upon our encouraging clinical experience with Prohema in adult patients, we look forward to expanding our clinical program to pediatric patients with life-threatening forms of leukemia and other hematologic malignancies. Our goal is to deliver a pharmacologically-optimized HSC therapeutic that can support rapid and durable hematologic and immunologic reconstitution, and enable the curative potential of HSC transplantation in patients across a wide range of ages and a broad spectrum of life-threatening malignant and rare genetic disorders.”
Prohema (16, 16-dimethyl prostaglandin E2, or dmPGE2, modulated cord blood), which is currently being evaluated in a Phase 2 clinical trial in adults, is the company’s lead pharmacologically-modulated HSC therapeutic. In 2010, the FDA granted Prohema orphan designation for the enhancement of stem cell engraftment in patients undergoing allogeneic HSC transplantation. The PROMPT study is an open-label Phase 1b clinical trial of Prohema in pediatric patients undergoing single umbilical cord blood transplantation for the treatment of various hematologic malignancies, such as acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), following myeloablative conditioning. Consistent with the recently initiated Phase 2 clinical trial in adults, the manufacture of Prohema in the PROMPT study will utilize the company’s nutrient-rich media formulation, which has been shown in in vivo preclinical studies to improve HSC viability as well as HSC engraftment by more than two-fold as compared to a standard cell processing media. The primary endpoint of the PROMPT study is safety as assessed by neutrophil engraftment. The study will also evaluate various parameters of efficacy including additional measures of neutrophil engraftment, platelet engraftment, rates of graft failure, acute graft versus host disease and serious infection, and disease-free and overall survival.
Date: April 23, 2014