
“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
Source: Fate