PhaseRx, Inc. (NASDAQ:PZRX), a biopharmaceutical company developing treatments for life-threatening inherited liver diseases in children, today announced positive results from the company’s single escalating dose response study in non-human primates. The administration of mRNA delivered using the company’s proprietary Hybrid mRNA TechnologyTM platform in a large animal model was safe and well tolerated at all dose levels tested – an important step prior to advancing its lead mRNA drug candidate towards clinical development. The company has previously shown therapeutic effect in its lead program, PRX-OTC, in the preclinical model of Ornithine Transarbamylase Deficiency (“OTCD”), including both lowering of blood ammonia and rescue of 100% of treated mice.
In the non-human primate study, human Erythropoietin (“hEPO”) mRNA dosed at 0.1, 0.3 and 1.0 mg/kg delivered with the Hybrid mRNA TechnologyTM platform demonstrated dose-dependent increases in levels of hEPO protein with a very favorable safety profile. Elevations of hEPO protein levels at three orders of magnitude above normal physiological levels were achieved. In addition, a commensurate increase was also seen in reticulocyte count, a cell type expressed in response to hEPO which was robust and seen in all dose groups. The formulation was extremely well tolerated in non-human primates with no statistically significant dose-related changes in liver enzymes or cytokines, including IL-6, TNF-a, IFN-g, IL-12, or IP-10.
“We have now demonstrated that our mRNA delivery system is safe in a large animal study,” said Robert W. Overell, Ph.D., president and chief executive officer. “These data together with previously demonstrated efficacy in the mouse model increase our confidence in the safety and the therapeutic potential of our mRNA delivery technology and our lead program, PRX-OTC. We plan to complete IND-enabling studies in 2017 and initiate human studies of PRX-OTC in 2018 in OTC deficient patients.”
About PhaseRx
PhaseRx is a biopharmaceutical company dedicated to developing products for the treatment of children with inherited enzyme deficiencies in the liver using intracellular enzyme replacement therapy (i-ERT). PhaseRx’s initial product development focus is on urea cycle disorders, a group of rare genetic diseases that generally present before the age of twelve and are characterized by the body’s inability to remove ammonia from the blood with potentially devastating consequences for patients. The company’s i-ERT approach is enabled by its proprietary Hybrid mRNA TechnologyTM platform. PhaseRx is headquartered in Seattle. For more information, please visit www.phaserx.com.
