SEATTLE, June 21, 2016 /PRNewswire/ -- PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing treatments for life-threatening inherited liver diseases in children, today announced product pipeline advancements including the selection of its lead product candidate, PRX-OTC, for treatment of Ornithine Transcarbamylase Deficiency (OTCD) and positive proof-of-concept data for a second product candidate, PRX-ASL, for treatment of Argininosuccinate Lyase Deficiency (ASLD).
OTCD and ASLD are urea cycle disorders (UCDs), a family of rare metabolic disorders that generally affect children. Each UCD is caused by an inherited single-gene deficiency that results in hyperammonemia (elevated ammonia in the blood), and can lead to irreversible neurological impairment, coma and death. Current treatments for these diseases focus on ameliorating effects of the disease, while the only curative option is liver transplant. PhaseRx is developing drugs to replace the missing or defective enzyme using intracellular enzyme replacement therapy (i-ERT), with the objective of reinstating function of the urea cycle.
"We believe PRX-OTC, which is expected to correct the disease, has a compelling product profile in comparison to the existing therapies for OTC deficiency," said Michael Houston, Ph.D., chief scientific officer of PhaseRx. "In addition, the preclinical data with PRX-OTC and PRX-ASL validates our i-ERT approach."
PhaseRx expects to generate Phase 2a (single-dose) and 2b (repeat-dose) clinical proof-of-concept data for PRX-OTC in 2018, including measurement of blood ammonia. This clinical development plan is supported by positive preclinical data showing that PRX-OTC normalized blood ammonia and resulted in 100% survival in the OTC-spfash mouse model. In this model, mice are defective in the same gene as humans with OTCD, and PRX-OTC replaced the human gene in the mouse.
Preclinical studies in ASLD were conducted using ASL-deficient mice, which are defective in the same gene as humans with ASL deficiency. The new data with PRX-ASL therapy in the ASL-deficient mice showed statistically significant reduction in ammonia levels following two weeks of mRNA treatment.
"We are excited to name PRX-OTC as our lead drug product candidate and look forward to advancing its development for the treatment of this rare genetic disorder," said Robert Overell, Ph.D., PhaseRx's president and chief executive officer. "We are also delighted with the results of the proof-of-concept study in our ASLD program and believe these data show that our platform is applicable to the treatment of other single-gene inherited disorders of metabolism in the liver."
PhaseRx is a leading 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. 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.
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SOURCE PhaseRx, Inc.