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PhaseRx announces advancements in treatment for rare liver diseases
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PhaseRx Inc. announced progressions in its product pipeline including its lead product candidate, PRX-OTC, and its positive proof-of-concept data for a second product candidate, PRX-ASL.
PhaseRx is a biopharmaceutical company specializing in life-threatening inherited liver diseases in children. The company’s product candidates offer treatment of two urea cycle disorders (UCDs). The first, PRX-OTC, treats ornithine transcarbamylase deficiency (OTCD). The second candidate, PRX-ASL, is for the treatment of argininosuccinate lyase deficiency (ASLD).
These UCDs are a family of rare metabolic disorders that generally affect children. Caused by an inherited single-gene deficiency, they result in hyperammonemia and can cause irreversible neurological impairment, coma and death. While currently there are treatments that focus on reducing effects of the disease, the only curative option is liver transplant. Drugs in development at PhaseRx hope to replace the missing or defective enzyme that causes UCDs by using intracellular enzyme replacement therapy (i-ERT) to reinstate 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,” Michael Houston, PhD, chief scientific officer of PhaseRx said in a press release.
In 2018, PhaseRx plans to produce phase 2a (single-dose) and 2b (repeat-dose) clinical proof-of-concept data for PRX-OTC.
“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,” Robert Overell, PhD, president and CEO of PhaseRx, said in the release. “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.”
Disclosures: Houston and Overell are employed by PhaseRx.