PhaseRx (PZRX)

PhaseRx is an early-clinical stage biotech company and first publicly traded mRNA therapy pure play that leverages its proprietary hybrid mRNA technology to develop intracellular enzyme replacement therapy (i-ERT) for treating inherited, single-gene disorders with the therapeutic action sites located specifically inside of a cell in the liver. PZRX’s hybrid mRNA technology consists of a two component system that separates transport from cell delivery affording it the safety advantage of avoiding activation of innate immunity and hepatocyte-specific delivery. PZRX has initially targeted therapies in urea cycle disorders (UCDs) indications with a focus on ornithine transcarbamylase deficiency (OTCD), argininosuccinate lyase deficiency (ASLD) and argininosuccinate synthase 1 deficiency (ASS1D). PZRX has already demonstrated promising preclinical study results in PRX-OTC as a potential treatment for OTCD, and expects to further develop PRX- ASLD and PRX- ASS1D as the other two indications. The initial product group could potentially treat >90% urea cycle disorders. Promising preclinical POC data of presumptive lead product, PRX-OTC include hyperammonemia reduction, prolonged survival and lack of innate immunity activation. Going forward, PZRX plans to conduct more preclinical studies and is scheduled to potentially file an IND in 4Q17, likely for PRX-OTC for Phase IIa and IIb studies. Top-line results of the two studies are expected in 1H18 and 2H18. In addition to UCDs, PZRX could also employ i-ERT potentially to treat other liver specific orphan indications. Together, PhaseRx plans to employ its hybrid mRNA technology platform to develop therapies for its in-house pipeline and for prospective partners.