PhaseRx has developed a therapeutics platform applicable to a host of diseases. Our initial focus is on three urea cycle disorders (UCDs) as first targets as we believe we can achieve life changing impacts within a reasonable timeframe:

  • PRX-OTC for Ornithine Transcarbamylase Deficiency (OTCD)
  • PRX-ASL for ArgininosuccinateLyase Deficiency (ASLD)
  • Argininosuccinatesynthetase 1 (ASS1) deficiency

PhaseRx believes its approach helps mitigate the costs and risks associated with biotech drug development, which are often attributable to a poor understanding of the disease biology and the mechanism of action of the drug candidate, as well as the need for large clinical trials and uncertainty about approvable endpoints. We believe our costs and risks will be lower because:

  • The biology of our disease programs is well understood: our target diseases are caused by a bad gene inherited from parents that results in a defective or missing enzyme inside the liver cell.
  • The mechanism of action of our therapeutics is clear: we are simply replacing the missing enzyme using our intracellular enzyme replacement therapy approach.
  • Clinical proof of concept can be obtained in relatively few patients: We believe we can obtain clinical proofs of concept in very few (10-20) patients, and ultimately achieve product registration based on 30-40 patients, as opposed to hundreds or even thousands of patients needed to obtain clinical proof of concept and registration in other diseases, such as cancer, diabetes, etc.

There is an approvable endpoint in lead programs: We believe lowering of blood ammonia is an approvable endpoint for our lead urea cycle disease (UCD) programs, since an ammonia scavenger was approved by FDA in 2013 for the treatment of the UCDs on the basis of lowering blood ammonia. Also, ammonia can be quickly and readily measured by a blood assay.


Future Indications Include:

  • Organic Acidemias
  • Glycogen Storage Diseases
  • Porphyria
  • Hyperoxalurea
    • Phenylketonuria
    • Tyrosinemia
    • Wilson’s Disease