PHP-303 (AATD) (deactived)



PHP-303 is a novel neutrophil elastase inhibitor for the treatment of nonalcoholic steatohepatitis (NASH). PHP-303 inhibits neutrophil elastase, a major inflammatory factor involved in the progression of NASH, improving insulin resistance and inflammatory responses, the main causes of NASH.

Development Status

  • pH Pharma acquired PHP-303 from Bayer AG in March 2017. Prior to acquisition, Bayer AG had conducted clinical studies on bronchiectasis, evaluating human safety and target inhibition.
  • pH Pharma is developing PHP-303 as a novel therapy for NASH indication, having completed Phase 1b SAD/MAD studies (US) in 2019.
  • pH Pharma has expanded PHP-303 indication to include the genetic disease alpha-1 antitrypsin deficiency (AATD) and completed a Phase 2 clinical study in EU.

What is alpha-1 antitrypsin deficiency (AATD)?

Alpha-1 antitrypsin deficiency (AATD) is a genetic disease that causes a lack of the protein, alpha-1 antrypsin (A1AT) due to the inability to release and accumulate antiprotease from the liver. The symptoms of AATD include emphysema, cryptogenic cirrhosis, portal hypertension and other liver conditions. The current AATD treatment market reaches over USD 1 billion, and it is expected to grow up to USD 1.8 billion.

(Reference: Market Insights, Epidemiology and Market Forecast-2015; 2017 data, Kamada)