ALPHA-1 ANTITRYPSIN DEFICIENCY(AATD)

AATD, a potentially life-threatening rare, genetic condition caused by a lack of alpha-1 antitrypsin (AAT) a protein (antiprotease) that protects the lungs from enzymatic degradation by endogenous proteases (such as NE). The disease manifests as early-onset pulmonary emphysema, caused by irreversible destruction of lung tissue supporting gas exchange. PHP-303 is designed to selectively inhibit NE, a neutrophil enzyme, which is the major protease responsible for the destruction of lung tissue in AATD.

• Currently no cure for AATD
• Existing therapies attempt to reduce the inflammatory process, reduce exacerbations and the eventual tissue/organ destruction. (E.g. Augmentation/infusions and traditional Bronchodilators, steroids etc.)
• Limitations of current therapies include: 1) low lung penetration, 2) cost 3) inconvenient weekly IV infusions, 4) procurement & 5) pathogen risk

There are an estimated 100,000 patients in the United States and 120,000 patients in Europe with severe AATD.