AZD-9668 (alvelestat) is an oral neutrophil elastase inhibitor being developed as an innovative new therapy for the treatment of alpha-1 antitrypsin deficiency (AATD).

AATD is a genetic disorder that causes a deficiency of alpha-1 antitrypsin. There are approximately 50,000 and 60,000 severe patients in North America and Europe respectively, who are either ZZ or Null.  It can cause pulmonary emphysema, a life-threatening lung disease, resulting in severe shortness of breath and wheeze.  The lung damage in AATD results from the loss of the normally protective effect of alpha-1 antitrypsin against damaging enzymes released during inflammation, specifically neutrophil elastase, that lead to the irreversible destruction of the lungs’ supportive elastic tissues.  The aim is to use AZD-9668 (alvelestat) to inhibit neutrophil elastase activity and prevent further damage to patients’ lungs.

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