Inhaled Alpha-1 antitrypsin (AAT) therapy fails endpoints in Phase III trial but has positive effects on lung function-Kamada

Kamada Ltd. a plasma-derived protein therapeutics company focused on orphan indications, announced results from the complete analysis of the European Phase II/III clinical study of its inhaled Alpha-1 antitrypsin (AAT) therapy for the treatment of Alpha-1 antitrypsin deficiency (AATD). Following a complete analysis of the study data, the Company confirms that the study�s primary endpoint of �Time to the first moderate or severe exacerbation event� did not show a statistically significant difference between inhaled AAT and placebo in the Intent-to-Treat (ITT) population, as reported in May 2014. The Company also reports that the study did not show statistically significant differences between inhaled AAT and placebo in the secondary exacerbation endpoints measured in the ITT population.

Despite not meeting the primary or secondary endpoints for the ITT population, lung function parameters, including Forced Expiratory Volume in One Second (FEV1) % of Slow Vital Capacity (SVC), FEV1 % predicted, FEV1 (liters) and Diffusing capacity (DLCO), which were collected to support safety endpoints, showed concordance of a potential treatment effect in the reduction of the inflammatory injury to the lung that is known to be associated with a reduced loss of respiratory function. These effects were most apparent in the frequently exacerbating patients (the �Most Frequent Exacerbators�), who represented more than 70% of those enrolled in the study.

Kamada continues with plans for EMA submission based on concordance of exacerbation data and positive lung function differences. Chiesi has EU rights.