Alpha 1 Antitrypsin Deficiency

May 4, 2015

Kamada issued results of a phase II/III study of inhaled alpha-1 antitrypsin (AAT) to treat AAT deficiency (AATD). For lung function, bronchial airflow measurements FEV1 (L) rose significantly in AAT treated patients and decreased in placebo treated patients (+15ml for AAT v. -27ml for placebo, a 42ml difference, p=0.0268). There was a trend toward better FEV1% predicted (0.54% for AAT v. -0.62% for placebo, a 1.16% difference, p=0.065). FEV1/SVC% rose significantly in AAT treated patients and decreased in placebo treated patients (0.62% for AAT vs. -0.87% for placebo, a 1.49% difference, p=0.0074). For lung function change at week 50 v. baseline, there was a trend toward reduced FEV1 (L) decline (-12ml for AAT v. -62ml for placebo, a 50ml difference, p=0.0956). There was a trend toward a reduced decline in FEV1% predicted (-0.1323% for AAT v. -1.6205% for placebo, a 1.4882% difference, p=0.1032). FEV1/SVC% rose significantly in AAT treated patients and decreased in placebo treated patients (0.61% for AAT v. -1.07% for placebo, a 1.68% difference, p=0.013). The company plans to submit the Marketing Authorization Application (MAA) to the EMA within the coming year. The company also plans to initiate discussions this year with the FDA on the regulatory pathway for registration of the product in the U.S.