Clinical Data
The U.S. Phase 2 clinical trial was a double-blind, placebo-controlled study evaluating the safety and efficacy of AAT by inhalation in 36 AATD patients. Patients were treated with Kamada’s AAT for inhalation (80 mg/day or 160 mg/day) or placebo via the eFlow device for 12 weeks during the double-blind period. Primary efficacy measures included antigenic AAT levels and Anti-Neutrophil Elastase inhibitory (ANEC) levels in the lung as well as additional anti-proteolitic and anti-inflammatory biomarkers. Following this double-blind period, eligible patients (total of 26) entered an additional 12-week open-label extension study with the active drug (160mg/day) to further assess safety and tolerability. AATD patients treated with Kamada’s inhaled AAT demonstrated a significant increase in endothelial lining fluid (ELF) AAT antigenic level compared to the placebo group [median increase 4551 nM, p-value<0.0005 (80 mg/day, n=12), and 13454 nM, p-value<0.002 (160mg/day, n=12)]. These results are more than twice the increase of ELF antigenic AAT level (+2600 nM) observed in Kamada’s previously completed intravenous (IV) AAT pivotal study (60mg/kg/week). Antigenic AAT represents the total amount of AAT in the lung, both active and inactive.


In addition, ELF ANEC level also increased significantly [median increase 2766 nM, p-value<0.0005 (80mg/day) and 3557 nM., p-value<0.004 (160 mg/day)]. The increase in ELF ANEC level was also more than twice that demonstrated in Kamada’s previously completed IV AAT pivotal study. The ANEC level represents the active AAT that can counterbalance further damage by neutrophil elastase.

Kamada’s inhaled AAT also demonstrated a strong safety profile, adding to the safety data generated in the Company’s previously completed European Phase 2/3 clinical study of Inhaled AAT for the treatment of AATD.  There were no differences seen in safety parameters (treatment emergent adverse events, serious adverse events, etc.) between the placebo and treatment groups (both 80mg and 160mg) during the double-blind and open-label extension periods. Two patients discontinued the study during the double-blind period, including one in the treated group, which was determined to be unrelated to treatment, and one in the placebo group.