Kamada Ltd. announced that the company has submitted to the U.S. Food and Drug Administration (FDA) for review a proposed pivotal Phase 3 protocol for its proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy (Inhaled AAT) for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). The proposed Phase 3 pivotal study is intended to treat AATD subjects with inhaled AAT at a dose of 80 mg once daily for a period of two years, with a placebo arm at a 2:1 ratio with cross over to the treatment arm following a period of 12 months. In parallel, a concurrent Intravenous AAT (AAT IV) arm will be evaluated for two years. The study is planned to include approximately 200-300 patients, and is expected to measure lung function as a primary endpoint and lung density as a secondary endpoint. As previously announced, the company has completed a Phase 2/3 clinical trial in Europe and a Phase 2 clinical trial in the United States (U.S.) with its Inhaled AAT for the treatment of AATD. The U.S. Phase 2 study, which was double-blind and placebo-controlled, met its primary endpoint and the results indicated that patients treated with it's Inhaled AAT, in both dosage arms (80 mg/day and 160 mg/day), demonstrated a significant increase of antigenic AAT, and of Anti-Neutrophil Elastase inhibitory Capacity (ANEC) in the endothelial lining fluid (ELF) levels in the lungs compared to the placebo group. When the company presented the data from the European Phase 2/3 study to the FDA, the agency expressed concerns and questions about that data, primarily related to the safety and efficacy of Inhaled AAT for the treatment of AATD and the risk/benefit balance to patients based on that data. Those questions and concerns will need to be resolved before the FDA will allow the company to proceed with additional clinical development of Inhaled AAT in the U.S., including the planned Phase 3 pivotal study. In order to address the FDA comments, in April 2017, the Company submitted to the agency the results of the U.S. Phase 2 data together with a proposed Phase 3 synopsis. The FDA has provided the company with guidance for further development of the synopsis and requested that the company submit a complete proposed study protocol for the next phase prior to enabling the company to continue clinical development in the U.S.