Kamada Submits Proposed Phase 3 Protocol to FDA for Inhaled Alpha-1 Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency Disease
REHOVOT, Israel, July 20, 2017 (GLOBE NEWSWIRE) -- Kamada Ltd. (KMDA) (KMDA), a plasma-derived protein therapeutics company focused on orphan indications, today 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.
“The proposed study design is based on Kamada’s experience with previous Inhaled AAT studies. Our Phase 2 US study showed increases in AAT levels in the lungs to the upper normal range with a daily dose of 80 mg of inhaled AAT. The primary endpoint of lung function was selected based on the results of our European Phase 2/3 study, and the secondary endpoint is consistent with prior AAT IV studies,” said Dr. Naveh Tov, MD, PhD, Kamada’s Vice President of Clinical Development & Medical Director for Pulmonary Diseases.
As previously announced, Kamada 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 Kamada’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.
On July 18, 2017, Kamada submitted a full study protocol, which the Company believes addresses the remaining concerns and questions identified by the FDA.
“The submission of our proposed Phase 3 protocol represents an important accomplishment for our inhaled AAT program,” said Amir London, Kamada’s Chief Executive Officer. “Following the guidance received from the FDA, we believe we have appropriately designed our proposed Phase 3 clinical trial protocol. Based on the collective clinical data generated to date, we continue to believe that Inhaled AAT has the potential to be a safe and effective treatment for AATD. If approved to move forward by the FDA, we intend to proceed with a U.S. Phase 3 pivotal clinical trial as expeditiously as possible for the potential benefit of the AATD patient population.”
About eFlow® Technology and PARI Pharma
The Company’s Inhaled AAT therapy is delivered by an investigational eFlow® Nebulizer System developed by PARI Pharma specifically for Kamada. The optimized device uses eFlow Technology to enable highly efficient aerosolization of medication via a vibrating, perforated membrane that includes thousands of laser-drilled holes. Compared with other nebulization technologies, eFlow Technology produces aerosols with a very high density of active drug, a precisely defined droplet size and a high proportion of respirable droplets delivered in the shortest possible period of time. Combined with its quiet mode of operation, small size, light weight and battery use, eFlow Technology reduces the burden of taking daily, inhaled treatments.
