Kamada Presents Updated Data from Phase 2 Clinical Trial of Inhaled Alpha-1-Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency at 2017 American Thoracic Society International Conference
NESS ZIONA, Israel, May 24, 2017 (GLOBE NEWSWIRE) -- Kamada Ltd. (KMDA) (KMDA.TA), a plasma-derived protein therapeutics company focused on orphan indications, today announced that a poster comprising updated data from the Company’s U.S. Phase 2 clinical trial of its proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD) was presented by Professor Mark Brantly, M.D., Vice Chair of Research, Department of Medicine, Chief Division of Pulmonary, Critical Care and Sleep Medicine, Professor of Medicine, Molecular Genetics and Microbiology at the University of Florida College of Medicine and Alpha One Foundation Research Professor, at the 2017 American Thoracic Society (ATS) International Conference, being held May 19-24 in Washington, D.C. AATD is an orphan disease currently treated by intravenous AAT augmentation therapy.
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-proteolytic 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 (160 mg/day) to further assess safety and tolerability. Previously announced top-line data from this trial indicated that patients treated with Kamada’s inhaled AAT demonstrated a significant increase in endothelial lining fluid (ELF), AAT antigenic and ANEC levels compared to the placebo group.
The updated data included in the poster presentation demonstrated that ELF-AAT, neutrophil elastase (NE)-AAT and ANEC complexes concentration significantly increased in subjects receiving the 80 mg and 160 mg doses, (median increase of 38.7 neutrophil migration (nM), p-value<0.0005 (80 mg/day, n=12), and median increase of 46.2 nM, p-value<0.002 (160 mg/day, n=10)). This is a specific measure of the anti-proteolytic effect in the ELF and represents the amount of NE that was broken down by AAT. The increase in levels of functional AAT was six times higher (160 mg per day) than is achievable with intravenous (IV) AAT. In addition, ELF NE decreased significantly. Also, the 80 mg data demonstrated a significant reduction in the percentage of neutrophils. Finally, aerosolized M-specific AAT was detected in the plasma of all subjects receiving inhaled AAT, consistent with what was seen in the Phase 2/3 clinical trial of inhaled AAT conducted by Kamada in the EU.
“Inhaled AAT significantly increases functional AAT levels in the ELF versus placebo and versus what can be achieved with IV AAT,” said Naveh Tov, M.D., Ph.D., Kamada’s Vice President, Clinical Development and Medical Director for Pulmonary Diseases. “Inhaled AAT also restores protease anti-protease homeostasis and reduces the percentage of neutrophils and NE concentration in the lower respiratory tract of AAT deficient individuals. Finally, detection of normal M-specific AAT in the plasma of study subjects indicates that inhaled AAT passed from the alveolar compartment and the interstitial space. Collectively, we believe that these findings, as well as the previously announced top-line data from this trial, and the clinically significant results of our European Phase 2/3 study, support the use of inhaled AAT for the treatment of AATD.”
“The positive updated data from this clinical trial build on the previously obtained compelling top-line results,” said Professor Brantly, the Primary Investigator of the clinical trial. “These most recent encouraging results further support the use of inhaled AAT as a safe and effective treatment for AATD. I continue to be excited about the prospect of conducting a pivotal clinical trial in the U.S. to further evaluate inhaled AAT in the clinic.”
“We are currently discussing a regulatory pathway in the U.S. for the Company’s inhaled AAT with the U.S. Food and Drug Administration,” said Amir London, Kamada’s Chief Executive Officer. “We expect to have an approved Investigational New Drug Application to conduct a pivotal Phase 3 study prior to the end of the year, which would allow us to initiate the clinical study in the U.S. in 2018. In Europe, we anticipate a regulatory decision from the European Medicines Agency in regards to our inhaled AAT for the treatment of AATD in the second half of 2017.”
