Israeli-based therapeutics company Kamada has reported positive top-line results of its US Phase II clinical trial of inhaled Alpha-1 Antitrypsin (AAT) therapy to treat Alpha-1 Antitrypsin Deficiency (AATD).
AAT is developed as a highly purified product in liquefied form composed of proteins extracted and purified from human plasma based on the company’s patented platform technology.
It also consists of other plasma-derived immune globulins.
AAT is a protein derived from human plasma with immunomodulatory, anti-inflammatory, tissue-protective and antimicrobial properties.
The US Phase II clinical trial was designed as a double-blind, placebo-controlled study intended to test the safety and efficacy of AAT.
It involved 36 AATD patients who were treated Kamada’s AAT for inhalation through the eFlow device for 12 weeks during the double-blind period.
After completing the double-blind phase, 26 patients qualified to enter into an additional 12-week, open-label extension study with the active drug for a further assessment of safety and tolerability.
The study was primarily focused on determining the antigenic AAT levels and Anti-Neutrophil Elastase inhibitory (ANEC) levels in the lung, as well as additional anti-proteolitic and anti-inflammatory biomarkers.
Results suggested that AAT led to an increase in endothelial lining fluid (ELF) AAT antigenic level compared to placebo while ensuring safety.
Additionally, it has also resulted to an increase in the ELF ANEC level, which represents the AAT combating further damage by neutrophil elastase.
Primary investigator of study professor Mark Brantly said: “Based on the results of this study, it is clear that inhaled AAT is the most effective mode of treatment for reaching the primary sites of potential lung injury, and restoring AAT inhibitory capacity.”
“I look forward to the start of a pivotal study in the US to confirm these results.”
A previously held European Phase II/III clinical study also suggested the safety profile of Kamada’s inhaled AAT.
According to Kamada CEO Amir London, the company is planning to initiate a pivotal US study to further validate the data.