Baxter Healthcare Corporation and its development partner Arriva Pharmaceuticals, Inc. announced the successful completion of a Phase I clinical trial of their recombinant human Alpha1-Antitrypsin (recombinant AAT) molecule administered by nebulization. Recombinant AAT is currently under investigation for an inherited form of emphysema caused by Alpha 1-Antitrypsin Deficiency, also known as Hereditary Emphysema. The Phase I clinical trial was conducted at four centres in the United States, and evaluated the safety, immunogenicity, and tolerability of the jointly developed recombinant AAT therapy. Results of the trial show that all doses were well tolerated including the maximum amount administered.

“We are very pleased to be making progress in investigating this treatment for patients suffering from Hereditary Emphysema,” said Martin Preuveneers, CEO of Arriva. ”Results of our Phase I study showed that nebulized recombinant AAT was well tolerated in patients with Hereditary Emphysema. The successful completion of this first clinical study is an essential step toward moving forward into further clinical trials”.

The companies’ recombinant AAT is the first AAT product in clinical trials that is produced without the addition of human or animal-derived raw materials in the fermentation process, purification, or final formulation. This recombinant AAT is being developed in a formulation that allows for delivery to the lungs using a nebulizer. Current treatment for this condition involves intravenous infusion of plasma-derived alpha 1-antitrypsin. While the plasma-derived therapy has been shown to be effective, large doses are required to get therapeutic amounts of the protein to the lung where it is most needed.

Hereditary Emphysema is a serious hereditary disorder characterized by low levels of the natural protease inhibitor AAT. Up to 100,000 individuals in the US are affected by this disorder that, in certain cases, leads to excessive inflammation, progressive emphysema, and premature death if left untreated. The only currently available treatment option for individuals with Hereditary Emphysema involves a weekly infusion of plasma-derived AAT.

“Baxter is committed to bringing to market new therapies to address Hereditary Emphysema, where options have been limited,” said Ron Lloyd, vice president, Marketing, Baxter BioScience. “We recently launched Aralast, which is a plasma-derived AAT therapy, to help address some of the needs in the marketplace. In addition, we recognize the potential for a recombinant therapy.” Clinical trials for recombinant AAT are ongoing.