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Mixed Results Reported for Inhaled Emphysema Drug in Pivotal Trial

Study fails to meet primary endpoint

Mixed results have been reported from a phase II/III pivotal clinical study conducted in Europe and Canada of inhaled alpha-1 antitrypsin (AAT) therapy (Kamada Ltd.) for the treatment of AAT deficiency (AATD), also known as inherited emphysema.

The study’s endpoints included the frequency of, time to first, duration of, and severity of exacerbation events.

With regard to the primary endpoint of “time to first moderate or severe exacerbation,” early data did not show any differences between the AAT therapy group and the placebo group. However, in a secondary endpoint, the frequency of severe exacerbations was approximately 50% lower in patients receiving AAT therapy than in those given placebo.

The randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of an inhaled formulation of human AAT in 168 patients with AATD. The patients inhaled 160 mg of human AAT or placebo twice daily via the eFlow device for 50 weeks.

The study’s primary endpoint was the time to the first moderate or severe exacerbation event at 1 year. Secondary endpoints included additional parameters of exacerbation events, pulmonary function tests, and safety. Other exploratory endpoints included computed tomography (CT) densitometry in a subset of subjects and quality-of-life measurements.

Eligible patients in this phase II/III trial were given the option to participate in a 50-week, open-label safety study. Additional data from this study are expected to be used in regulatory submissions.

A phase II clinical trial of inhaled AAT therapy for patients with AATD has been initiated in the U.S.

AATD is a hereditary genetic disorder that can lead to the development of lung and/or liver disease. It is the most common genetic cause of liver disease in children. Adults can also be affected and may develop lung conditions, such as emphysema, as well as liver problems. In late childhood or adulthood, the disorder can be detected because of fatigue, poor appetite, swelling of the abdomen and legs, or abnormal liver tests. Currently, there is no cure for AATD.

Sources: Kamada Ltd.; May 16, 2014; and American Liver Foundation; November 13, 2013.

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