The phase 3 InnovAATe study has shown promising safety data, leading inclusion criteria to be revised to include those with severe airflow limitations, such as patients with alpha-1 antitrypsin deficiency (AATD).
The expanded potential patient population is part of Kamada’s trial on the safety and efficacy of inhaled alpha-1 antitrypsin (AAT).
Earlier this year, the study was expanded to new sites in Europe, and a total of 30 patients have now been enrolled.
AATD is a progressive, hereditary condition in which patients lack the AAT protein that protects lung tissue from neutrophil elastase, a destructive enzyme in the body that is released during infections or in response to tobacco smoke. Patients with AATD are at significantly increased risk of chronic obstructive pulmonary disease (COPD), a group of diseases that includes emphysema and chronic bronchitis.
“The strong association between AATD and [COPD] or emphysema suggests that inhaled administration of AAT directly to the lungs may benefit AATD patients,” said Jan Stolk, MD, department of pulmonology, Leiden University Medical Center, The Netherlands. “Based on results published in the European Respiratory Journal in 2019, Kamada’s previously completed randomized placebo-controlled clinical trial suggested that a decline in lung function as measured by FEV1 could be attenuated by daily AAT inhalation.”
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Thus far, no significant adverse events have been reported and no patients in the study have discontinued the therapy prematurely. The independent Data Safety Monitoring Board has, for the fourth time since the study began, recommended the trial continue without changes.
Furthermore, the Data Safety Monitoring Board supports expanding the eligible participant criteria to include those with severe airflow limitation from less than 50% of forced expiratory volume to less than 80%, to less than 40% of forced expiratory volume to less than 80%, which should expedite participant enrollment.
Company officials plan to meet with the US Food and Drug Administration and the European Medicines Agency in the first half of 2023 to review the study’s progress and assess possibilities for an accelerated regulatory pathway for inhaled AAT.
Kamada provides update on recent progress achieved in ongoing pivotal phase 3 clinical trial of inhaled AAT. News release. Kamada, Ltd.; November 7, 2022.