A phase 2, single-arm, open-label extension study, evaluating the long -term safety and clinical efficacy of inbrx-101 in adults with alpha-1 antitrypsin deficiency (aatd) emphysema (inbrx101)

This is a Phase 2. Multicentre (multiple study sites), single arm (one investigational medicine), open label extension (you and your study doctor will know the medication you receive) study to evaluate the long-term safety and efficacy of the study medication INBRX-101 in patients with alpha-1 antitrypsin deficiency (AATD) emphysema (lung disease, due to damage).Participants with AATD emphysema have significantly lower blood level of the protein alpha 1 antitrypsin (AAT), AAT has an important function protecting the lungs. INBRX-101 is expected to raise the AAT level in the blood to ‘normal’ physiological levels.Current A1PI therapies fail to achieve normal levels. INBRX-101 differs from other AATD treatments as it has a special modification designed to prevent loss of the AAT activity over time, meaning the AAT is more stable and so remains in the lungs to function forlonger, creating a clear unmet need for INBRX-101. There will be 3 cohorts: cohort 1 will consist of participants (ppts) with newly diagnosed AATD emphysema. Cohort 2 will be ppts who completed the parent study, IRAS ID 1008715, and received Zemaira. Cohort 3 will also be ppts who completed the parent study, but received INBRX-101.INBRX-101 will be administered intravenously (IV, into the vein) every 3 weeks at a dose level of 120 mg/kg. The treatment duration in this study is 3 years. After completing the study treatment, patients will proceed into the safety follow-up phase of the study after the End of Trial Visit. Assessments will include assessment of vital signs as well as blood/urine tests, electrocardiogram, questionnaires, lung function tests, bronchoscopy (if in cohort 1) and a CT scans.