This is a Phase 1/2, multicenter, open-label study conducted in 3 parts (Part A, Part B, and Part C) to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease. Parts A and B are designed to evaluate safety and tolerability and identify the optimal biological dose (OBD) in patients with AATD-associated lung disease (Part A) and/or liver disease (Part B), with optional expansion cohorts to further evaluate a potential OBD. Part C is designed to demonstrate the efficacy and safety of BEAM-302 at the OBD in patients with AATD-associated lung disease with or without liver disease in the pivotal cohort.
Age 18-70 years.
Confirmed PiZZ AATD diagnosis.
Blood total AAT level <11 μM.
Willing to discontinue augmentation therapy as required by protocol.
BMI >30.
Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction.
Clinical evidence of severe bronchiectasis.
Significant liver disease as defined in the protocol.
Single group
BEAM-302
DrugBEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD
Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Absolute blood levels of total AAT
Mean total and functional AAT in blood
Rate of decline in whole-lung density at TLC
Change in COPD exacerbations
Change in FEV1
Change in SGRQ
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