Experimental drug for rare lung disease shows early promise but trial halted

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: (Part A - Healthy Volunteers) * Male or female healthy volunteers 18-50 years of age * Written informed consent * Body mass index between 18.0 and 28.0 kg/m2 * 12-lead electrocardiogram (ECG) at Screening and pre-dose assessment with no clinically significant abnormalities * Non-pregnant/non-nursing females * Non-smoker for at least one year with current non-smoking status confirmed by urine cotinine * Normal lung function (or not clinically significant per investigator assessment) based on spirometry and diffusion capacity of lung for carbon monoxide (DLCO) according to American Thoracic Society (ATS) - European Respiratory Society (ERS) criteria * Highly effective, double barrier contraception (both male and female partners) during the study and for 3 months following the dose of ARC-AAT * Willing and able to comply with all study assessments and adhere to protocol schedule * Suitable venous access for blood sampling * No abnormal finding of clinical relevance at screening * Normal AAT level (Part B-Patients) - As for Part A with the following exceptions: * Male or female patients 18-70 years of age * Confirmed diagnosis of homozygous alpha 1-protease inhibitor deficiency (PiZZ genotype) not receiving alpha-1 antitrypsin augmentation therapy for more than 4 weeks * BMI between 18.0 and 35.0 kg/m2 * Non-smoker for at least three years with current non-smoking status confirmed by urine cotinine Exclusion Criteria: (Part A-Healthy Volunteers) * Current regular smoker of cigarettes or cigars or was a regular smoker over the past 1 year * Recent (within last 6 weeks) transfusion of fresh frozen plasma, platelets, or packed red blood cells, or anticipated need for transfusion during study * Acute signs of hepatitis/other infection within 4 weeks of screening and/or baseline * Concurrent anticoagulants * Use of dietary and/or herbal supplements that can interfere with liver metabolism within 7 days of screening * Use of any drugs known to induce or inhibit hepatic drug metabolism within 14 days prior to study treatment * Depot injection/implant of any drug other than birth control within 3 months prior to study treatment * Diagnosis of diabetes mellitus or history of glucose intolerance * History of poorly controlled autoimmune disease or any history of autoimmune hepatitis * Human immunodeficiency virus (HIV) infection * Seropositive for hepatitis B virus (HBV) or hepatitis C virus (HCV), and/or history of delta virus hepatitis * Uncontrolled hypertension (blood pressure \> 150/100 mmHg) * History of cardiac rhythm disturbances * Family history of congenital long QT syndrome or unexplained sudden cardiac death * Symptomatic heart failure (per New York Heart Association \[NYHA\] guidelines) * Unstable angina, myocardial infarction, severe cardiovascular disease, transient ischemic attack (TIA) or cerebrovascular accident (CVA) within past 6 months * History of malignancy within last 5 years except adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer. * History of major surgery within 3 months of screening * Regular use of alcohol within 1 month prior to screening (i.e., more than fourteen units of alcohol per week) * Evidence of acute inflammation, sepsis or hemolysis or clinical evidence of lower respiratory tract infection * Diagnosis of significant psychiatric disorder * Use of illicit drugs (such as cocaine, phencyclidine \[PCP\] and crack) within 1 year prior to screening or positive urine drug screen * History of allergy or hypersensitivity reaction to bee venom * Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study * Clinically significant history/presence of any gastrointestinal pathology, unresolved gastrointestinal symptoms, liver or kidney disease * Other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs * Any clinically significant history/presence of poorly controlled neurological, endocrinal, cardiovascular, pulmonary, hematological, immunologic, psychiatric, metabolic or other uncontrolled systemic disease * Blood donation (500 mL) within 7 days prior to study treatment * History of fever within 2 weeks of screening * Concomitant medical/psychiatric condition or social situation that would affect compliance or result in additional safety risk * Excessive exercise/physical activity within 3 days of screening or enrollment or planned during the study * History of thromboembolic disease, stroke within 6 months of baseline, and/or concurrent anticoagulant medication(s) (Part B-Patients) - As for Part A with the following exceptions: * History of major surgery within 2 months of Screening * Forced expiratory volume at one second (FEV1) at baseline \< 60% * AATD patients with liver elastography score \> 11 at Screening