VivoSeq
Respiratory

Alpha-1 Antitrypsin Deficiency

27

Pipeline Programs↗

8

Active Trials↗

17

Companies Involved↗

Alpha-1 Antitrypsin Deficiency is an emerging landscape with 27 programs across 17 companies. The most active players are Inhibrx Biosciences, Inc, Krystal Biotech, Inc., Roche. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

52/100

Pipeline Velocity

19/100

Mech. Concentration

25/100

Completion Rate

68/100

Attrition Rate

22/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

I

Inhibrx Biosciences, Inc

1 program

Phase 1K

Krystal Biotech, Inc.

1 program

Phase 1Roche logo

Roche

1 program

Phase 1P

Parkedale

1 program

Phase 1Novartis logo

Novartis

2 programs

Phase 1D

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

93%

Small molecule

25 of 27 classified programs — the dominant modality here.

7%ALL OTHERSRNA therapy14%Gene therapy14%

Small molecule leads; the ring shows all 27 classified programs, each share of the total. Modality classified for 88% of 32 pipeline programs.

Analysis

Trial Status

27

clinical trials

Completed13 · 48%
Recruiting6 · 22%
Terminated4 · 15%
Withdrawn2 · 7%
Active, not recruiting1 · 4%
Not yet recruiting1 · 4%

Clinical Trials

Trial Activity

27 trials

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Recruiting·Sanofi logoSanofi·Phase 2·NCT05897424·185 enrolled
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
Recruiting·UUniversity of Alabama at Birmingham·Phase 4·NCT07135427·30 enrolled
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Recruiting·KKrystal Biotech, Inc.·Phase 1·NCT06049082·15 enrolled
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
Recruiting·AAIRNA Corporation·Phase 1·NCT07431112·54 enrolled
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Recruiting·Beam Therapeutics logoBeam Therapeutics·Phase 1/2·NCT06389877·106 enrolled
An Open-Label, Multicenter, Randomized, Non-Inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Weekly Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Patients With Alpha1-Antitrypsin Deficiency Compared to Corresponding Standard 60 mg/kg/Week and 120 mg/kg/Week Doses of Intravenous Alpha1-Proteinase Inhibitor (5%)
Recruiting·Grifols Therapeutics LLC logoGrifols Therapeutics LLC·Phase 3·NCT07555483·40 enrolled
Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
Active, not recruiting·RRenJi Hospital·Phase 1·NCT07193615·18 enrolled
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
Not yet recruiting·TTessera Therapeutics, Inc.·Phase 1/2·NCT07227207·72 enrolled
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
Completed·—·Phase 2·NCT03114020·27 enrolled
Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)
Completed·UUniversity of Florida·N/A·NCT01810458·109 enrolled
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Completed·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 2·NCT03946449·16 enrolled
Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
Completed·—·Phase 2·NCT03008915·15 enrolled
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Completed·BBeacon Therapeutics·Phase 2·NCT01054339·9 enrolled
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
Completed·IInhibrx Biosciences, Inc·Phase 1·NCT03815396·31 enrolled
Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency
Completed·Grifols Therapeutics LLC logoGrifols Therapeutics LLC·Phase 2·NCT01213043·30 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)