VivoSeq
Rare Disease

Hereditary Angioedema

38

Pipeline Programs↗

14

Active Trials↗

15

Companies Involved↗

Hereditary Angioedema is a moderately active landscape with 38 programs across 15 companies. The most active players are ADARx Pharmaceuticals, Inc., Shanghai Argo Biopharmaceutical Co., Ltd., Ionis Pharmaceuticals. 6 therapies are approved with 20 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

57/100

Pipeline Velocity

15/100

Mech. Concentration

17/100

Completion Rate

91/100

Attrition Rate

7/100

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.

A

ADARx Pharmaceuticals, Inc.

1 program

Phase 2S

Shanghai Argo Biopharmaceutical Co., Ltd.

1 program

Phase 2Ionis Pharmaceuticals logo

Ionis Pharmaceuticals

1 program

Phase 3K

KalVista Pharmaceuticals, Ltd.

1 program

Phase 3A

Amring

1 program

Phase 3Octapharma logo

Octapharma

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

85%

Small molecule

29 of 34 classified programs — the dominant modality here.

15%ALL OTHERSMonoclonal antibody26%Protein / enzyme13%Peptide13%RNA therapy13%

Small molecule leads; the ring shows all 34 classified programs, each share of the total. Modality classified for 100% of 44 pipeline programs.

Analysis

Trial Status

76

clinical trials

Completed53 · 70%
Active, not recruiting8 · 11%
Withdrawn4 · 5%
Recruiting3 · 4%
Available2 · 3%
Enrolling by invitation2 · 3%
Other4 · 5%

Clinical Trials

Trial Activity

76 trials

Donidalorsen Treatment in Children With Hereditary Angioedema
Recruiting·Ionis Pharmaceuticals logoIonis Pharmaceuticals·Phase 3·NCT07298447·20 enrolled
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
Recruiting·CSL Behring logoCSL Behring·Phase 4·NCT06806657·30 enrolled
Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients
Recruiting·IInstitute for Asthma and Allergy·Phase 1/2·NCT07046806·10 enrolled
A Phase 2 in Adult Subjects With Hereditary Angioedema
Active, not recruiting·SShanghai Argo Biopharmaceutical Co., Ltd.·Phase 2·NCT06846398·25 enrolled
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Active, not recruiting·Intellia Therapeutics logoIntellia Therapeutics·Phase 3·NCT06634420·60 enrolled
A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema
Active, not recruiting·AAstria Therapeutics, Inc.·Phase 2·NCT06007677·56 enrolled
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Active, not recruiting·Ionis Pharmaceuticals logoIonis Pharmaceuticals·Phase 3·NCT05392114·154 enrolled
Berotralstat Treatment in Children With Hereditary Angioedema
Active, not recruiting·BBioCryst Pharmaceuticals·Phase 3·NCT05453968·29 enrolled
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
Active, not recruiting·AADARx Pharmaceuticals, Inc.·Phase 1/2·NCT05691361·53 enrolled
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Active, not recruiting·Intellia Therapeutics logoIntellia Therapeutics·Phase 1/2·NCT05120830·37 enrolled
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
Active, not recruiting·KKalVista Pharmaceuticals, Ltd.·Phase 3·NCT05505916·145 enrolled
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
Not yet recruiting·AADARx Pharmaceuticals, Inc.·Phase 3·NCT07428499·90 enrolled
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Enrolling by invitation·—·Phase 2/3·NCT05396105·150 enrolled
A Long-Term Study of Navenibart in Participants With Hereditary Angioedema
Enrolling by invitation·AAstria Therapeutics, Inc.·Phase 3·NCT07204938·145 enrolled
Study of Heparin Prophylaxis of Hereditary Angioedema Exacerbations
Completed·UUniversity of Iowa·N/A·NCT00004694·24 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)