VivoSeq
Hematology

Hemophilia

47

Pipeline Programs↗

12

Active Trials↗

24

Companies Involved↗

Hemophilia is a moderately active landscape with 47 programs across 24 companies. The most active players are Catalyst Biosciences, GC Biopharma Corp, Vertex Pharmaceuticals. 11 therapies are approved with 7 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

60/100

Pipeline Velocity

27/100

Mech. Concentration

31/100

Completion Rate

78/100

Attrition Rate

15/100

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.

C

Catalyst Biosciences

2 programs

Phase 1G

GC Biopharma Corp

1 program

Phase 1Vertex Pharmaceuticals logo

Vertex Pharmaceuticals

1 program

Phase 1Boehringer Ingelheim logo

Boehringer Ingelheim

1 program

Phase 1Pfizer logo

Pfizer

3 programs

Phase 1AstraZeneca logo

AstraZeneca

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

90%

Small molecule

37 of 41 classified programs — the dominant modality here.

10%ALL OTHERSMonoclonal antibody37%RNA therapy12%

Small molecule leads; the ring shows all 41 classified programs, each share of the total. Modality classified for 100% of 57 pipeline programs.

Analysis

Trial Status

53

clinical trials

Completed28 · 53%
Recruiting7 · 13%
Terminated6 · 11%
Unknown status5 · 9%
Active, not recruiting4 · 8%
Withdrawn2 · 4%
Other1 · 2%

Clinical Trials

Trial Activity

53 trials

Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients
Recruiting·JJiangsu BioJeTay Biotechnology Co., Ltd.·Phase 3·NCT06922045·32 enrolled
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Recruiting·AAmerican Thrombosis and Hemostasis Network·Phase 4·NCT04647227·55 enrolled
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
Recruiting·Sanofi logoSanofi·Phase 4·NCT06145373·20 enrolled
Monitoring of Anti-TFPI in Hemophilia
Recruiting·HHospices Civils de Lyon·N/A·NCT07101926·11 enrolled
PCC Treatment for Hemophilia Patients With Inhibitor（2022PCC-A）
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 4·NCT07406139·30 enrolled
Effects of a Multimodal Telerehabilitation Intervention Versus Face-to-Face Care in Adults With Hemophilia and Chronic Pain: A Randomized Controlled Non-Inferiority Trial
Recruiting·UUniversity of Chile·Preclinical·NCT07682519·24 enrolled
An Open-label, Parallel, Phase 3, Two-arm Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B With or Without Inhibitory Antibodies to Factors VIII or IX
Recruiting·Sanofi logoSanofi·Phase 3·NCT07285460·85 enrolled
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Active, not recruiting·Pfizer logoPfizer·N/A·NCT07161687·50 enrolled
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Active, not recruiting·Sanofi logoSanofi·Phase 3·NCT05662319·91 enrolled
A Phase 2 Trial of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection with Hemophilia (OP-724-H201)
Active, not recruiting·KKiminori Kimura, MD·Phase 2·NCT06144086·6 enrolled
ATLAS-PEDS: An Open-label, Multinational Study of Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Active, not recruiting·Sanofi logoSanofi·Preclinical·NCT03974113·32 enrolled
Adult and Adolescent Hemophilia Patients Treated With Marstacimab: a Patient Experience Registry
Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·N/A·NCT06992076·100 enrolled
Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia
Completed·Pfizer logoPfizer·Phase 1·NCT04878731·6 enrolled
PF-06741086 Multiple Dose Study in Severe Hemophilia
Completed·Pfizer logoPfizer·Phase 2·NCT02974855·27 enrolled
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
Completed·Merck & Co. logoMerck & Co.·Phase 4·NCT00002386·55 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)