VivoSeq
Hematology

Bone Marrow Failure Syndrome

11

Pipeline Programs↗

2

Active Trials↗

5

Companies Involved↗

Bone Marrow Failure Syndrome is an emerging landscape with 11 programs across 5 companies. The most active players are Alnylam Pharmaceuticals, Eastern Cooperative Oncology Group, Akero Therapeutics. 1 therapy is approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

0/100

Mech. Concentration

33/100

Completion Rate

80/100

Attrition Rate

14/100

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.

Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Phase 1E

Eastern Cooperative Oncology Group

2 programs

Phase 2Akero Therapeutics logo

Akero Therapeutics

1 program

Phase 3Novartis logo

Novartis

2 programs

Phase 3Sanofi logo

Sanofi

2 programs

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

73%

Small molecule

8 of 11 classified programs — the dominant modality here.

27%ALL OTHERSProtein / enzyme218%Cell therapy19%

Small molecule leads; the ring shows all 11 classified programs, each share of the total. Modality classified for 100% of 12 pipeline programs.

Analysis

Trial Status

7

clinical trials

Completed4 · 57%
Terminated1 · 14%
Active, not recruiting1 · 14%
Recruiting1 · 14%

Clinical Trials

Trial Activity

7 trials

Phase Ib Study of Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
Recruiting·National Cancer Institute (NCI)·Phase 1·NCT06090669·75 enrolled
A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)
Active, not recruiting·CCellenkos, Inc.·Phase 1·NCT03773393·18 enrolled
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
Completed·UUniversity of California, San Francisco·Phase 1·NCT00295971·21 enrolled
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Completed·NNational Center for Research Resources (NCRR)·Phase 2·NCT00004787·20 enrolled
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
Completed·—·Phase 2·NCT00004143·2 enrolled
Stem Cell Transplant for Bone Marrow Failure Syndromes
Completed·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00176878·10 enrolled
G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome
Terminated·EEmory University·N/A·NCT00228813·8 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)