VivoSeq
Hematology

Aplastic Anemia

64

Pipeline Programs↗

14

Active Trials↗

31

Companies Involved↗

Aplastic Anemia is a moderately active landscape with 64 programs across 31 companies. The most active players are Astellas, Johnson & Johnson, Arog Pharmaceuticals, Inc.. 11 therapies are approved with 11 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

64/100

Pipeline Velocity

19/100

Mech. Concentration

19/100

Completion Rate

86/100

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.

Astellas logo

Astellas

1 program

Phase 1Johnson & Johnson logo

Johnson & Johnson

1 program

Phase 1A

Arog Pharmaceuticals, Inc.

1 program

Phase 1J

Jiangsu Topcel-KH Pharmaceutical Co., Ltd.

1 program

Phase 1Amgen logo

Amgen

2 programs

Phase 2S

Shenzhen Zhongxing Yangfan Biotech Co. Ltd.

1 program

Phase 2

Pipeline

Programs by Phase

Preclinical9

Dexamethasone Sodium Phosphate

Quince Therapeutics S.p.A.

Methotrexate

Alnylam Pharmaceuticals

Therapeutic Allogeneic Lymphocytes

Eastern Cooperative Oncology Group

PEG-Rhg-CSF

CSPC Baike (Shandong) Biopharmaceutical Co., Ltd.

Dapsone

Almirall, S.A.

Phase 17

Alefacept

Astellas

Hetrombopag Olamine

Jiangsu HengRui Medicine Co., Ltd.

Daratumumab

Johnson & Johnson

G-CSF

Arog Pharmaceuticals, Inc.

Mesenchymal Stem Cells

Jiangsu Topcel-KH Pharmaceutical Co., Ltd.

Phase 226

Decitabine

Sun Pharma

Fludarabine

Sanofi

Cyclophosphamide

Baxter Healthcare Corporation

Romiplostim

Amgen

Methylprednisolone

Pfizer

Phase 311

Melphalan

Apotex Inc.

Fludarabine Phosphate

Sanofi

Thiotepa

Alnylam Pharmaceuticals

Busulfan

Akero Therapeutics

PF-06462700

Pfizer

Approved11

Acetylcysteine

Arbor Pharmaceuticals, Inc.

Avatrombopag

Akarx

Danazol

Sanofi

Dexamethasone

Aspen Global Incorporated

Prednisone

Roxane Laboratories

Analysis

Modality Breakdown

75%

Small molecule

50 of 67 classified programs — the dominant modality here.

25%ALL OTHERSCell therapy69%Monoclonal antibody57%Protein / enzyme57%Vaccine11%

Small molecule leads; the ring shows all 67 classified programs, each share of the total. Modality classified for 100% of 71 pipeline programs.

Analysis

Trial Status

54

clinical trials

Completed25 · 46%
Unknown status10 · 19%
Recruiting7 · 13%
Not yet recruiting4 · 7%
Terminated3 · 6%
Active, not recruiting3 · 6%
Other2 · 4%

Clinical Trials

Trial Activity

54 trials

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Recruiting·MMedical College of Wisconsin·Phase 2·NCT06517641·60 enrolled
Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia
Recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT06752694·20 enrolled
Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06254560·20 enrolled
Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A
Recruiting·PPeking Union Medical College Hospital·Phase 2·NCT06802055·40 enrolled
Adverse Effects of ATG/ALG Therapy in Aplastic Anemia
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·N/A·NCT07461116·200 enrolled
A Study of Emapalumab for Pediatric Aplastic Anemia
Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT06430788·35 enrolled
Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)
Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT01624805·140 enrolled
Multi-omics Profiling of Patients With Aplastic Anemia Before and After CD7-CAR-T Therapy
Active, not recruiting·XXuzhou Medical University·N/A·NCT07139600·5 enrolled
Eltrombopag for Moderate Aplastic Anemia
Active, not recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT01328587·34 enrolled
T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia
Active, not recruiting·MMemorial Sloan Kettering Cancer Center·Phase 1·NCT03531736·17 enrolled
Reduced-dose Conditioning Regimen Containing TBI in HSCT Treating Elderly Patients With Aplastic Anemia
Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06769568·20 enrolled
Efficacy and Safety of UCBT With TMI -Based Conditioning Regimen for Adults With Refractory/Relapsed Aplastic Anemia
Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT07078721·11 enrolled
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 1/2·NCT07287228·37 enrolled
Study of CM313(SC) Injection in Subjects With Relapsed/Refractory Aplastic Anemia
Not yet recruiting·KKeymed Biosciences Co.Ltd·Phase 1/2·NCT06791824·125 enrolled
A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Completed·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00922883·43 enrolled
Page 1 of 4

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