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Hematology/Aplastic Anemia

Aplastic Anemia

43

Pipeline Programs↗

45

Active Trials↗

23

Companies Involved↗

Aplastic Anemia is a moderately active landscape with 43 programs across 23 companies. The most active players are Keymed Biosciences Co.Ltd. 8 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

63/100

Pipeline Velocity

37/100

Mech. Concentration

16/100

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

Keymed Biosciences Co.Ltd

1 program

Pipeline

Programs by Phase

Preclinical14

Dexamethasone Sodium Phosphate

Quince Therapeutics S.p.A.

Methotrexate

Jazz Pharmaceuticals

Therapeutic Allogeneic Lymphocytes

Eastern Cooperative Oncology Group

PEG-Rhg-CSF

CSPC Baike (Shandong) Biopharmaceutical Co., Ltd.

Dapsone

Almirall, S.A.

Phase 12

Pentostatin

Pfizer

RD13-02

Institute of Hematology & Blood Diseases Hospital, China

Phase 219

Rituximab

Roche

Mycophenolate Mofetil

Roche

Methylprednisolone

Pfizer

Filgrastim

Eastern Cooperative Oncology Group

Pegfilgrastim

Amgen

Phase 38

Fludarabine

Sanofi

N-Acetylcysteine

Novartis

Melphalan

Apotex Inc.

Eltrombopag

GSK

Anti-Thymocyte Globulin

Novartis

Approved

No programs

Analysis

Modality Breakdown

71%

Small molecule

29 of 41 classified programs — the dominant modality here.

Small molecule leads; the ring shows all 41 classified programs, each share of the total. Modality classified for 100% of 43 pipeline programs.

Analysis

Trial Status

132

clinical trials

Completed55 · 42%

Recruiting26 · 20%

Unknown status22 · 17%

Not yet recruiting10 · 8%

Active, not recruiting8 · 6%

Terminated7 · 5%

Other4 · 3%

Clinical Trials

Trial Activity

132 trials

Construction of Abnormal Projection Regions of Cognitive Neural Networks in Patients With Anemia Using Functional Near-Infrared Spectroscopy (fNIRS)

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·N/A·NCT07578675·323 enrolled

A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia

Recruiting·PPeking University People's Hospital·Preclinical·NCT07629947·180 enrolled

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-Cells

Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT01174108·120 enrolled

Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia

Recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT04558736·21 enrolled

The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·N/A·NCT05012111·1,000 enrolled

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome

Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Preclinical·NCT03173937·37 enrolled

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT03520647·56 enrolled

A Phase I/II Study on the Safety and Efficacy of CD38 Monoclonal Antibody in the Treatment of Refractory Severe Aplastic Anemia

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Preclinical·NCT07714512·32 enrolled

A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial

Recruiting·PPeking University People's Hospital·N/A·NCT07586735·64 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

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

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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)