Severe Aplastic Anemia

59

Pipeline Programs↗

24

Active Trials↗

26

Companies Involved↗

Severe Aplastic Anemia is a moderately active landscape with 59 programs across 26 companies. The most active players are Pfizer, Eastern Cooperative Oncology Group, PersonGen BioTherapeutics (Suzhou) Co., Ltd.. 5 therapies are approved with 9 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

64/100

Pipeline Velocity

19/100

Mech. Concentration

24/100

Completion Rate

88/100

Attrition Rate

6/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved

Phase 3

Phase 2

Phase 1

Preclinical

Key Players

Top Companies

Pfizer

2 programs

Eastern Cooperative Oncology Group

1 program

PersonGen BioTherapeutics (Suzhou) Co., Ltd.

1 program

Regeneron

1 program

The Korean Society of Pediatric Hematology Oncology

1 program

Prism Pharmaceuticals

1 program

Pipeline

Programs by Phase

Preclinical6

Methotrexate

Alnylam Pharmaceuticals

Levamisole Hydrochloride

SWOG Cancer Research Network

Androgens

Shengyun Lin

Horse Anti-Thymocyte Globulin (ATG)

Michael Pulsipher

Luspatercept Combine With Deferasirox

The First Affiliated Hospital of Zhejiang Chinese Medical University

Phase 16

Methylprednisolone

Pfizer

Filgrastim

Eastern Cooperative Oncology Group

Fludarabine Phosphate

Sanofi

Pentostatin

Pfizer

RD13-02

Institute of Hematology & Blood Diseases Hospital, China

Phase 233

Rituximab

Roche

Sirolimus

Prism Pharmaceuticals

Tacrolimus

Veloxis Pharmaceuticals

G-CSF

Arog Pharmaceuticals, Inc.

Mycophenolate Mofetil

Roche

Phase 39

Hetrombopag Olamine

Jiangsu HengRui Medicine Co., Ltd.

Cyclophosphamide

Baxter Healthcare Corporation

Fludarabine

Sanofi

Eltrombopag

GSK

Romiplostim N01

Qilu Pharmaceutical Co., Ltd.

Approved5

Avatrombopag

Akarx

Cyclosporine

Novartis

Thymoglobulin

Sanofi

Rabbit ATG

Novartis

Cord Blood

Joanne Kurtzberg, MD

Analysis

Modality Breakdown

86%

Small molecule

62 of 72 classified programs — the dominant modality here.

Small molecule leads; the ring shows all 72 classified programs, each share of the total. Modality classified for 100% of 78 pipeline programs.

Analysis

Trial Status

70

clinical trials

Completed30 · 43%

Recruiting15 · 21%

Unknown status12 · 17%

Active, not recruiting5 · 7%

Not yet recruiting4 · 6%

Terminated3 · 4%

Other1 · 1%

Clinical Trials

Trial Activity

70 trials

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Recruiting·NNorthside Hospital, Inc.·Phase 2·NCT02828592·20 enrolled

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06695741·75 enrolled

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT04304820·80 enrolled

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT06412497·60 enrolled

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

Recruiting·HHematology department of the 920th hospital·Phase 2·NCT06378060·30 enrolled

Haploidentical HCT for Severe Aplastic Anemia

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

Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT07010237·65 enrolled

CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia

Recruiting·ZZhejiang University·Phase 1·NCT06633328·30 enrolled

CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 1·NCT06622694·15 enrolled

Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06613880·48 enrolled

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

Recruiting·—·Phase 1·NCT05757310·6 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

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

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

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

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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)