VivoSeq
Hematology

Diamond-Blackfan Anemia

8

Pipeline Programs↗

1

Active Trials↗

6

Companies Involved↗

Diamond-Blackfan anemia is an early-stage landscape with 8 programs across 6 companies. The most active players are Merck & Co., Karuna Therapeutics, Inc., a Bristol Myers Squibb company, Eastern Cooperative Oncology Group. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

34/100

Pipeline Velocity

6/100

Mech. Concentration

20/100

Completion Rate

75/100

Attrition Rate

20/100

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.

Merck & Co. logo

Merck & Co.

1 program

Phase 1K

Karuna Therapeutics, Inc., a Bristol Myers Squibb company

1 program

Phase 1E

Eastern Cooperative Oncology Group

1 program

Phase 2A

Arog Pharmaceuticals, Inc.

1 program

Phase 2Roche logo

Roche

1 program

Phase 2GSK logo

GSK

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

71%

Small molecule

5 of 7 classified programs — the dominant modality here.

29%ALL OTHERSProtein / enzyme229%

Small molecule leads; the ring shows all 7 classified programs, each share of the total. Modality classified for 100% of 8 pipeline programs.

Analysis

Trial Status

5

clinical trials

Completed3 · 60%
Not yet recruiting1 · 20%
Terminated1 · 20%

Clinical Trials

Trial Activity

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Not yet recruiting·AApriligen, Inc.·Phase 1·NCT07476183·4 enrolled
Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
Terminated·—·Phase 1·NCT01034592·2 enrolled
Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
Completed·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT00011505·15 enrolled
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Completed·National Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT04269889·15 enrolled
A Phase I/II, Intra-Patient Dose-Escalation Study of the Selective GlyT1 Inhibitor, Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia.
Completed·National Heart, Lung, and Blood Institute (NHLBI)·Preclinical·NCT05828108·15 enrolled

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