VivoSeq
Metabolic & Endocrine

Osteogenesis Imperfecta

25

Pipeline Programs↗

10

Active Trials↗

15

Companies Involved↗

Osteogenesis Imperfecta is an emerging landscape with 25 programs across 15 companies. The most active players are Roche, Jiangsu Topcel-KH Pharmaceutical Co., Ltd., Mario Negri Institute for Pharmacological Research. 8 therapies are approved with 6 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

51/100

Pipeline Velocity

22/100

Mech. Concentration

14/100

Completion Rate

81/100

Attrition Rate

13/100

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.

Roche logo

Roche

1 program

Phase 1J

Jiangsu Topcel-KH Pharmaceutical Co., Ltd.

1 program

Phase 1Mario Negri Institute for Pharmacological Research logo

Mario Negri Institute for Pharmacological Research

1 program

Phase 1Ultragenyx logo

Ultragenyx

2 programs

Phase 2EMD Serono logo

EMD Serono

1 program

Phase 3Eli Lilly logo

Eli Lilly

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

76%

Small molecule

19 of 25 classified programs — the dominant modality here.

24%ALL OTHERSMonoclonal antibody416%Peptide14%Cell therapy14%

Small molecule leads; the ring shows all 25 classified programs, each share of the total. Modality classified for 96% of 28 pipeline programs.

Analysis

Trial Status

38

clinical trials

Completed22 · 58%
Recruiting5 · 13%
Terminated4 · 11%
Active, not recruiting3 · 8%
Not yet recruiting2 · 5%
Unknown status1 · 3%
Other1 · 3%

Clinical Trials

Trial Activity

38 trials

Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta
Recruiting·Amgen logoAmgen·Phase 3·NCT05972551·106 enrolled
Registry of Osteogenesis Imperfecta
Recruiting·—·N/A·NCT04115774·5,000 enrolled
A Phase 2, Multi-center, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety and Efficacy of AGA2115 in Adults With Type I, III, or IV Osteogenesis Imperfecta (OI)
Recruiting·AAngitia Incorporated Limited·Phase 2·NCT07062588·80 enrolled
Multicenter, Safety Follow-up Study to Assess Safety of Prior Treatment With Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
Recruiting·Amgen logoAmgen·Phase 3·NCT07366086·71 enrolled
A Phase 2 Multi-center, Randomized, Open-Label, Dose Regimen-Finding Study of AGA2115 in Chinese Adults and Adolescents With Type I, III, or IV Osteogenesis Imperfecta
Recruiting·AAngitia Biopharmaceuticals Guangzhou Limited·Phase 2·NCT07557446·48 enrolled
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
Active, not recruiting·Ultragenyx logoUltragenyx·Phase 3·NCT06636071·6 enrolled
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Active, not recruiting·Ultragenyx logoUltragenyx·Phase 3·NCT05768854·69 enrolled
Setrusumab vs Placebo for Osteogenesis Imperfecta
Active, not recruiting·Ultragenyx logoUltragenyx·Phase 2/3·NCT05125809·183 enrolled
Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
Not yet recruiting·EEmory University·Phase 1/2·NCT05559801·12 enrolled
Geometric Morphometric Characterization of Oro-Dental Anomalies in Rare Bone and Cartilage Diseases From 3D Digital Data (MOSAIC)
Not yet recruiting·UUniversity Hospital, Bordeaux·Preclinical·NCT07666269·240 enrolled
Bisphosphonate Therapy for Osteogenesis Imperfecta
Completed·IIndiana University School of Medicine·Phase 4·NCT00159419·18 enrolled
Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
Completed·SSt. Jude Children's Research Hospital·N/A·NCT00187018·9 enrolled
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Completed·HHospital de Cruces·Phase 1·NCT02172885·2 enrolled
Growth Hormone Therapy in Osteogenesis Imperfecta
Completed·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·Phase 3·NCT00001305·42 enrolled
Treatment of Osteogenesis Imperfecta With Parathyroid Hormone and Zoledronic Acid
Completed·UUniversity of Edinburgh·Phase 4·NCT03735537·350 enrolled
Page 1 of 3

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