VivoSeq
Metabolic & Endocrine

Primary Hyperoxaluria Type 1

8

Pipeline Programs↗

3

Active Trials↗

5

Companies Involved↗

Primary Hyperoxaluria Type 1 is an early-stage landscape with 8 programs across 5 companies. The most active players are Alnylam Pharmaceuticals, Arbor Biotechnologies, Advancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

35/100

Pipeline Velocity

0/100

Mech. Concentration

N/A

Completion Rate

86/100

Attrition Rate

8/100

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.

Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Phase 2A

Arbor Biotechnologies

1 program

Phase 2A

Advancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections

1 program

Phase 2D

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

2 programs

Phase 2Novo Nordisk logo

Novo Nordisk

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

75%

Small molecule

6 of 8 classified programs — the dominant modality here.

25%ALL OTHERSRNA therapy225%

Small molecule leads; the ring shows all 8 classified programs, each share of the total. Modality classified for 100% of 10 pipeline programs.

Analysis

Trial Status

12

clinical trials

Completed6 · 50%
Recruiting2 · 17%
Approved for marketing1 · 8%
Not yet recruiting1 · 8%
No longer available1 · 8%
Terminated1 · 8%

Clinical Trials

Trial Activity

12 trials

Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
Recruiting·HHospices Civils de Lyon·N/A·NCT06225882·100 enrolled
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
Recruiting·AArbor Biotechnologies·Phase 1/2·NCT06839235·23 enrolled
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
Not yet recruiting·BBiocodex·Phase 3·NCT06465472·42 enrolled
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Completed·Novo Nordisk logoNovo Nordisk·Phase 2·NCT03847909·35 enrolled
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 2·NCT03350451·20 enrolled
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT04152200·21 enrolled
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT03681184·39 enrolled
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT03905694·18 enrolled
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 1/2·NCT02706886·52 enrolled
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
Terminated·DDicerna Pharmaceuticals, Inc., a Novo Nordisk company·Phase 1·NCT02795325·41 enrolled
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
Approved for marketing·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·N/A·NCT04125472
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
No longer available·DDicerna Pharmaceuticals, Inc., a Novo Nordisk company·N/A·NCT05993416

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