VivoSeq
Metabolic & Endocrine

Primary Hyperoxaluria

8

Pipeline Programs↗

5

Active Trials↗

4

Companies Involved↗

Primary Hyperoxaluria is an early-stage landscape with 8 programs across 4 companies. The most active players are Dicerna Pharmaceuticals, Inc., a Novo Nordisk company, Allena Pharmaceuticals, RECORDATI GROUP. 2 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

37/100

Pipeline Velocity

0/100

Mech. Concentration

50/100

Completion Rate

83/100

Attrition Rate

12/100

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.

D

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

1 program

Phase 1A

Allena Pharmaceuticals

1 program

Phase 2R

RECORDATI GROUP

1 program

Phase 2Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

90%

Small molecule

9 of 10 classified programs — the dominant modality here.

10%ALL OTHERSRNA therapy110%

Small molecule leads; the ring shows all 10 classified programs, each share of the total. Modality classified for 100% of 12 pipeline programs.

Analysis

Trial Status

17

clinical trials

Completed10 · 59%
Recruiting3 · 18%
Active, not recruiting2 · 12%
Terminated1 · 6%
Withdrawn1 · 6%

Clinical Trials

Trial Activity

17 trials

Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
Recruiting·RRenJi Hospital·Phase 1·NCT06511349·21 enrolled
Rare Kidney Stone Consortium Registry for Hereditary Kidney Stone Diseases
Recruiting·MMayo Clinic·N/A·NCT00588562·730 enrolled
ILLUMINATE-C: Givosiran in Pediatric AHP
Recruiting·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT05700005·60 enrolled
Long Term Extension Study in Patients With Primary Hyperoxaluria
Active, not recruiting·DDicerna Pharmaceuticals, Inc., a Novo Nordisk company·Phase 3·NCT04042402·75 enrolled
Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
Active, not recruiting·GGuangzhou Women and Children's Medical Center·Phase 1·NCT06892301·2 enrolled
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria
Completed·OOxThera·Phase 1/2·NCT02012985·28 enrolled
Study to Evaluate the Efficacy and Safety of OxabactTM on Reduction of Urinary Oxalate in Primary Hyperoxaluria Patients
Completed·OOxThera·Phase 2/3·NCT00638703·43 enrolled
Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
Completed·MMayo Clinic·Phase 2·NCT00283387·15 enrolled
A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Completed·OOxThera·Phase 3·NCT03116685·25 enrolled
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Primary Hyperoxaluria Patients Who Are on Dialysis
Completed·OOxThera·Phase 2·NCT02000219·12 enrolled
Study of ALLN-177 in Patients Aged 12 Years or Older With Enteric or Primary Hyperoxaluria and Hyperoxalemia
Completed·AAllena Pharmaceuticals·Phase 2·NCT03391804·15 enrolled
Trial on Treatment of Patients With Primary Hyperoxaluria Type I With Pyridoxal-phosphate
Completed·UUniversity of Cologne·Phase 2·NCT01281878·12 enrolled
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
Completed·DDicerna Pharmaceuticals, Inc., a Novo Nordisk company·Phase 1·NCT03392896·43 enrolled
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
Completed·BBiocodex·Phase 2·NCT03819647·15 enrolled
ENVISION: Givosiran in Acute Hepatic Porphyria
Completed·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 3·NCT04611828·94 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)