VivoSeq
Other

Erythropoietic Protoporphyria

9

Pipeline Programs↗

3

Active Trials↗

9

Companies Involved↗

Erythropoietic Protoporphyria is an early-stage landscape with 9 programs across 9 companies. The most active players are Roche, Portal Therapeutics, Inc., DT MedTech, LLC. 2 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

37/100

Pipeline Velocity

6/100

Mech. Concentration

50/100

Completion Rate

86/100

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.

Roche logo

Roche

1 program

Phase 1P

Portal Therapeutics, Inc.

1 program

Phase 2D

DT MedTech, LLC

1 program

Phase 2Eli Lilly logo

Eli Lilly

1 program

Phase 2C

Clinuvel Pharmaceuticals Limited

1 program

Phase 3T

Tanabe Pharma America, Inc.

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

89%

Small molecule

8 of 9 classified programs — the dominant modality here.

11%ALL OTHERSPeptide111%

Small molecule leads; the ring shows all 9 classified programs, each share of the total. Modality classified for 90% of 10 pipeline programs.

Analysis

Trial Status

17

clinical trials

Completed12 · 71%
Enrolling by invitation2 · 12%
Withdrawn1 · 6%
Recruiting1 · 6%
Terminated1 · 6%

Clinical Trials

Trial Activity

17 trials

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Recruiting·TTanabe Pharma America, Inc.·Phase 3·NCT05005975·301 enrolled
GATEWAY: A Phase 2a Study of PORT-77 in Adults With Erythropoietic Protoporphyria
Enrolling by invitation·PPortal Therapeutics, Inc.·Phase 2·NCT06971900·14 enrolled
HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
Enrolling by invitation·DDisc Medicine, Inc·Preclinical·NCT05883748·230 enrolled
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
Completed·TTanabe Pharma America, Inc.·Phase 2·NCT03520036·102 enrolled
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Completed·CClinuvel Pharmaceuticals Limited·Phase 3·NCT00979745·74 enrolled
Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Completed·CClinuvel Pharmaceuticals Limited·Phase 2·NCT01097044·77 enrolled
Phase III Confirmatory Study in Erythropoietic Protoporphyria
Completed·CClinuvel Pharmaceuticals Limited·Phase 3·NCT01605136·93 enrolled
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Completed·CClinuvel Pharmaceuticals Limited·Phase 3·NCT04578496·16 enrolled
Oral Iron for Erythropoietic Protoporphyrias
Completed·IIcahn School of Medicine at Mount Sinai·N/A·NCT02979249·16 enrolled
Multicentre Phase III Erythropoietic Protoporphyria Study
Completed·CClinuvel Pharmaceuticals Limited·Phase 3·NCT04053270·100 enrolled
Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria
Completed·—·Phase 3·NCT00004940·50 enrolled
Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria
Completed·FFDA Office of Orphan Products Development·N/A·NCT00004831·20 enrolled
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
Completed·TTanabe Pharma America, Inc.·Phase 3·NCT04402489·184 enrolled
Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients
Completed·CClinuvel Europe Limited·Phase 1/2·NCT06388642·28 enrolled
Effect of Oral Cimetidine in the Protoporphyrias
Completed·AAmy K. Dickey, M.D.·Phase 2·NCT05020184·26 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)