VivoSeq
Hematology

Paroxysmal Nocturnal Hemoglobinuria (PNH)

2

Pipeline Programs↗

3

Active Trials↗

4

Companies Involved↗

Paroxysmal Nocturnal Hemoglobinuria (PNH) is an early-stage landscape with 2 programs across 4 companies. The most active players are Apellis Pharmaceuticals, Regeneron, Eli Lilly.

Intelligence

Pipeline Metrics

Competitive Density

22/100

Pipeline Velocity

11/100

Mech. Concentration

100/100

Completion Rate

N/A

Attrition Rate

0/100

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.

Apellis Pharmaceuticals logo

Apellis Pharmaceuticals

1 program

Phase 2Regeneron logo

Regeneron

1 program

PreclinicalEli Lilly logo

Eli Lilly

1 program

PreclinicalNovartis logo

Novartis

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical1

ALN-CFB

Regeneron

Phase 1
No programs
Phase 21

Pegcetacoplan

Apellis Pharmaceuticals

Phase 3
No programs
Approved
No programs

Analysis

Modality Breakdown

2programs
Small molecule150%
Peptide150%

Modality classified for 67% of 3 pipeline programs.

Analysis

Trial Status

3

clinical trials

Recruiting3 · 100%

Clinical Trials

Trial Activity

A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy
Recruiting·Regeneron logoRegeneron·Preclinical·NCT07187401·24 enrolled
An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
Recruiting·Apellis Pharmaceuticals logoApellis Pharmaceuticals·Phase 2·NCT04901936·12 enrolled
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
Recruiting·Novartis logoNovartis·N/A·NCT07229235·50 enrolled

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