VivoSeq
Rare Disease

Paroxysmal Nocturnal Hemoglobinuria

37

Pipeline Programs↗

28

Active Trials↗

17

Companies Involved↗

Paroxysmal Nocturnal Hemoglobinuria is a moderately active landscape with 37 programs across 17 companies. The most active players are Shanghai Lanyi Therapeutics Co., Ltd., Roche, Haisco Pharmaceutical Group Co., Ltd.. 9 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

59/100

Pipeline Velocity

34/100

Mech. Concentration

29/100

Completion Rate

85/100

Attrition Rate

9/100

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.

S

Shanghai Lanyi Therapeutics Co., Ltd.

1 program

Phase 1Roche logo

Roche

1 program

Phase 2Haisco Pharmaceutical Group Co., Ltd. logo

Haisco Pharmaceutical Group Co., Ltd.

1 program

Phase 2L

Linno Pharmaceuticals, Inc.

1 program

Phase 2A

ADARx Pharmaceuticals, Inc.

1 program

Phase 2L

Longbio Pharma

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

80%

Small molecule

28 of 35 classified programs — the dominant modality here.

20%ALL OTHERSMonoclonal antibody411%Protein / enzyme13%Peptide13%RNA therapy13%

Small molecule leads; the ring shows all 35 classified programs, each share of the total. Modality classified for 100% of 40 pipeline programs.

Analysis

Trial Status

79

clinical trials

Completed39 · 49%
Recruiting13 · 16%
Active, not recruiting10 · 13%
Terminated7 · 9%
Not yet recruiting5 · 6%
Unknown status3 · 4%
Other2 · 3%

Clinical Trials

Trial Activity

79 trials

A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting·SShanghai Lanyi Therapeutics Co., Ltd.·Phase 1·NCT07256301·24 enrolled
Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
Recruiting·WWuhan Createrna Science and Technology Co., Ltd·Phase 3·NCT06932744·66 enrolled
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
Recruiting·Regeneron logoRegeneron·Phase 3·NCT05133531·190 enrolled
Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Recruiting·Haisco Pharmaceutical Group Co., Ltd. logoHaisco Pharmaceutical Group Co., Ltd.·Phase 2·NCT06745622·47 enrolled
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
Recruiting·WWuhan Createrna Science and Technology Co., Ltd·Phase 2/3·NCT06933914·120 enrolled
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting·SS-INFINITY Pharmaceuticals Co., Ltd·Phase 2·NCT06978699·24 enrolled
Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Recruiting·Jiangsu Hansoh Pharmaceutical Co., Ltd. logoJiangsu Hansoh Pharmaceutical Co., Ltd.·Phase 1/2·NCT07470762·50 enrolled
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
Recruiting·OOmeros Corporation·Phase 2·NCT06298955·25 enrolled
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
Recruiting·Regeneron logoRegeneron·Phase 3·NCT05744921·202 enrolled
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
Recruiting·Regeneron logoRegeneron·Phase 3·NCT07154745·35 enrolled
A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting·SSwedish Orphan Biovitrum·N/A·NCT05776472·200 enrolled
Danicopan Regulatory Post-Marketing Study in Korea
Recruiting·AstraZeneca logoAstraZeneca·N/A·NCT07457151·8 enrolled
A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)
Recruiting·Novartis logoNovartis·N/A·NCT07416162·21 enrolled
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Active, not recruiting·Roche logoRoche·Phase 3·NCT04432584·190 enrolled
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Active, not recruiting·Roche logoRoche·Phase 3·NCT04434092·210 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)