Cemdisiran

Phase 3RNA therapy

Cemdisiran is one of 27 small molecule programs targeting Paroxysmal Nocturnal Hemoglobinuria. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Paroxysmal Nocturnal HemoglobinuriaRecruiting

Generalized Myasthenia GravisActive, not recruiting

Paroxysmal Nocturnal HemoglobinuriaPhase 3

Recruiting

Generalized Myasthenia GravisPhase 3

Active, not recruiting

Competition

Competitive Landscape

17 competitors in Paroxysmal Nocturnal Hemoglobinuria

#DrugCompanyPhaseTrials

Soliris

AstraZeneca

Approved

25 2

Ultomiris

AstraZeneca

Approved

12 3

Fabhalta

Novartis

Phase 3

2 4

VEOPOZ

Regeneron

Phase 3

7 5

PIASKY

Roche

Phase 3

3 6

NTQ5082

NNanjing Chia-tai Tianqing Pharmaceutical

Phase 3

2 7

Cemdisiran

Regeneron

Phase 3

7 8

NM8074

NNovelMed Therapeutics

Phase 2

1 9

HRS-5965

CChengdu Suncadia Medicine Co., Ltd.

Phase 3

3 10

ADX-038

AADARx Pharmaceuticals, Inc.

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT05133531

Q4 2028

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT05744921

Phase 3Active, not recruiting

Generalized Myasthenia Gravis

NCT05070858

Q4 2031

Phase 3Recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT07154745

Q2 2033

Phase 3Recruiting

Age-Related Macular Degeneration (AMD)

NCT06541704

Clinical

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria7 trials

Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Completed· Regeneron logo

Regeneron·Phase 2·NCT04888507·6 enrolled

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Terminated· Regeneron logo

Regeneron·Phase 3·NCT05131204·3 enrolled

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Completed· Regeneron logo

Regeneron·Phase 2·NCT04811716·24 enrolled

Show 4 more trials

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 logo

Regeneron·Phase 3·NCT05133531·190 enrolled

Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

No longer available· Regeneron logo

Regeneron··NCT06028594

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 logo

Regeneron·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 logo

Regeneron·Phase 3·NCT07154745·35 enrolled

Nephropathy1 trial

A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)

Terminated· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 2·NCT03841448·31 enrolled

Adult1 trial

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers

Completed· Regeneron logo

Regeneron·Phase 1·NCT04601844·19 enrolled

Intravenous1 trial

A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers

Completed· Regeneron logo

Regeneron·Phase 1·NCT04940364·30 enrolled

Atypical Hemolytic-Uremic Syndrome1 trial

A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome

Withdrawn· Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals·Phase 2·NCT03303313

3 more indications

Age-related Macular Degeneration (AMD)1 trial

A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Recruiting· Regeneron logo

Regeneron·Phase 3·NCT06541704·975 enrolled

Generalized Myasthenia Gravis1 trial

Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients With Symptomatic Generalized Myasthenia Gravis

Active, not recruiting· Regeneron logo

Regeneron·Phase 3·NCT05070858·288 enrolled

Unspecified1 trial

Eculizumab to Cemdisiran Switch in aHUS

Withdrawn· Mario Negri Institute for Pharmacological Research logo

Mario Negri Institute for Pharmacological Research·Phase 2·NCT03999840

Sources

References

[1]
ClinicalTrials.gov — 14 trial records for Cemdisiran. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Cemdisiran compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Cemdisiran. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Regeneron company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: RNA therapy[2]
Highest Phase: Phase 3[1]
Company: Regeneron

Cemdisiran

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q4 2028

Q4 2031

Q2 2033

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria7 trials

Nephropathy1 trial

Adult1 trial

Intravenous1 trial

Atypical Hemolytic-Uremic Syndrome1 trial

Age-related Macular Degeneration (AMD)1 trial

Generalized Myasthenia Gravis1 trial

Unspecified1 trial

References

Drug Info

Quick Facts

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