Cemdisiran

Summary

Cemdisiran (CHEMBL4297722) is a phase-3 clinical-stage oligonucleotide; indicated across 5 conditions including myasthenia gravis and hemoglobinuria.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 5 conditions
• Clinical trials: 15

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297722
Name	Cemdisiran
Type	Oligonucleotide
Max phase	3

Also known as: AD-62643, AD-62643 FREE ACID, ALN-62643, ALN-62643 FREE ACID, Cemdisiran, Cemdisiran sodium, CEMDISIRAN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
myasthenia gravis	3	MONDO:0009688	EFO:0004991
hemoglobinuria	3	MONDO:0003656	MONDO:0100244
IgA glomerulonephritis	2	MONDO:0005342	EFO:0004194
atypical hemolytic-uremic syndrome	2	MONDO:0016244	MONDO:0016244

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 15.

Phase distribution

Phase	Trials
PHASE3	6
PHASE2	5
PHASE1	2
EARLY_PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05070858	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis
NCT05133531	PHASE3	ACTIVE_NOT_RECRUITING	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
NCT05744921	PHASE3	RECRUITING	A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
NCT06541704	PHASE3	RECRUITING	A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
NCT07154745	PHASE3	RECRUITING	A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
NCT05131204	PHASE3	TERMINATED	Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT03303313	PHASE2	WITHDRAWN	A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
NCT03841448	PHASE2	TERMINATED	A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)
NCT03999840	PHASE2	WITHDRAWN	Eculizumab to Cemdisiran Switch in aHUS
NCT04811716	PHASE2	COMPLETED	Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
NCT04888507	PHASE2	COMPLETED	Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
NCT04601844	PHASE1	COMPLETED	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers
NCT04940364	PHASE1	COMPLETED	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
NCT06479863	EARLY_PHASE1	RECRUITING	Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
NCT06028594	Not specified	NO_LONGER_AVAILABLE	Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).