casimersen   Click here for help

GtoPdb Ligand ID: 11444

Synonyms: Amondys 45® | SRP-4045 | SRP4045
Approved drug
casimersen is an approved drug (FDA (2021))
Compound class: Synthetic organic
Comment: Casimersen is an antisense phosphorodiamidate morpholino oligomer (PMO) that is approved to treat certain patients with Duchenne muscular dystrophy (DMD). It promotes functional dystrophin synthesis in patients who have dystrophin gene variants that are amenable to exon 45 skipping, that is sufficient to restore some muscle function and slow disease progression.
The INN record for casimersen provides chemical identity as all-P-ambo-[2',3'-azanediyl-P-(dimethylamino)-P,2',3'- trideoxy-2',3'-seco](2'-N→5')(C-A-A-T-G-C-C-A-T-C-C-T-G-G-A-G-T-T-C-C-T-G) 5'-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,Ndimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or full IUPAC descriptor.
Physico-chemical Properties
Click here for help

Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

✖
Hydrogen bond acceptors 16
Hydrogen bond donors 3
Rotatable bonds 16
Topological polar surface area 190.77
Molecular weight 577.57
XLogP -3.68
No. Lipinski's rules broken 3
No information available.
Summary of Clinical Use Click here for help
The FDA approved casimersen in February 2021. It is indicated as a treament for patients with Duchenne muscular dystrophy (DMD) who have dystrophin gene variants that are amenable to exon 45 skipping
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03532542 An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy Phase 3 Interventional Sarepta Therapeutics, Inc.
NCT04179409 A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications Phase 2 Interventional Nationwide Children's Hospital
NCT02500381 Study of SRP-4045 and SRP-4053 in DMD Patients Phase 3 Interventional Sarepta Therapeutics, Inc.
NCT02530905 Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients Phase 1 Interventional Sarepta Therapeutics, Inc.