viltolarsen   Click here for help

GtoPdb Ligand ID: 11430

Synonyms: NCNP-01 | NS-065 | Viltepso®
Approved drug
viltolarsen is an approved drug (FDA (2020))
Compound class: Synthetic organic
Comment: Viltolarsen is an antisense phosphorodiamidate morpholino oligomer (PMO) class nucleotide drug. Its abbreviated chemical/nucleotide structure is all-P-ambo-[2',3'-azanediyl-P,2',3'-trideoxy-P-(dimethylamino)-2',3'-seco](2'-N→5')(CCTCCGGTTCTGAAGGTGTTC). Viltolarsen restores the reading frame of certain dystrophin gene mutations and promotes synthesis of a shorter but functional dystrophin protein.
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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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.

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Hydrogen bond acceptors 141
Hydrogen bond donors 29
Rotatable bonds 122
Topological polar surface area 2504.15
Molecular weight 6921.36
XLogP -21.82
No. Lipinski's rules broken 3
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

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Canonical SMILES OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CNC[C@@H](O1)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1ncnc2N)n1cnc2c1ncnc2N)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N
Isomeric SMILES OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CN(C[C@@H](O1)n1ccc(nc1=O)N)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@H]1O[C@H](CN(C1)P(=O)(N(C)C)OC[C@@H]1CNC[C@@H](O1)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1ncnc2N)n1cnc2c1ncnc2N)n1cnc2c1nc(N)[nH]c2=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cc(C)c(=O)[nH]c1=O)n1cnc2c1nc(N)[nH]c2=O)n1cnc2c1nc(N)[nH]c2=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N)n1cc(C)c(=O)[nH]c1=O)n1ccc(nc1=O)N)n1ccc(nc1=O)N
InChI Key ZVXLKCOOOKREJD-OERAVCCFSA-N
No information available.
Summary of Clinical Use Click here for help
Used in the treatment of Duchenne muscular dystrophy (DMD), in patients with mutations amenable to exon 53 skipping [1]. In the EU, viltolarsen holds EMA orphan designation (granted in June 2020) for this indication.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04060199 Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) Phase 3 Interventional NS Pharma, Inc.
NCT03167255 Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) Phase 2 Interventional NS Pharma, Inc.
External links Click here for help
For extended ADME data see the following:

Drugs.com