vamorolone   Click here for help

GtoPdb Ligand ID: 9247

Synonyms: Agamree® | VBP-15 | VBP15
Approved drug Immunopharmacology Ligand
vamorolone is an approved drug (FDA & EMA (2023))
Compound class: Synthetic organic
Comment: Vamorolone (VBP15) is a novel, oral non-hormonal steroid modulator, that was investigated in Duchenne muscular dystrophy. It is a multi-functional drug that exhibits anti-inflammatory, membrane stabilisation, and mineralocorticoid receptor antagonist activities. Vamorolone was proposed as a potential replacement for the corticosteroids that are currently used in DMD, as it appears to cause fewer of the common corticosteroid adverse effects [4].
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
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 4
Hydrogen bond donors 2
Rotatable bonds 2
Topological polar surface area 74.6
Molecular weight 356.2
XLogP 2.49
No. Lipinski's rules broken 0
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 OCC(=O)C1(O)C(C)CC2C1(C)CC=C1C2CCC2=CC(=O)C=CC12C
Isomeric SMILES OCC(=O)[C@@]1(O)[C@H](C)C[C@@H]2[C@]1(C)CC=C1[C@H]2CCC2=CC(=O)C=C[C@]12C
InChI InChI=1S/C22H28O4/c1-13-10-18-16-5-4-14-11-15(24)6-8-20(14,2)17(16)7-9-21(18,3)22(13,26)19(25)12-23/h6-8,11,13,16,18,23,26H,4-5,9-10,12H2,1-3H3/t13-,16-,18+,20+,21+,22+/m1/s1
InChI Key ZYTXTXAMMDTYDQ-DGEXFFLYSA-N
No information available.
Summary of Clinical Use Click here for help
VBP15 (vamorolone) was progressed to clinical trials in boys with Duchenne muscular dystrophy (DMD). Safety and efficacy results from three studies were reported in September 2020 [4]. Trial evidence supports vamorolone's benefit to motor function in young boys with DMD, and with a favourable safety profile. The EMA granted orphan designation for DMD treatment in September 2022. The FDA approved vamorolone to treat children with DMD in October 2023 [2], with full EMA approval following in December of that year.
Mechanism Of Action and Pharmacodynamic Effects Click here for help
VBP15 selectively engages the glucocorticoid receptor, exerting antiinflammatory and membrane stabilising effects [1].
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT02760264 A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Phase 2 Interventional ReveraGen BioPharma, Inc. 4
NCT03439670 A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) Phase 2 Interventional ReveraGen BioPharma, Inc. This trial is known as the VBP15-004 study, and is a Phase 2b/3 trial. Top-line data from this study is expected in the second quarter of 2021. Patients who complete this study will be eligible to enroll in an expanded access study to maintain treatment whilst a new drug application is submitted for regulatory approval of vamorolone as a treatment for DMD. 4