risdiplam   Click here for help

GtoPdb Ligand ID: 11170

Synonyms: compound 1 [PMID: 30044619] | Evrysdi® | RG-7916 | RG7916 | RO-7034067 | RO7034067
Approved drug
risdiplam is an approved drug (FDA (2020), EMA (2021))
Compound class: Synthetic organic
Comment: Risdiplam is a first-in-class, orally bioavailable splicing modifier [3]. Like nusinersen, it is intended to improve the efficiency of SMN2 (HGNC ID 11118; Uniprot Q16637) gene transcription in patients with spinal muscular atrophy (SMA) [2].
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 5
Hydrogen bond donors 1
Rotatable bonds 2
Topological polar surface area 79.83
Molecular weight 401.2
XLogP 3.52
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 Cc1cn2c(n1)c(C)cc(n2)c1cc(=O)n2c(n1)ccc(c2)N1CCNC2(C1)CC2
Isomeric SMILES Cc1cn2c(n1)c(C)cc(n2)c1cc(=O)n2c(n1)ccc(c2)N1CCNC2(C1)CC2
InChI InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
InChI Key ASKZRYGFUPSJPN-UHFFFAOYSA-N
References
1. Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C et al.. (2019)
The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.
Neuromuscul Disord, 29 (1): 21-29. [PMID:30553700]
2. Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H et al.. (2018)
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Pharmacol Res Perspect, 6 (6): e00447. [PMID:30519476]
3. Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M et al.. (2018)
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
J Med Chem, 61 (15): 6501-6517. [PMID:30044619]