Details of the Drug

General Information of Drug (ID: DMAGE91)

Drug Name

RG7800

Indication

Disease Entry	ICD 11	Status	REF
Spinal muscular atrophy	8B61	Phase 1/2	[1]
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Structure

3D MOL

2D MOL

#Ro5 Violations (Lipinski): 0

Molecular Weight (mw)

416.5

Logarithm of the Partition Coefficient (xlogp)

1.6

Rotatable Bond Count (rotbonds)

3

Hydrogen Bond Donor Count (hbonddonor)

0

Hydrogen Bond Acceptor Count (hbondacc)

5

Chemical Identifiers

Formula: C24H28N6O
IUPAC Name: 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methylpiperidin-4-yl)pyrido[1,2-a]pyrimidin-4-one
Canonical SMILES: CCC1=NC(=CN2C1=CC(=N2)C3=CC(=O)N4C=C(C=C(C4=N3)C)C5CCN(CC5)C)C
InChI: InChI=1S/C24H28N6O/c1-5-19-22-11-21(27-30(22)13-16(3)25-19)20-12-23(31)29-14-18(10-15(2)24(29)26-20)17-6-8-28(4)9-7-17/h10-14,17H,5-9H2,1-4H3
InChIKey: GYFRQCMDLBNZSF-UHFFFAOYSA-N

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Survival motor neuron protein (SMN1)	TT8QL6X	SMN_HUMAN	Modulator	[2]

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Drug Off-Target (DOT)

DOT Name	DOT ID	UniProt ID	Interaction	REF
Survival motor neuron protein (SMN2)	OT54RLO1	SMN_HUMAN	Gene/Protein Processing	[3]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800039744)
2	Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy. Assay Drug Dev Technol. 2014 August 1; 12(6): 315-341.
3	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. 2019 Jan;29(1):21-29. doi: 10.1016/j.nmd.2018.10.001. Epub 2018 Oct 30.