Votoplam

DB21645InvestigationalSmall molecule

Votoplam is a small molecule drug. The usage of the INN stem '-plam' in the name indicates that Votoplam is a SMN2 gene splicing modulator. Votoplam has a monoisotopic molecular...

Mechanism: Pharmacologically active on Huntingtin
Curator reviewed
Formula / weight: C₂₁H₂₅N₉O · 419.493 g/mol (avg)
Code names: HTT227 · PTC518

Resolves to

Compound

UNIID7EZ7B585XCAS2407849-89-0ChEMBLCHEMBL4850295ChEBI758585PubChem147894286

Structure

InChIKeyICZNVPJUHBURBG-UHFFFAOYSA-NSMILESCC1(C)CC(CC(C)(C)N1)N1N=NC2=CC(=NN=C12)C1=CC=C(C=C1O)N1N=CC=N1

Targets

P42858HTT

What you can answer from here — as of September 23, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

3Clinical trialsPhase, status and sponsor resolved per trial

1+ReferenceStructured and connected to the statements they support

Which drugs share a target with Votoplam, and which of those have an active Phase 3 trial?

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