Branaplam

DB14918InvestigationalSmall molecule

Branaplam is under investigation in clinical trial NCT02268552 (An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)).

Chemical structure of Branaplam
Formula / weight
C22H27N5O2 · 393.491 g/mol (avg)

Resolves to

Compound
UNIIP12R69543ACAS1562338-42-4
Structure
InChIKeySTWTUEAWRAIWJG-UHFFFAOYSA-NSMILESCC1(C)CC(CC(C)(C)N1)OC1=NN=C(C=C1)C1=C(O)C=C(C=C1)C1=CNN=C1
Targets
Q16637SMN1

What you can answer from here — as of April 23, 2025

1Protein targetEach mapped to UniProt, with action and pharmacological action
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3Clinical trialsPhase, status and sponsor resolved per trial
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3+ReferencesStructured and connected to the statements they support
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