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MCI-9042

Tanabe Pharma Corporation · Phase 3 active Small molecule

MCI-9042 is a selective inhibitor of transthyretin (TTR) amyloid fibril formation that prevents the aggregation and deposition of misfolded transthyretin protein.

MCI-9042 is a selective inhibitor of transthyretin (TTR) amyloid fibril formation that prevents the aggregation and deposition of misfolded transthyretin protein. Used for Transthyretin amyloidosis (ATTR amyloidosis).

At a glance

Generic nameMCI-9042
Also known asSarpogrelate(INN), ANPLAG(R)
SponsorTanabe Pharma Corporation
Drug classTransthyretin stabilizer
TargetTransthyretin (TTR)
ModalitySmall molecule
Therapeutic areaNeurology
PhasePhase 3

Mechanism of action

MCI-9042 binds to transthyretin and stabilizes its native tetrameric structure, preventing dissociation into monomers that would otherwise misfold and aggregate into amyloid fibrils. By inhibiting TTR amyloid fibril formation, the drug aims to slow or halt the progression of transthyretin amyloidosis, a progressive neurodegenerative disease caused by accumulation of amyloid deposits in peripheral nerves and other tissues.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results