mediumClinical trial

Oral NLRP3 inhibitor dapansutrile enters clinical trials for CAPS

Cryopyrin-Associated Periodic Syndromes →

Summary

Dapansutrile (OLT1177), an oral NLRP3 inhibitor with favorable safety profile, is in Phase 2a clinical trial for CAPS maintenance therapy (NCT04524858), potentially replacing injectable biologics.

Related treatments

DapansutrileOral NLRP3 inflammasome inhibitor
Experimental linePhase 2a (no CAPS efficacy data yet)

Related genes

NLRP3Multiple pathogenic variants (>250)
Gain-of-function mutations causing constitutive inflammasome activation. Most in exon 3 (NACHT domain).
NLRP3R260W
Most frequent MWS-associated variant in French population.
NLRP3T348M
Associated with early onset, chronic course, hearing loss.
NLRP3Somatic mosaicism variants
Low-level allele frequency (1.9–45%); can increase over time. Explains 'mutation-negative' CAPS.
NLRP3De novo mutations
Spontaneous new mutations; no family history.
NLRP3Y861 LRR domain variants
Atypical phenotype with minimal cold-triggered rash.

More from Cryopyrin-Associated Periodic Syndromes

mediumGenetic discovery

Deep sequencing reveals somatic mosaicism in 40% of mutation-negative CAPS children

highMechanism

Constitutive inflammasome activation in CAPS mutants characterized

mediumDiagnostic advance

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ID: cryopyrin-associated-periodic-syndromes-update-3Type: clinical_trialImpact: medium