DrugExperimental line
Dapansutrile
Oral NLRP3 inflammasome inhibitor
- Response rate
- Phase 2a (no CAPS efficacy data yet)
- Onset
- Unknown
- Route
- Oral
- Line
- Experimental
- IgM effect
- Unknown
- Evidence level
- red
Evidence summary
First oral small-molecule NLRP3 inhibitor to reach clinical trials. Directly binds NLRP3 NACHT domain, preventing inflammasome assembly. Phase I showed favorable safety profile without hepatotoxicity seen with MCC950. Could represent paradigm shift from injectable biologics to oral therapy if proven effective in CAPS.
Molecular targets
| Molecule | Role | Expression | Evidence |
|---|---|---|---|
| NLRP3 (Cryopyrin) | Inflammasome sensor and scaffold | mutated | established |
Sources (1)
I3Marchetti C et al. (2018) OLT1177, a β-sulfonyl nitrile compound, safe in humans, inhibits the NLRP3 inflammasome and reverses the metabolic cost of inflammationPubMed ↗