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Amsulostat

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Investigational pan-lysyl-oxidase inhibitor targeting fibrotic extracellular-matrix remodeling in myelofibrosis and related blood disorders.

Modality
oral pan-lysyl-oxidase inhibitor
Availability (US)
Completed Phase 2a in myelofibrosis; FDA-supported Phase 2b design planned as of April 28, 2026
Biomarkers
lysyl oxidase activity; collagen cross-linking; bone-marrow fibrosis grade; hematologic and spleen measures
Hallmark targeted
extracellular-matrix dysregulation, fibrosis, altered intercellular communication
Kind
investigational drug
Clinical trials
NCT04676529 myelofibrosis study; AZALOX Phase 1b/2 study in high-risk MDS and CMML; planned placebo-controlled Phase 2b in myelofibrosis