amsulostat

Amsulostat is an orally available small-molecule pan-lysyl oxidase (LOX) inhibitor in clinical development by Syntara for the treatment of myelofibrosis and other hematologic malignancies characterized by pathologic bone marrow fibrosis.[5][9][10][12] By irreversibly inhibiting all LOX family enzymes, amsulostat blocks oxidative deamination of lysine residues on collagen and elastin, preventing cross-link formation and extracellular matrix remodeling, which may reverse or reduce fibrotic tissue and improve bone marrow function.[5][7][9] Initially developed under the code names SNT-5505 and PXS-5505, it has shown promising Phase 1/2a and Phase 2 data in myelofibrosis, including durable symptom improvement and spleen volume reduction when used alone and in combination with the JAK inhibitor ruxolitinib, and has received FDA Orphan Drug and Fast Track designations as well as a positive EMA orphan drug opinion in myelofibrosis.[2][3][9][10][11][12] Amsulostat is also being evaluated in combination with hypomethylating agents in myelodysplastic syndromes and chronic myelomonocytic leukemia, and is part of a broader Syntara pan‑LOX inhibitor program targeting fibrotic diseases.[3][4][8][9]

Other names

2-buten-1-amine, 3-fluoro-4-(8-quinolinylsulfonyl)-, (2z)-UNII DO94E28WYW