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dimethylaminomicheliolide
- Development stage
- Phase 2
- Lead developer
- Accendatech
- Modality
- Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Administration
- Oral, Intravenous (investigational), Injection (investigational), Topical/external Application (investigational), Inhalation (investigational)
01
Overview
Dimethylaminomicheliolide (DMAMCL), also known as ACT001 or ACT-001, is a small molecule prodrug of the natural product micheliolide. It is an orally available derivative designed to improve stability and bioavailability over its parent compound. DMAMCL slowly releases micheliolide under physiological conditions and exhibits anti-tumor and anti-inflammatory activities. Its primary mechanism involves targeting pyruvate kinase M2 (PKM2), leading to inhibition of cancer cell proliferation—especially in glioblastoma multiforme (GBM) and acute myeloid leukemia models. Additional mechanisms include covalent modification of STAT3/STAT5 to suppress JAK/STAT signaling and modulation of PPAR-gamma/NF-kappaB pathways for anti-inflammatory effects. The drug has orphan designation for diffuse midline glioma and is being investigated in clinical trials for recurrent GBM and other cancers[1][5][7]. Preclinical studies also suggest potential utility in pulmonary fibrosis[4].
- Brand names
- ACT001ACT-001ACT 001
- Other names
- 11.beta.,13-dihydro-13-(dimethylamino)micheliolideazuleno(4,5-b)furan-2(3h)-one, 3-((dimethylamino)methyl)-3a,4,5,7,8,9,9a,9b-octahydro-9-hydroxy-6,9-dimethyl-, (3r,3as,9r,9as,9bs)-dimethylaminomicheliolide
02
Targets
PKM2 (Pyruvate kinase M2 isoform)RELA (Nuclear Factor Kappa B Subunit p65)PPARG (Peroxisome proliferator-activated receptor gamma)STAT3 (Signal Transducer and Activator of Transcription 3)STAT5A (STAT5)
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