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AMXT1501 + eflornithine
- Development stage
- Unknown
- Lead developer
- Aminex Therapeutics
- Modality
- Small Molecules
- Administration
- Oral, Intravenous
01
Overview
**AMXT1501 + eflornithine** is a **combination therapy** targeting the polyamine metabolic pathway for cancer treatment. *AMXT1501* is a polyamine transport inhibitor that blocks the uptake of polyamines into cancer cells, reducing intracellular polyamine concentrations. *Eflornithine* (DFMO) is an ornithine decarboxylase inhibitor that blocks polyamine biosynthesis. Used together, they deplete tumor cell polyamine pools, inhibit proliferation, and induce apoptosis via G1 cell cycle arrest and activation of apoptotic pathways. This combination has shown promise in preclinical studies against neuroblastoma and diffuse intrinsic pontine glioma (DIPG), and has been investigated in early-phase clinical trials for various solid tumors, neuroblastoma, glioma, and sarcoma. The combination is being developed by Aminex Therapeutics, and uses both oral and intravenous formulations in clinical studies[1][4][5][6][7].
- Brand names
- eflornithine
- Other names
- AMXT1501 + DFMOAMXT-1501 + eflornithine
02
Targets
ODC1 (Ornithine decarboxylase)Polyamine transporter
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