Investigational Drug
AMXT 1501 Dicaprate
TrialFetch AI Analysis
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Overview
AMXT 1501 dicaprate is an investigational oral cancer drug studied primarily with difluoromethylornithine (DFMO, or eflornithine). Human results have been published from a phase I study in advanced solid tumors; they are preliminary and do not establish that the combination improves survival or outcomes compared with standard treatment. (doi.org)
Mechanism of action
AMXT 1501 inhibits cellular uptake of polyamines, molecules that support cell growth. DFMO inhibits ornithine decarboxylase, an enzyme involved in making polyamines. Combining the drugs is intended to limit both the production and import of polyamines by cancer cells. The precise polyamine transport target of AMXT 1501 has not been established in the clinical report. (doi.org)
Efficacy
In the phase I study, 56 heavily pretreated patients received AMXT 1501 alone briefly and/or with oral DFMO. The investigators reported two confirmed responses and 16 cases of stable disease, corresponding to a 6% overall response rate and 49% clinical benefit rate among patients assessed for response. Because this was an early-phase study without a comparison group, the results cannot establish how much benefit the combination provides. (doi.org)
Safety
The most common treatment-emergent adverse events were diarrhea (39.3%), nausea (37.5%), and vomiting (33.9%). The study reported no grade 4 or 5 treatment-emergent adverse events and no deaths attributed to them. (doi.org)
Further reading
- Phase I clinical study, ESMO Open (2025)
- Preclinical mechanism study, International Journal of Cancer (2013)
Last updated: Sep 2026
Active trials using AMXT 1501 Dicaprate
Found 4 active trials using this drug:
TrialFetch AI summary: Adults with advanced or metastatic solid tumors harboring homozygous MTAP deletion receive BMS-986504, an investigational MTA-cooperative PRMT5 inhibitor, alone or in tumor-specific combinations. Combination cohorts pair it with pumitamig (a PD-L1/VEGF-A bispecific antibody) and chemotherapy; daraxonrasib (a RAS(ON) inhibitor), with or without chemotherapy; nivolumab plus relatlimab; or temozolomide and radiotherapy.
ClinicalTrials.gov ID: NCT07492680
TrialFetch AI summary: Adults with ECOG 0–1 and advanced ER-positive/HER2-negative breast cancer harboring a PIK3CA, AKT1, or PTEN alteration after endocrine therapy, or unresectable/metastatic cutaneous melanoma after checkpoint inhibition, receive oral AMXT 1501 (a polyamine transport inhibitor) plus oral DFMO (an inhibitor of polyamine synthesis). The breast cancer cohort also receives fulvestrant and capivasertib; the melanoma cohort receives pembrolizumab.
ClinicalTrials.gov ID: NCT07287917
TrialFetch AI summary: Adults with non-uveal melanoma and at least one measurable, previously untreated brain metastasis are enrolled in separate asymptomatic and symptomatic cohorts; the symptomatic cohort permits stable or tapering dexamethasone up to 4 mg/day. All patients receive ipilimumab (CTLA-4 blockade) plus nivolumab (PD-1 blockade) and relatlimab (LAG-3 blockade).
ClinicalTrials.gov ID: NCT06712927
TrialFetch AI summary: This trial enrolls patients diagnosed by age 21 with relapsed or refractory high-risk neuroblastoma, ETMR, ATRT, Ewing sarcoma, or osteosarcoma, or newly diagnosed DIPG after radiation. Patients receive oral eflornithine (DFMO), an ornithine decarboxylase inhibitor that blocks polyamine synthesis, with or without AMXT 1501, an investigational polyamine-uptake inhibitor; the neuroblastoma cohort is randomized between the two regimens.
ClinicalTrials.gov ID: NCT06465199
