One oral combination.
Adult and pediatric programs.
Clinical development across adult and pediatric cancers.
Clinical programs.
| Program | Preclinical | Phase 1 | Phase 2 | Phase 3 |
|---|---|---|---|---|
|
ER+/HER2− metastatic breast cancer
AMXT 1501 + DFMO + fulvestrant + capivasertib |
✓ | 1b | — | — |
|
Advanced / metastatic melanoma
AMXT 1501 + DFMO + pembrolizumab |
✓ | 1b | — | — |
|
Six pediatric solid tumors
AMXT 1501 + DFMO |
✓ | 1 | — | — |
Diagram is a simplified phase overview. See trial records for verified program status.
Phase 1b/2 adult program.
Multicenter, open-label Phase 1b/2 adding oral AMXT 1501 and DFMO to standard of care. Phase 1b confirms the dose; Phase 2 measures response rate and duration of response (RECIST 1.1). Approximately 20 US sites; approximately 92 patients planned.
Status: Enrolling.
View trial NCT07287917 →
Six pediatric tumor types.
Phase 1/2 sponsored and funded by Penn State College of Medicine and the Beat Childhood Cancer Consortium, a network of 50+ US clinics.
FDA Orphan Drug Designations.
Neuroblastoma
Malignant glioma
Research in advanced solid tumors.
Published in ESMO Open (2025): 56 patients with advanced solid tumors, a median of 10 prior therapies, recommended Phase 2 dose established, no treatment-related Grade 4/5 adverse events.
View publication →Explore trial eligibility.
Eligibility and participating sites are listed on ClinicalTrials.gov. Patients should speak with their oncologist about whether a trial may be right for them.