Aminex Therapeutics has recently announced the commencement of a multicenter Phase 1b/2 clinical trial aimed at evaluating the investigational compound AMXT 1501 when used in conjunction with difluoromethylornithine (DFMO). This study will focus on patients diagnosed with metastatic melanoma or advanced breast cancer.
The clinical trial, registered under the identifier NCT07287917, will investigate the safety, tolerability, and preliminary efficacy of this oral combination therapy alongside standard treatment protocols. The inclusion of patients suffering from metastatic melanoma underscores a sustained commitment to targeting tumor-intrinsic metabolic pathways, which may enhance existing immuno-oncology strategies.
AMXT 1501 is a groundbreaking polyamine transport inhibitor designed to obstruct the cellular uptake of polyamines, which play a vital role in tumor growth, survival, and immune evasion. On the other hand, DFMO, known as a polyamine biosynthesis inhibitor, is anticipated to work synergistically with AMXT 1501 to holistically suppress polyamine metabolism.
“The initiation of this study marks a pivotal milestone for both Aminex and patients grappling with advanced cancers that have few treatment options,” stated Mark Burns, PhD, the chief scientific officer and president of Aminex, in a recent press release. “This trial is built upon our foundational preclinical and early clinical data, which indicates the potential of the AMXT 1501 and DFMO combination to inhibit polyamine metabolism—a pathway that not only promotes tumor growth but also suppresses immune responses.”
Burns further elaborated, “We are propelling this initiative forward, collaborating with prominent investigators and pediatric oncologists, and targeting eight different tumor types.” According to Aminex, both preclinical and early clinical findings suggest that the dual inhibition of polyamine synthesis and transport could curtail tumor proliferation while also alleviating the immunosuppressive effects prevalent within the tumor microenvironment.
This mechanistic approach holds particular relevance for melanoma, where metabolic adaptation is often tied to treatment resistance and disease progression. The open-label trial plans to enroll patients with metastatic melanoma, as well as pre- and postmenopausal women diagnosed with ER-positive, HER2-negative breast cancer who have shown progression after previous therapies.
The study will commence with initial safety cohorts, followed by phases aimed at dose expansion to further delineate tolerability and to identify early signs of therapeutic activity.
In addition to its adult oncology initiatives, Aminex is advancing the AMXT 1501 and DFMO combination into pediatric oncology through a randomized Phase 1/2 trial in partnership with The Beat Childhood Cancer Consortium at Penn State College of Medicine. This trial will focus on various tumor types, including neuroblastoma, diffuse intrinsic pontine glioma, and several rare sarcomas.
Importantly, the combination therapy has received Orphan Drug Designation from the FDA for its application in treating neuroblastoma. While clinical efficacy data are still forthcoming, this trial signifies a growing interest in metabolic targets as a complementary approach in managing advanced melanoma—a field where dermatologists frequently engage in multidisciplinary oncology care.
