AMXT 1501/DFMO is Aminex’s lead drug. It reduces polyamines in cancer cells resulting in reduction of MDSCs in the tumor microenvironment. These reductions slow cancers’ growth and activate the immune system by removing the suppressive barrier cancer cells invoke to evade the immune system. Lowering this suppressive barrier exposes cancer cells, allowing the innate immune system to attack the tumors. It additionally empowers the adaptive immune system with a “memory” to provide long-term resistance to the cancer. Importantly, this occurs without the major side effects of radiation or chemotherapy. It is anticipated that AMXT 1501/DFMO will not present significant toxic side effects for cancer patients.
Aminex’s AMXT 1501/DFMO consists of two drugs, an FDA approved polyamine synthesis inhibitor (DFMO, difluoromethylornithine) and a novel potent polyamine uptake, or transport, inhibitor (AMXT 1501). Convincing animal data confirm the potential for AMXT 1501/DFMO to be an important cancer therapeutic against tumors driven, or caused, by Myc and/or Ras oncogenes. It is believed that AMXT 1501/DFMO should be effective against numerous difficult-to-treat, adult cancers driven by mutations in the Ras and Myc oncogenes including pancreatic, prostate, skin/melanoma, breast, non-small cell lung and colorectal cancers. Additionally, early tests have shown AMXT 1501/DFMO to be effective against neuroblastoma (NB), an especially aggressive childhood nerve cancer driven by the Myc oncogene. AMXT 1501/DFMO also has potential to be used in combination with promising immune checkpoint inhibitors, other suppressor cell modulators and other immunotherapy approaches.
Dr. Michael Palfreyman, Aminex Senior VP, Drug Development, and Drs. Paul Schechter and Philippe Bey, members of Aminex’s Scientific Advisory Board (SAB), were members of the Marion Merrell Dow team (now Sanofi) which developed and tested DFMO, in the 1980s and 1990s. DFMO was extensively tested in human cancer clinical trials. Unfortunately, trial results did not achieve clinical goals. DFMO (alone) did show short-term efficacy against cancer but it was not lasting. DFMO subsequently received FDA approval in a separate trial for African sleeping sickness and has since saved many thousands of lives – a ”miracle” drug. Several clinical trials are currently being supported by the NCI using DFMO as a cancer preventative agent with promising early results. DFMO is also showing promise in preventing recurrence in several neuroblastoma trials. Drs. Palfreyman, Schechter and Bey expect the combination of DFMO and AMXT 1501 to achieve positive results in cancer therapy as originally envisioned for DFMO alone. This is based on Aminex’s discovery and patenting of a polyamine transport inhibitor (AMXT 1501) which limits the ability of cancer cells to import polyamines from the bloodstream, present due to food intake and intestinal bacteria. It is believed AMXT 1501 eliminates DFMO’s original treatment deficiency at the time of Marion Merrell Dow’s earlier DFMO cancer trials.