CX-01 is a highly negatively charged macromolecule which interacts with positively charged amino acids on selected biologic proteins.
The Company believes that, as a consequence of the interaction of CX-01 with selected proteins, CX-01 has the following biological properties:
- Disruption of the CXCL12/CXCR4 axis: This axis may be involved in the protection of leukemia cells from the killing effects of chemotherapy and in the metastasis and resistance of several cancers. Cantex believes that CX-01 binds to CXCL12 and, by doing so, disrupts attachment of CXCL12 to stromal cells in the bone marrow or other body organs, driving malignant cells out of protective environments as well as disrupting gradients needed for movement of cells with CXCR4 on their surface to these destinations.
- Neutralization of the negative effect of platelet factor 4 on bone marrow recovery: Randomized human clinical data in patients with metastatic pancreatic cancer receiving chemotherapy with or without CX-01 indicated significantly enhanced platelet recovery in patients receiving CX-01. In addition, randomized pre-clinical data has indicated enhanced platelet recovery and survival in rodents exposed to total body irradiation and enhanced platelet recovery in rodents exposed to high dose chemotherapy. Cantex believes that this effect reflects the binding of CX-01 to platelet factor 4 and neutralization of its inhibitory effect on platelet production.
- Inhibition of the HMGB1 inflammatory pathway: HMGB1 is a pro-inflammatory cytokine released when cells are damaged, which transmits its inflammatory signals via TLR4 and the receptor for advanced glycation end-products (RAGE). CX-01 is a potential potent inhibitor of the interaction between HMGB1 and these receptors.
CX-01 is administered intravenously and has a half-life of approximately 2 hours, permitting a very controlled delivery of its biologic activity and allowing the initiation and continuation of the compound’s biologic effect only as long as needed. This pharmacokinetic profile increases the safety of this compound. In the 188 patients who have received intravenous CX-01 prior to initiation of the ongoing randomized phase IIb AML study, CX-01 has demonstrated an excellent safety profile.