On 27th August 2018, the US Food and Drug Administration (FDA) granted Fast Track Designation to CX-01, a low anti-coagulant heparin, for the treatment of patients aged over 60 receiving induction therapy for newly diagnosed acute myeloid leukemia (AML).1 In January 2018, CX-01 was granted Orphan Drug Designation by the FDA for the treatment of AML.
Therapy for AML is associated with pancytopenia and a high failure rate due to resistant leukemia stem cells that use CXCL12/CXCR4 axis to home to marrow niches. Additionally, Platelet Factor 4 (PF4) negatively regulates bone marrow recovery after chemotherapy. CX-01 binds and inhibits PF4, thus neutralizing its activity and may also disrupt the CXCL12/CXCR4 axis, therefore improving the efficacy of AML therapy.
Preliminary data from a phase IIa non-randomized trial have demonstrated that CX-01 in combination with induction chemotherapy is well-tolerated and may enhance count recovery and treatment efficacy in patients with newly diagnosed primary AML.2 The drug manufacturers, Cantex Pharmaceuticals, noted that the Fast Track Designation granted by the FDA to CX-01 “represents recognition of CX-01's potential to address a significant unmet need in the treatment of AML by enhancing the efficacy of front-line AML chemotherapy”.
At present, CX-01 is being explored in a phase II randomized study (NCT02873338) in combination with standard chemotherapy for patients with newly diagnosed AML. CX-01 is also being evaluated in a phase I study (NCT02995655) in combination with azacitidine for patients with relapsed or refractory AML.