FDA grants Orphan Drug Designation to CX-01 for the treatment of AML

On 3^rd January 2018, the US Food and Drug Administration (FDA) granted Orphan Drug Designation to CX-01, a low anti-coagulant heparin, for the treatment of patients with Acute Myeloid Leukemia (AML).¹

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, thus 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 Based on this finding, CX-01 is currently 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.