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

Jan 9, 2018

On 3 rdJanuary 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. 2Based 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.

  1. PR Newswire: Cantex Pharmaceuticals, Inc. Announces FDA Orphan Drug Designation Has Been Granted to CX-01 for Treatment of Acute Myeloid Leukemia. 2018 Jan 3. [Accessed 2018 Jan 4].
  2. Kovacsovics T.J. et al. CX-01, a Low Anticoagulant Heparin, May Enhance Count Recovery and Treatment Efficacy in Acute Myeloid Leukemia. Blood. 2016; 128: 5220.
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