Cantex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CX-01 for the treatment of acute myeloid leukemia (“AML”). CX-01 is an investigational agent that has the potential to enhance the effectiveness of leukemia treatments by disrupting the adhesion of leukemia cells in the protective bone marrow environment.
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides important incentives, including seven-year marketing exclusivity upon FDA approval, tax credits for qualified clinical testing, and prescription drug user fee exemption.
Cantex is currently conducting a 75-patient randomized phase IIb study in more than 20 U.S. medical centers to determine whether CX-01 can improve the efficacy of front-line chemotherapy of acute myeloid leukemia. This study builds upon a previously completed open-label, non-randomized phase IIa clinical trial in AML where CX-01 was administered in combination with induction chemotherapy to 11 patients with newly diagnosed primary AML. Results from the phase IIa study indicated that all 11 patients achieved a complete remission following a single cycle of chemotherapy treatment.
Stephen Marcus, MD, Chief Executive Officer of Cantex Pharmaceuticals, Inc., stated, “We are pleased to have been granted FDA Orphan Drug Designation for CX-01 in AML, which follows the receipt of FDA Orphan Drug Designation for our second product candidate, Dicopp™ (previously known as CX-02), for glioblastoma. In addition to our randomized study of CX-01 in front-line treatment of acute myeloid leukemia, we are also supporting an investigator-initiated study of CX-01 in refractory myelodysplastic syndrome and refractory acute myeloid leukemia”.