The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.
A fast track designation has been granted by the US Food and Drug Administration (FDA) to Cantex Pharmaceuticals, Inc.’s CX-01 for the treatment of patients over 60 years of age who are receiving induction therapy for newly-diagnosed acute myeloid leukemia (AML).
AML is the most common form of acute leukemia in adults and it is estimated that about 60% of the 19,500 projected cases for 2018 will occur in individuals over the age of 60.
“Over age 60, the response to initial ‘induction’ therapy is lower, the risk of relapse is higher, and the overall survival is generally shorter, creating a significant unmet medical need for improvement in the effectiveness of this induction therapy,” Stephen Marcus, MD, chief executive officer of Cantex, explained in a recent statement. “We believe that the award of Fast Track Designation 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.”
A polysaccharide derived from a class of compounds referred to as heparinoids, CX-01 has been designed to “block” chemokine activity that encourages the resistance of blood cancers to available treatment and contributes to the suspension of bone marrow recovery post-chemotherapy.
The product is currently being evaluated in a randomized phase 2b trial in which investigators seek to determine if adding CX-01 to standard induction and consolidation therapy for AML can boost the effectiveness of the induction/consolidation therapy compared with patients receiving standard induction/consolidation therapy alone.
The trial will enroll 75 patients over 60 years of age with newly-diagnosed AML at several major research centers throughout the United States. For the trial, participants will be randomized into 1 of 3 treatment groups: those who will receive standard induction/consolidation therapy alone, those who will receive standard induction/consolidation therapy with CX-01 at lower dose level or higher dose level.
Participants will receive up to 2 induction cycles and up to 2 consolidation cycles within the 18-month trial. During this time, investigators will conduct routine clinical laboratory tests and perform bone marrow aspirates and biopsies during the induction cycles. They will monitor participants for adverse events to determine the safety of the combination.
Morphologic complete remission revaluated by IWG criteria is the trial’s primary endpoint; this will be assessed during induction and re-induction phases of the treatment—up to 60 days after the start of each treatment cycle.
Event-free survival, leukemia-free survival, overall survival, composite complete remission rate, neutrophil recovery, platelet recovery, as well as 30-day, 60-day, and 90-day mortality rate, are listed as the trial’s secondary outcome measures.
Top-line results from the trial are expected in Q4 2018.
The drug is also being evaluated in a separate investigator-initiated phase 2 trial in patients with myelodysplastic syndrome. Top-line results for this trial were reported by representatives from Cantex at the American Society of Clinical Oncology 2018 (ASCO 2018) annual meeting.
Previous to this announcement, CX-01 received an orphan drug designation from the FDA as well for the treatment of AML.