Hypomethylating agents are considered standard first line therapy for MDS patients requiring drug treatment. Unfortunately, these agents are not curative and only achieve remission in approximately 20% to 30% of patients, with a median duration of response of eight to ten months. Median survival time of MDS patients who have failed hypomethylating drugs is less than six months. Outcomes after hypomethylating agents are poor and there remains an unmet need for better patient treatment.
For patients with MDS, allogeneic hematopoietic stem cell transplantation remains the only curative treatment option. However, MDS is a disease of older patients, with more than 95% of cases occurring in patients over 50 years of age and the majority diagnosed at 70 years of age or older. Because of age, comorbidities, and other factors, less than 10% of all MDS patients are able to proceed to allogeneic HSCT.
Cantex believes that the combination of azacitidine and CX-01 may increase the cytotoxic effects of this current standard therapy on MDS stem cells, by disrupting the CXCL12/CXCR4 axis. Given the affinity of CX-01 for platelet factor 4, it may also help promote count recovery after treatment. The Company plans to support an investigator-initiated Phase II clinical trial to assess the efficacy of CX-01 in combination with azacitidine for the treatment of MDS. Cantex anticipates top-line results of this clinical trial will be available in the first half of 2018.