Publication in Blood Advances® Reports that the Combination of Cantex Pharmaceuticals' CX-01 with Chemotherapy for the Treatment of Acute Myeloid Leukemia Showed Encouraging Complete Remission Rates and Rapid Platelet Count Recovery

WESTON, Fla., March 12, 2018 /PRNewswire/ -- Cantex Pharmaceuticals Inc. ("Cantex"), a clinical stage biopharmaceutical company developing proprietary pharmaceuticals that enhance the treatment of cancers and other life-threatening disorders, today announced that results from the pilot study of its CX-01 product candidate in acute myeloid leukemia ("AML") have been published in the peer-reviewed journal, Blood Advances^®. Findings from the study demonstrated that CX-01 in combination with chemotherapy showed encouraging complete remission rates, rapid platelet recovery and was well tolerated. Cantex's CX-01 is an investigational agent that could enhance the effectiveness of leukemia treatments. One potential mechanism of action of CX-01 is disruption of the adhesion of leukemia cells in the protective bone marrow environment.

The paper, titled "Combination of the Low Anticoagulant Heparin CX-01 with Chemotherapy for the Treatment of Acute Myeloid Leukemia," reported results from Cantex's pilot study of CX-01 in combination with chemotherapy (cytarabine and idarubicin given on a "7 + 3" schedule) for the treatment of AML. Data from the study indicated that 92% (11 of 12) of patients achieved a complete remission following a single induction cycle of CX-01 in combination with chemotherapy treatment. Additionally, hematologic recovery appeared to be enhanced, with a median of 22 days to reach an absolute neutrophil count (ANC) of 0.5 x 10⁹/L or higher, and 21 days to reach a platelet count higher than 20 x 10⁹ /L in induction cycles.

The study was conducted at Huntsman Cancer Institute (HCI) at the University of Utah, the Medical University of South Carolina, and Augusta University. HCI was the lead cancer center for this trial. The design was developed by Paul Shami, MD, and Tibor Kovacsovics, MD, at HCI and their colleagues at the other study centers.

Stephen Marcus, M.D., Chief Executive Officer of Cantex and a co-author of the paper, stated, "Results from the pilot study suggest that CX-01 in combination with chemotherapy could provide a potentially efficacious therapy for the treatment of AML. Not only did the study produce highly encouraging remission rates after a single induction cycle of chemotherapy, but the therapy also suggested a more rapid white blood cell and platelet count recovery, which is important to recovery from intensive chemotherapy."

Based upon the results of this study, 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 enhance the efficacy of front-line chemotherapy of AML. Top-line results from this study are expected later this year. In January of this year, the U.S. Food and Drug Administration granted Orphan Drug Designation to CX-01 for the treatment of AML.

For access to the full paper in Blood Advances^®, please proceed to: http://www.bloodadvances.org/content/2/4/381

An additional Phase II study in refractory myelodysplastic syndrome and refractory AML is in progress at Washington University in St. Louis.