Portola Pharmaceuticals Reveals Full Results of Final Stage APEX Trial of Betrixaban

Portola Pharmaceuticals Inc. PTLA revealed that the full results of its pivotal final stage APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) trial were presented at the 62nd Annual International Society on Thrombosis and Haemostasis (ISTH) Scientific and Standardization Committee (SSC) Meeting. Portola Pharmaceuticals said that full results from the APEX study were published simultaneously online in The New England Journal of Medicine. The company disclosed top line results from the APEX study in March last. The company enrolled 7,513 patients at over 450 clinical sites throughout the world, assessed the superiority of extended-duration betrixaban for 35 days compared to standard-duration enoxaparin for 10+4 days. The pharmaceutical firm said that the study was designed in cooperation with the FDA and EMA to integrate a novel patient enrichment and statistical analysis plan derived from the 2012 FDA guidance document on enrichment strategies for clinical trials. Senior Author of the NEJM publication and APEX Executive Committee Member and Steering Committee Chairman, Michael Gibson, said, "In a pre-specified subgroup of medically ill patients who were D-dimer positive, extended- duration betrixaban demonstrated a reduction in VTE events approaching statistical significance. In the pre-specified exploratory analyses of central lab D-dimer values and in progressively larger cohorts, including all study patients, the data demonstrated a consistent and significant reduction in VTE with betrixaban with no statistical difference in major bleeding between the betrixaban and enoxaparin arms." Portola said that it intends to submit the APEX Study data as part of a New Drug Application (NDA) for betrixaban in the United States. The company indicated that it would also submit its data as part of a Marketing Authorization Application (MAA) in the EU. Its CEO, Bill Lis, added, "We recently held a pre-NDA meeting with the FDA where we reconfirmed Fast Track Designation for betrixaban in this indication and agreed to upcoming meetings in preparation for an NDA submission in the second half of 2016." On Friday, the stock traded 4.22 percent down.