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Portola Pharmaceuticals Announces Full Results of Phase 3 APEX Study of Betrixaban Presented at International Society on Thrombosis and Haemostasis (ISTH) Meeting
--Company Plans to Submit NDA and MAA for Betrixaban in Second Half of 2016--
--Webcast with APEX Study Executive Committee Members Today at
APEX, which enrolled 7,513 patients at more than 450 clinical sites worldwide, assessed the superiority of extended-duration betrixaban for 35 days compared to standard-duration enoxaparin for 10+4 days. The trial was designed in cooperation with the
“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,” said
Betrixaban, an investigational drug, is an oral, once-daily Factor Xa inhibitor anticoagulant. It is an
“The APEX Study results show consistent evidence that VTE events can be reduced with betrixaban with no statistical difference in major bleeding between the betrixaban and enoxaparin arms. This is particularly true for the most clinically relevant symptomatic disease where we observed a 30 to 45 percent reduction in events over the duration of the study,” said lead author of the NEJM publication
Later this year, Portola plans to submit the APEX Study data as part of a New Drug Application (NDA) for betrixaban in
“We recently held a pre-NDA meeting with the
About VTE in Acute Medically Ill Patients
An estimated 20 million acute medically ill patients in the G7 countries are at risk of developing VTE either while in the hospital or following discharge. Each year, more than 1 million VTEs and 150,000 VTE-related deaths occur in acute medically ill patients in the G7 countries, despite the standard use of injectable enoxaparin and other heparins in the hospital. Although more than half of VTE events occur after the patient is discharged from the hospital, no anticoagulant, including enoxaparin or any of the marketed oral Factor Xa inhibitors, is approved for extended VTE prophylaxis in the more than 24 million medically ill patients hospitalized in the G7 countries annually.
Betrixaban directly inhibits the activity of Factor Xa, an important validated target in the blood coagulation pathway, to prevent life-threatening thrombosis. Betrixaban has distinct properties that may allow it to demonstrate clinical benefit without the significant imbalance in the risk of major bleeding seen with other agents in the class. These include a 19-25-hour half-life for once-daily dosing; a low peak-to-trough drug concentration ratio that minimizes anticoagulant variability; low renal clearance; and no significant CYP3A4 metabolism, which may reduce the risk of drug-drug interactions.
Portola will host an investor event today,
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the potential for study results to support an application for regulatory approval of betrixaban, the potential for betrixaban, subject to regulatory approval, to play a role in both in-hospital and post-discharge reduction of VTE events and deaths, our interpretation and characterization of APEX Study results and the anticipated timing of our submission of filings seeking regulatory approval. Risks that contribute to the uncertain nature of the forward-looking statements include the results of discussions with regulatory authorities regarding interpretation of full APEX Study results, that the
Ana Kapor Portola Pharmaceuticalsir@portola.com 1.650.246.7252 Media Contact: Julie Normart W2O Groupjnormart@w2ogroup.com 1.559.974.3245