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Portola Pharmaceuticals Completes Enrollment in Pivotal Phase 3 APEX Study of Betrixaban for Prevention of Blood Clots in Acute Medically Ill Patients
--Company Expects to Report Top-Line Results at End of First Quarter 2016--
--Betrixaban Has Potential to Become First Oral Factor Xa Inhibitor Anticoagulant
Approved for Prevention of VTE in Acute Medically Ill--
Portola plans to report top-line data at the end of the first quarter of 2016. If the trial is successful, the Company expects to submit a New Drug Application (NDA) to the
“Enrolling patients in the APEX Study was a significant accomplishment for Portola, the academic leadership of the study, and the more than 450 trial sites worldwide that enrolled patients. We would like to thank all of the patients who participated in this groundbreaking study,” said
An estimated 20 million acute medically ill patients in the G7 countries are at risk of developing VTE -- which includes both deep vein thrombosis (DVT), a blood clot in the leg, and pulmonary embolism (PE), a clot that breaks loose and travels to the lungs -- either while in the hospital or following discharge. More than 1 million VTEs and 150,000 VTE-related deaths occur in acute medically ill patients each year in the G7 countries, despite the use of injectable enoxaparin in the hospital. Although more than half of VTE events occur after the patient is discharged from the hospital, no anticoagulant, including any of the marketed oral Factor Xa inhibitors, is approved for use in both the hospital setting and for the extended post-discharge period.
About the APEX Study
The Phase 3 APEX Study is evaluating the superiority of extended-duration anticoagulation with oral betrixaban (for 35-42 days) compared with standard of care anticoagulation with injectable enoxaparin (for up to 14 days) for the prevention of VTE in acute medically ill patients. APEX enrolled 7,500 patients at 450 clinical sites worldwide. The study used a biomarker-based design to identify and enroll patients at highest risk of VTE and most likely to benefit from betrixaban – specifically, those with an elevated level of D-dimer (a protein fragment present after a blood clot has developed) and those over age 75.
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 fatal 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.
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 timing of reporting of topline APEX results, the potential for betrixaban to become the first oral anticoagulant approved for both in-hospital and post-discharge prevention of VTE, including properties of betrixaban that may allow it to demonstrate a clinical benefit without the significant imbalance in the risk of fatal bleeding seen with other agents in the class, potential for a biomarker-based design to identify and enroll patients at highest risk of VTE and most likely to benefit from betrixaban, and the potential for novel biomarker and genetic approaches to increase the likelihood of clinical, regulatory and commercial success of Portola’s product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: the accuracy of Portola's estimates regarding its ability to initiate and/or complete its clinical trials; the success of Portola's clinical trials and the demonstrated efficacy of Portola's product candidates to the satisfaction of regulatory authorities; the accuracy of Portola's estimates regarding its expenses and capital requirements; Portola's ability to manufacture andexanet alfa; regulatory developments in
Ana Kapor Portola Pharmaceuticalsir@portola.com Media Contact: Julie Normart W2O Groupjnormart@w2ogroup.com