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Portola Pharmaceuticals Announces FDA Grants Fast Track Designation to Betrixaban for Prevention of Blood Clots in Acute Medically Ill Patients
Portola is currently evaluating betrixaban in the global, pivotal, 7,500-patient Phase 3 APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) Study for the prevention of VTE in acute medically ill patients. The Company expects to complete patient enrollment in APEX by the end of 2015 and report top-line data in the first quarter of 2016. If the trial is successful, the Company expects to submit a New Drug Application (NDA) later in 2016 under the Fast Track designation.
The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables a company to have early and frequent communication with the
“FDA’s Fast Track designation of betrixaban recognizes the unmet medical need in the prevention of VTE in acute medically ill patients. Importantly, it raises public awareness of this life-threatening condition in a large, underserved patient population. Betrixaban has the potential to become the first anticoagulant approved for extended-duration prevention of VTE,” said
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. The study is being conducted at 450 clinical sites worldwide. APEX is using a biomarker-based trial design to identify and enroll patients who are 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. The APEX Study is the only biomarker-based Phase 3 study for hospital-to-home prophylaxis of VTE in acute medically ill patients. It is also the only pivotal thrombosis trial evaluating a single anticoagulant for VTE prophylaxis in both the in-hospital and post-discharge settings.
Betrixaban is an investigational oral, Factor Xa inhibitor anticoagulant. 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 the potential advantages of Fast Track designation, timing of patient enrollment in APEX and 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. 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