SOUTH SAN FRANCISCO, Calif., Oct. 13, 2015 (GLOBE NEWSWIRE) -- Portola Pharmaceuticals (Nasdaq:PTLA) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to betrixaban for extended-duration prevention of venous thromboembolism (VTE), or blood clots, in acute medically ill patients (i.e., those who are hospitalized for serious medical conditions, such as heart failure, stroke, infection and pulmonary disease). Betrixaban is an investigational oral anticoagulant that directly inhibits the activity of Factor Xa, an important validated target in the blood coagulation pathway, to prevent life-threatening thrombosis.
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 throughout the drug development and review process, often leading to earlier drug approval.
“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 John T. Curnutte, M.D., Ph.D., executive vice president, research and development, for Portola. “We’re now focused on completing the APEX trial and reporting results in early 2016.”
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.
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a biopharmaceutical company developing product candidates that could significantly advance the fields of thrombosis and other hematologic diseases. The Company is advancing its three wholly-owned programs using novel biomarker and genetic approaches that may increase the likelihood of clinical, regulatory and commercial success of its potentially life-saving therapies. These programs include betrixaban, an oral, once-daily Factor Xa inhibitor being evaluated in the APEX Phase 3 study for prophylaxis of venous thromboembolism (VTE); andexanet alfa, a recombinant protein designed to reverse the anticoagulant effect in patients treated with an oral or injectable Factor Xa inhibitor; and cerdulatinib, a Syk/JAK inhibitor in development to treat hematologic cancers. Portola's partnered program is focused on developing selective Syk inhibitors for inflammatory conditions. For more information, visit www.portola.com and follow the Company on Twitter @Portola_Pharma.
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 the United States and foreign countries; Portola's ability to obtain and maintain intellectual property protection for its product candidates; and the loss of key scientific or management personnel. These and other risks and uncertainties are described more fully in Portola's most recent filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K, which was filed on March 2, 2015, and Quarterly Report on Form 10-Q, which was filed on August 5, 2015. All forward-looking statements contained in this press release speak only as of the date on which they were made. Portola undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Source:Portola Pharmaceuticals, Inc.