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Portola Pharmaceuticals Reports Third Quarter 2015

The following excerpt is from the company's SEC filing.

Financial Results and Provides Corporate Update

Initiated Rolling BLA submission to FDA for Andexanet alfa

South San Francisco, Calif. (November 9, 2015) Portola Pharmaceuticals (Nasdaq: PTLA) today provided a corporate update and reported its financial results for the third quarter ended September 30, 2015.

We achieved a number of significant clinical, regulatory and manufacturing milestones over the past quarter, said William Lis, chief executive officer of Portola. We completed enrollment in the APEX study of betrixaban, our Fast Track -designated product. For andexanet alfa, our breakthrough Factor Xa reversal agent, we initiated our rolling submission of our Biologics License Application, or BLA, to the U.S. Food and Drug Administration, or FDA. That comes on the heels of two important andexanet manufacturing milestones: we completed the commercial manufacturing process validation in support of our submission and commercial launch, and successfully scaled-up our first Generation 2 process, which will allow us to increase our production capacity at lower costs. These achievements keep us on track for our planned launch of andexanet alfa in 2016 followed by betrixaban in 2017, assuming clinical and regulatory success.

Other Recent Achievements, Upcoming Events and Milestones


Completed APEX (Acute Medically Ill VTE Prevention with Extended Duration Betrixaban) study enrollment.

Successfully completed a sixth and final review of the APEX study Data and Safety Monitoring Committee, to correspond with an increase in sample size from 6,850 to 7,500 patients.

Received Fast Track designation from the FDA for prevention of VTE in acute medically ill patients.

APEX Study metrics, including pooled blinded aggregate event rates, remain on target.

Plan to report topline APEX study data late in the first quarter of 2016.

Plan to submit a New Drug Application to the FDA in 2016, subject to positive data.

Andexanet Alfa

Initiated rolling submission of BLA); we expect the application to be complete by the end of 2015 under Accelerated Approval pathway.

Completed commercial manufacturing process validation at CMC Biologics to support the BLA and commercial launch at 2,500 liter scale.

Successfully scaled up our first Generation 2, 10,000 liter batch product at Lonza.

Announced topline data from the second part of the Phase 3 ANNEXA

-R study, which demonstrated that andexanet alfa administered as an intravenous bolus followed by a continuous two-hour infusion produced rapid reversal of the anticoagulant effect of rivaroxaban and sustained it for the duration of the infusion. The full data set from Part 2 of the study will be presented during a Late-Breaking Clinical Trial session at the American Heart Associations Scientific Sessions 2015 on November 11 in Orlando.

Enrollment remains on track in ANNEXA-4, a Phase 4 confirmatory study of patients receiving apixaban, rivaroxaban, edoxaban or enoxaparin who present with an acute major bleed.

Initiated a Phase 2 proof-of-concept study in healthy volunteers to evaluate the safety and efficacy of andexanet alfa in reversing the anticoagulant effect of betrixaban, and define the dose of betrixaban required for inclusion in ANNEXA-4.

Plan to present two abstracts at the American Society of Hematology (ASH) Annual Meeting in December in Orlando.

Cerdulatinib Oral, dual Syk/JAK kinase inhibitor for hematologic cancers

Continued to dose-escalate in the Phase 1 part of the ongoing Phase 1/2a study in patients with relapsed/refractory B-cell malignancies who have failed...