CDMO Lonza To Manufacture Portola's Andexanet Alfa, Anticoagulant Antidote

Portola Pharmaceuticals has engaged contract manufacturing and development organization (CDMO) Lonza to supply andexanet alfa, a Food and Drug Administration (FDA) designated Breakthrough Therapy. The partnership was announced in a press release, in which Portola states that andexanet alfa is the only recombinant Factor Xa molecule that reverses the effects of Factor Xa inhibitors. Portola is developing the anticoagulant antidote for people undergoing emergency surgery or suffering from a major bleed.

Lonza and Portola Agreement

The agreement between Lonza and Portola calls for Lonza to provide large-scale manufacturing after andexanet alfa launches in the U.S. Portola estimates the launch in 2016, with Lonza scaling supply 18 to 24 months after launch. Portola has contracted with CMC Biologics to manufacture andexanet alfa for the initial launch. Lonza’s efforts are directed at meeting global demand for the product. Lonza has 40 facilities worldwide.

Andexanet Alfa

Andexanet alfa, is a potential antidote to Factor Xa inhibitors, acting as a decoy to sequester direct and indirect Factor Xa inhibitors in the blood. Once the Factor Xa inhibitors are bound, they cannot bind to and inhibit native Factor Xa, restoring normal hemostatic processes. Andexanet alfa is a flexible agent, capable of acting short-term with IV bolus administration or long-term with an extended infusion.

Portola is conducting a placebo-controlled Phase III trial (ANNEXA) to demonstrate efficacy using pharmacodynamic endpoints. A Phase III trial (ANNEXA-A) met its primary and secondary endpoints with high statistical significance. The trial tested andexanet alfa reversal of the anticoagulation activity of Bristol-Myers Squibb’s and Pfizer’s direct Factor Xa inhibitor Eliquis (apixaban). Trial results from a study testing andexanet alfa against Bayer’s and Janssen's Xarelto (rivaroxaban) are expected later in the year. In 2015, Portola expects results from a study testing andexanet alfa against Daiichi Sankyo's edoxaban. The company is using these studies to support a Biological License Application (BLA) for Accelerated Approval.

About Portola

Portola Pharmaceuticals is based in San Francisco, CA. Founded in 2003, the company is now publically traded and has over 80 employees. The company focuses on novel biomarkers and genetic approaches. Portola has a hematologic cancer candidate, cerdulatinib, that inhibits tumor proliferation pathways – spleen tyrosine kinase (Syk) and janus kinase (JAK), in particular. The company is evaluating the agent in Phase 1/2 studies in B cell leukemias or lymphomas with a focus on genetically-defined subtypes.