Baxter ($BAX) charted another late-stage success in its growing hemophilia franchise, posting positive top-line results for an orphan bleeding disorder treatment.
In a Phase III trial, Baxter's investigative BAX 111 met its primary efficacy endpoint of treating bleeding episodes in patients with von Willebrand disease, an inherited bleeding disorder. The drug is a recombinant version of the von Willebrand coagulation factor, which is either insufficient or defective in patients with the eponymous disease. The trial tested 111 both as a stand-alone therapy and in tandem with Baxter's hemophilia-treating Advate, finding that the drug worked on demand in 100% of patients who experienced bleeds during the study. The company intends to reveal the full results at a medical congress later this year.
Baxter expects to file for FDA approval for the orphan drug by the end of 2014, and the company plans to kick off a study to determine whether 111 can reduce bleeding rates when used as a prophylaxis.
If 111 wins the agency's blessing, it'll join Baxter's multibillion-dollar bleeding franchise led by the treatments Advate and Feiba. The company is planning to file an FDA application for the Phase III factor VIII treatment BAX 855 this year, and Baxter is working through Phase III for a factor VIIa treatment and Phase I for a hemophilia gene therapy, pipeline drugs the company believes present a roughly $2 billion global opportunity.
Meanwhile, Baxter is preparing to split its sprawling med tech operation from its hemophilia-focused pharma business. By mid-2015, the company expects to consolidate its dialysis-dominant device segment under the old company name and spin out its drug business under an as-yet-undisclosed moniker, installing bioscience boss Ludwig Hantson as CEO of the planned $6 billion new company.
Baxter's biopharma future isn't entirely hemophilia-dependent. The company has expanded into hematology and oncology through partnerships with Onconova ($ONTX) and Cell Therapeutics ($CTIC), and Baxter has its eye on biosimilars, teaming up with Coherus BioSciences to develop a copy of Amgen ($AMGN) and Pfizer's ($PFE) blockbuster Enbrel with plans to file for FDA approval in 2016.
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