Baxalta's first-of-its-kind bleeding drug lines up for FDA approval

Baxalta CEO Ludwig Hantson

Baxalta ($BXLT), recently spun out from Baxter ($BAX), is charging toward its first drug approvals under a new identity, publishing positive results for a bleeding treatment awaiting word from the FDA.

In Phase III data published in Blood, the company's BAX 111 successfully managed bleeding episodes for patients with the rare von Willebrand disease, which results from the absence of a protein needed for coagulation and affects about 1% of Americans. The treatment met its goals by helping all of the trial's 37 patients stem bleeding when used on demand, Baxalta said, and one infusion was enough to control about 82% of bleeds.

Baxalta filed BAX 111 with the FDA in December and expects to win approval for the orphan treatment before the end of the year, hoping to launch the first factor-replacement therapy for what is the world's most common inherited bleeding disorder. The company submitted BAX 855, a long-acting hemophilia A treatment, around the same time, and Baxalta is looking to the pair of therapies to mark its inaugural product launches since separating from its sister company.

Beyond its two lead assets, Baxalta is moving forward with a wide pipeline of hematology and immunology treatments. Under CEO Ludwig Hantson, the company has promised to launch 20 of its in-development therapies by 2020, heaping $2.5 billion in annual sales onto the roughly $6 billion in revenue it currently pulls in.

Outside of its home base of hemophilia, Baxalta picked up a promising immunology drug in its $225 million buyout of Germany's SuppreMol earlier this year, pushing that treatment through Phase II trials in lupus and idiopathic thrombocytopenic purpura. And in oncology, Baxalta's checkbook has brought in treatments from Merrimack ($MACK), CTI BioPharma's ($CTIC) and Onconova ($ONTX), each of which is in late-stage development.

Meanwhile, the fledgling company is mapping out a future in the biotech hub of Cambridge, MA, signing a 12-year lease on a 200,000-square-foot space in Kendall Square and settling in alongside a slew of world-class research organizations and industry luminaries.

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