Baxter's ($BAX) latest hemophilia candidate met its main goals in a late-stage study, clearing the way for an FDA application as the biopharma giant works to expand its cadre of bleeding drugs.
The treatment, BAX 855, is a factor VIII-replacement therapy, designed to provide patients with the coagulation protein they're lacking and help them better form blood clots. In a Phase III study on 138 hemophilia A sufferers, the drug met its primary endpoint of controlling and preventing bleeds in patients taking it as a twice-a-week prophylactic therapy. The drug resulted in no serious adverse events and no treatment-emergent inhibitors, the company said.
Now Baxter and partner Nektar Therapeutics ($NKTR) are planning to submit an FDA application for BAX 855 by year's end, eyeing a near-term approval for a drug that is essentially a longer-acting version of its top-selling factor VIII treatment Advate. The company's European application will have to wait on the results of an ongoing pediatric study, per EU regulations.
If approved, BAX 855 would have to contend with a coming crop of long-acting factor VIII treatments, led by Biogen Idec's ($BIIB) recently approved Eloctate, which can be dosed every three to five days. Novo Nordisk ($NVO) and Bayer are nearing approval for similar drugs, poised to expand a hemophilia market analysts say could reach more than $11 billion by 2016.
Baxter's latest clinical success comes on the heels of a Phase III victory for BAX 111, which replaces a different missing protein that prevents coagulation in patients with the rare von Willebrand disease. Baxter plans to submit that drug for FDA scrutiny later this year, using the agency's orphan pathway.
If everything goes according to plan, '855 and '111 will join Baxter's multibillion-dollar bleeding franchise, led by Advate and Feiba. The company is also working through Phase III for a factor VIIa treatment and Phase I for a hemophilia gene therapy, rounding out a pipeline Baxter believes is worth $2 billion a year at its peak.
Meanwhile, the company is preparing to split its sprawling med tech operation from its hemophilia-focused pharma business. By the middle of next year, Baxter expects to consolidate its dialysis-dominant device segment under the old company name and spin out its drug business under an as-yet-undisclosed brand, 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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