Fresh off of completing its spinout from Baxter, Baxalta has unveiled the positive data it's using to back an approval for BAX 855, a long-acting factor VIII-replacement therapy that's designed to do a better job at producing blood clots for hemophilia A patients.
The drug's twice-weekly dosing schedule produced a half-life extension of 1.4- to 1.5-fold compared with Advate, the older Baxalta drug it's designed to replace as the leaders in hemophilia scramble to field new and better drugs. Provided the FDA delivers an approval, the therapy will be sold as Adynovate.
Patients in the twice-weekly arm of the trial demonstrated a 95% reduction in median annualized bleed rate compared with the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating all bleeding episodes, 95.9% of which were controlled with one or two infusions at a median dose of 29.0 IU/kg per infusion.
This is an important drug for Deerfield, IL-based Baxalta, which has been setting up a new R&D shop in Cambridge, MA. Baxalta stepped off with a goal of adding 20 new drug approvals by 2020, and an initial success would help make the case that it can deliver on that promise.
Baxalta, though, is competing with a select group of market leaders that are scrambling to maintain or expand on their slice of the hemophilia market with next-gen drugs of their own. Biogen ($BIIB) gained the first approval for a long-acting factor VIII product with Eloctate, putting Advate on the defensive. Bayer's been at work on a Phase III for BAY 94-9027, a long-acting factor VIII replacement therapy that might work with once-weekly dosing. And Novo Nordisk ($NVO) and Biogen, along with its partner Sobi, have their own slate of new and experimental therapies in the pipeline.
Biogen recently launched a gene therapy program for hemophilia, while only today Sobi chief Geoffrey McDonough said he was also interested in jumping in on what is fast becoming the new-new thing in hemophilia R&D.
And there's a lot at stake. Morningstar says the global hemophilia market will grow to $11.4 billion by 2016.
- here's the release on Baxalta