Baxter ($BAX), on the eve of biotech spinout, declared Phase III victory for a hemophilia treatment designed to help patients not responding to standard therapy.
The drug, BAX 817, is designed to replace the missing blood factors that stop blood from clotting normally and cause hemophilia. Many hemophilia patients come to develop inhibitors of such factor-replacement therapies, and Baxter's latest treatment is designed with them in mind.
In an open-label Phase III study on hemophilia A or B patients with inhibitors, BAX 817 met its primary endpoint of treating acute bleeding episodes after 12 hours of administration, the company said. The injected drug notched an overall success rate of 92% after 12 hours, according to Baxter, and 89% of patients reported sustained bleeding control after 24 hours. And, importantly, the drug didn't spur the development of any new inhibitors or binding antibodies, Baxter said.
The company is saving full results for a medical meeting later this year, disclosing that no patients dropped out due to adverse events but that one was hospitalized after a major bleed.
Now Baxter is preparing for global regulatory filings for BAX 817, aiming to extend its wide range of hemophilia products with an eye on patients with inhibitors.
|Baxter R&D head John Orloff|
"The development of inhibitors remains one of the most significant challenges in treating hemophilia, as it may place patients at increased risk for life-threatening complications resulting from difficult-to-treat bleeding episodes," John Orloff, head of R&D at Baxter BioScience, said in a statement.
Baxter is months away from a long-planned split in two, in which its biopharma division will strike out on its own as the publicly traded Baxalta and leave med tech and dialysis services to its counterpart. In preparation, the soon-to-be Baxalta has signed a slew of deals to expand its pipeline beyond its home base of hemophilia, most recently buying autoimmune biotech SuppreMol for $225 million and signing a $970 million oncology deal with Merrimack ($MACK).
- read the release