(By Balaseshan) Baxter International Inc. (NYSE: BAX) said it has submitted an Investigational New Drug (IND) application for its hemophilia A treatment BAX 855 with the U.S. Food and Drug Administration, after positive results from a phase 1 trial.
A phase 2/3 multi-center, open-label study called PROLONG-ATE will be initiated in the U.S. during first quarter of 2013 with more than 100 previously treated adult patients with severe hemophilia A to assess the efficacy, safety and pharmacokinetics of BAX 855 for prophylaxis and on-demand treatment of bleeding.
BAX 855 is a full-length longer-acting recombinant factor VIII (rFVIII) that was developed to increase the half-life of Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method (ADVATE) – the most widely chosen rFVIII in the world.
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A phase 1 prospective, open-label trial assessing the safety, tolerability and pharmacokinetics of BAX 855 was conducted in 19 previously treated patients age 18 years or older with severe hemophilia A who completed the trial.
The half-life (measuring the duration of activity of the drug in the body) of BAX 855 was about 1.5-fold higher compared to ADVATE. A longer half-life was achieved in all patients in the study using BAX 855, no patients developed inhibitors to either base molecule, BAX 855 or PEG, and no patients had allergic reactions.
Eleven adverse events were reported in eight patients across both treatment arms, but none was serious, treatment-related or resulted in withdrawal from the study.
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Through a collaboration with Nektar Therapeutics (NKTR), BAX 855 leverages proprietary PEGylation technology designed to extend the duration of activity of proteins. The technology has been shown to be safe and tolerable, and is used in various approved treatments.
Hemophilia is a rare genetic blood clotting disorder that primarily affects males. In people with hemophilia A, clotting factor VIII is not present in sufficient amounts or is absent. Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.
ADVATE was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives.
BAX is trading up 0.04% at $68.46 on Monday. The stock has been trading between $48.98 and $68.91 for the past 52 weeks.