Baxter International Inc. recently provided an update on progress the company has made on its Phase I/II open-label clinical trial assessing the safety and optimal dosing level of BAX 335, an investigational factor IX (FIX) gene therapy. The trial is designed to determine the optimal single dose administered to up to 16 adult patients with hemophilia B at hemophilia treatment centers in the US. The updates were presented during a sponsored symposium at the 8th Annual Congress of the European Association for Haemophilia and Allied Disorders, February 11-13, 2015, in Helsinki, Finland.
To date, a total of six patients in three dosing cohorts have been treated in the trial, none of whom have developed FIX inhibitors. In the two highest dose cohorts, FIX activity levels of approximately 10% or above were reported in two patients, both of whom experienced no bleeding events. According to a Baxter press release, one of the patients showed elevated levels of liver enzymes indicative of an immune response and is being treated with oral corticosteroids. These drugs, similar to cortisones, are used to provide relief in areas of the body that are inflamed.
In April 2014, Baxter announced the acquisition of Chatham Therapeutics, LLC, an affiliate of Asklepios BioPharmaceutical, Inc., and its developmental gene therapy programs. As part of the agreement, Baxter acquired Chatham’s Biological Nano Particles™, a recombinant adeno-associated virus-(rAAV) based gene therapy technology for hemophilia B. Baxter is also advancing plans to evaluate the gene therapy technology in the treatment of hemophilia A.
“We continue to make steady progress in advancing our hemophilia B program with this technology and look forward to better understanding the applicability of this technology platform in hemophilia A patients as well,” said John Orloff, MD, vice president and global head of research and development at Baxter BioScience. “With the potential to redefine the treatment of hemophilia, this gene therapy technology is a central part of our R&D focus as we prepare to become an independent company this year.”
Source: Baxter press release dated February 12, 2015
