Date: 2015-02-12
Type of information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the 8th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Helsinki, Finland
Company: Baxter (USA - IL)
Product: BAX 335
Action
mechanism: gene therapy
Disease: hemophilia B
Therapeutic area: Hematological diseases - Genetic diseases - Rare diseases
Country: USA
Trial
details: Hemophilia B is a genetic X-linked bleeding disorder caused by a deficiency in blood-clotting Factor IX (FIX) activity. FIX is synthesized in the liver and circulates in the blood as a proenzyme. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes. This clinical program will test a gene transfer approach involving the use of a gene delivery vector carrying a FIX gene. This first-in-humans study is intended to evaluate the safety, kinetics, and if possible, the dose of AskBio009 required to achieve stable plasma FIX activity between 10% and 40% of normal activity. (NCT01687608)
Latest
news: * On February 12, 2015, Baxter provided an update on its gene therapy program, including progress on the Phase I/II open-label clinical trial assessing the safety and optimal dosing level of BAX 335, an investigational factor IX (FIX) gene therapy treatment for hemophilia B, during a sponsored symposium at the 8th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Helsinki, Finland . The trial is assessing the safety of ascending doses of BAX 335 to determine the optimal single dose in up to 16 adult patients with hemophilia B at treatment centers in the United States . The primary endpoint is the safety of a single dose of BAX 335 administered intravenously. Secondary endpoints include evaluation of the optimal dose to achieve stable therapeutic plasma FIX activity, as well as pharmacokinetics and immune response to treatment. As of the end of 2014, a total of six patients in three dosing cohorts have been treated in the trial with evidence of a dose-related response. No patients have developed FIX inhibitors to date. In the two highest dose cohorts, FIX activity levels around 10 percent or above have been observed in two patients, who also experienced no bleeding events. One of these patients showed elevated levels of liver enzymes indicative of an immune response, which is being treated with oral corticosteroids, per protocol. Immune responses have been reported in previous studies with gene therapy technology. \'\'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.\'\' In April 2014 , Baxter announced the acquisition of Chatham Therapeutics, LLC , an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio), and its developmental gene therapy programs. Chatham\'s Biological Nano Particles (BNP), an advanced recombinant adeno-associated virus- (rAAV-) based gene therapy technology, has shown potential therapeutic benefit in early studies. In addition to the research in hemophilia B, Baxter is also advancing plans to evaluate the gene therapy technology in the treatment of hemophilia A.
