Baxter AG and Jerini AG in Germany have announced progress in their collaborative research program to develop a non-intravenous therapy for the treatment of hemophilia.
Extensive in vitro and in vivo analysis has identified several synthetic lead molecules with promising pharmacological properties.
"Using our proprietary technology platform, we continue to make important progress in our collaboration with Baxter, the leader in hemophilia therapy," said Jens Schneider-Mergener, Jerini AG's chief executive officer. "We are encouraged by the identification of these molecules and the evaluation of the potential to treat hemophilia using a non-intravenous therapy with molecules that can be synthetically produced."
Scientists from Baxter and Jerini used proprietary tailored screening and analysis methods to design molecules with the ability to promote the coagulation of blood. Following further analysis, several principal molecules that showed promising activity in mouse models of hemophilia were selected for further development.
Because blood-clotting proteins are large, relatively unstable molecules, current hemophilia therapies can only be administered intravenously, which is an invasive and technically demanding procedure. It is presently not possible to administer these molecules by non-intravenous routes because of poor distribution or rapid inactivation of the molecule in the body. Therefore, potential non-intravenous therapy must be able to reach its target site quickly and effectively without being inactivated in the process.
"We are very pleased by the progress to date in this collaboration with Jerini," said Friedrich Scheiflinger, PhD, senior director of discovery research and technical assessment for Baxter's BioScience business. "This partnership builds on Baxter's rich history of innovation in hemophilia therapy development and underscores our long-term commitment to improving therapies for people with hemophilia."
The collaboration between Baxter and Jerini was initiated in 2001 and expanded in 2004. Under the terms of the agreement, Jerini received an upfront payment, with the potential for milestone payments for achieving discovery, preclinical and clinical goals as well as royalties in the event of marketing approval.
People with hemophilia do not produce adequate amounts of factor VIII or factor IX protein, which are necessary for blood to effectively clot. If untreated, patients with severe hemophilia have a greatly reduced life expectancy. According to the World Health Organization, more than 400,000 people in the world have hemophilia, corresponding to a prevalence of 15 to 20 in every 100,000 males born worldwide.
Baxter hemophilia research and development
Baxter is a global leader in hemophilia therapy, with innovations that span 40 years. Through its own scientific expertise and strategic partnerships, Baxter is applying novel technologies that will pioneer the next generation of hemophilia therapies. Baxter's goal is to greatly enhance treatment convenience through therapies that require fewer injections and offer non-intravenous dosing alternatives.
About Jerini AG
Jerini AG is a pharmaceutical company based in Berlin, Germany focusing on the discovery and development of peptide-based drugs. Having recognized the potential of peptides as natural starting molecules for drug discovery, the company has developed state-of-the-art technologies to identify and transform peptides into drugs. The company's lead product, Icatibant, is currently in Phase III clinical trials for the treatment of hereditary angioedema (HAE). It is estimated that between 1:10,000 and 1:50,000 people worldwide are affected with this disease. Based on its technology platform, Jerini has established several in-house development programs, which address indications within ophthalmology, oncology, and inflammatory therapeutic areas. The most advanced of these programs targets age-related macular degeneration (AMD), the leading cause of vision loss and blindness in people over the age of 55 in developed countries.
Baxter AG is a subsidiary of Baxter International Inc. (NYSE: BAX). Baxter International Inc., through its subsidiaries, assists healthcare professionals and their patients with the treatment of complex medical conditions, including cancer, hemophilia, immune disorders, kidney disease and trauma. The company applies its expertise in medical devices, pharmaceuticals and biotechnology to make a meaningful difference in patients' lives.
This release includes forward-looking statements concerning non-intravenous hemophilia therapy. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: continuing clinical developments in identifying and developing suitable molecules; results demonstrating the safety and efficacy of the products; other risks inherent in development of early-stage therapies; and other risks identified in the company's most recent filing on Form 10-Q and other SEC filings, all of which are available on the company's web site. The company does not undertake to update its forward-looking statements.