Bayer, Children’s Hospital of Philadelphia Working on Non-replacement Hemophilia Therapies

Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for hemophilia A and B.

Hemophilia is caused by a lack of blood clotting factors or their faulty production, often leading to uncontrolled bleeding after injury and to spontaneous bleeding.

Replacement therapy is a standard therapy for hemophilia, one that supplies the normal version of the missing clotting factor to patients. Hemophilia A patients are treated with on-demand and/or prophylactic (preventive) factor VIII (FVIII), while those with hemophilia B are treated with factor IX (FIX) replacement therapies.

However, many of the man-made version of clotting factors have short half-lives (time for a therapy to lose half its strength in the blood). Factor replacement therapy can also lead to the development of inhibitors (antibodies) against the supplied factor, which lessen its effectiveness. This means that replacement therapy has to be given as an injection to hemophilia patients often, usually multiple times a week.

An estimated 25% to 30% of children with severe hemophilia A develop inhibitors, as do an estimated 1% to 6% of patients with hemophilia B. According to the Centers for Disease Control and Prevention (CDC), care for a person who has inhibitors can cost an extra $800,000 each year.

Orally available small molecules would be a new class of therapy for hemophilia, and one with the potential to remove the burden of frequent injections.

Small molecules are substances that can easily enter cells due to their low molecular weight, and once inside the cells can affect other molecules. These features often make small molecules ideal for targeted therapies.

“Bayer is committed to investing and researching the next-generation of groundbreaking therapies. Small molecule therapies could help thousands of people with hemophilia A and B,” Joerg Moeller, a member of the executive committee of Bayer AG’s Pharmaceuticals division and head of research and development, said in a press release.

“This innovative approach is unprecedented in pharmaceutical history and would leverage significant opportunities for continued innovation in hemophilia,” Moeller added.

The Children’s Hospital of Philadelphia is an established pediatric research center, with expertise in hemophilia and coagulation. The collaboration between Bayer and the Children’s Hospital of Philadelphia is planned for three years, with the potential for extension.

During this time, Bayer will invest $5 million in the work. The company will have the option to exclusively license any therapies developed through the collaboration.