Oral therapy for hemophilia

WHIPPANY, N.J.—Bayer announced in December that it had entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules to develop a first-in-class oral non-replacement therapy for the treatment of hemophilia A and B. The partnership will combine CHOP’s expertise in hemophilia and coagulation and Bayer’s research capabilities.

Hemophilia is a genetic bleeding disorder in which one of the clotting proteins needed to form blood clots in the body is missing or defective. Worldwide, it is estimated that more than 400,000 people live with hemophilia and approximately 75 percent of them receive inadequate treatment. The main treatment for hemophilia is called replacement therapy and is often administered multiple times a week to help replace the clotting factor that’s missing or low. An orally available small molecule for the treatment of hemophilia would be a completely new modality in the market, and has the potential to remove the burden of frequent injections from patients.

“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, and we are looking forward to combining our strength in hemophilia research with Children’s Hospital of Philadelphia, which is a leading institution in basic and clinical research in the field of hemophilia,” said Dr. Joerg Moeller, member of the executive committee of Bayer’s Pharmaceuticals Division and head of research and development. “This innovative approach is unprecedented in pharmaceutical history and would leverage significant opportunities for continued innovation in hemophilia.”

Under the terms of the agreement, Bayer is investing $5 million in the joint research over three years, with the option of continuing the collaboration with the agreement of both parties. Bayer will have an option to exclusively license the collaboration results.

Bayer has a strong background in hemophilia products, with Jivi at the forefront of the hemophilia replacement therapies market. The new research alliance with CHOP, a world-renowned pediatric research center, has the potential to change the treatment paradigm for patients, the partners say. In addition, Bayer is currently developing a gene therapy treatment for hemophilia A patients, which could radically reduce the frequency of treatment these patients have to undergo.