Novimmune Grants Shire Global Rights to Its Antibody-based Hemophilia A Treatment

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A.

Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47.

Shire, which is based in Dublin, is a global pharmaceutical company that focuses on treatments for rare diseases such as hemophilia A.

The companies did not announce the terms of their agreement.

A missing or defective blood-clotting element known as the factor VIII protein causes hemophilia A. Without functioning factor VIII, a clotting cascade that includes two other coagulation factors will not occur. Those factors are IXa and X.

The antibody therapy that Novimmune developed activates Factors IXa and FX in hemophilia A patients so their blood will clot.

“Novimmune is building on a collaboration initiated in 2015 with Shire,” Ed Holdener, chairman and CEO of Novimmune, said in a press release.  “We are delighted that our research efforts have produced several promising and potentially highly differentiated leads for improving coagulation in hemophilia A.”

Shire plans to continue developing Novimmune’s treatment. Its ultimate goal is to create a therapy that is better and safer than a widely used current treatment: recombinant and plasma-derived factor VIII replacement therapy.

“This novel program builds on our extensive monoclonal antibody (MAb) capabilities, as well as on our in-depth scientific expertise in hematology [blood work],” said Fritz Scheiflinger, who heads Shire’s global research program. “While further development and clinical trials are needed to fully evaluate this antibody, we are encouraged by the potential of the data that we have seen in early discovery and the promise it may hold for hemophilia A patients.”

Hemophilia A is one of the world’s 7,000 rare diseases, Shire noted. Collectively, they affect an estimated 350 million people. “Rare diseases, most of which are genetic and are present throughout a person’s entire life, pose a significant medical and economic burden for patients, communities and healthcare systems,” the company said.