The deal once again highlights a growing trend towards the use of Factor IX for prophylaxis treatment of Hemophilia B, which requires several infusions per week with the currently available drugs.
The extended half-life of Syntonix's FIX:Fc product could enable effective treatment for both prophylaxis and on-demand therapy with less frequent intravenous injections and thereby provide a new, improved therapy.
The total market potential for Factor IX products is estimated to be in excess of $600 million (€488 million) worldwide, per year.
Under the terms of the agreement, Biovitrum and Syntonix will jointly develop and commercialise FIX:Fc. Syntonix is responsible for marketing in North America and Biovitrum is responsible for marketing in Europe, Russia and the Middle East.
The companies will equally share the costs and profits for development and commercialisation of FIX:Fc.
Syntonix will receive milestone payments and an additional equity investment from Biovitrum based on the progress of the program. In the future, Biovitrum and Syntonix may decide to expand the collaboration to include additional long-acting protein products. Additional financial terms were not disclosed.
"We are pleased to develop FIX:Fc with Syntonix because Syntonix' SynFusion technology has resulted in a promising, long-acting recombinant Factor IX product that has the potential to reduce infusions required for Haemophilia B patients to manage their disease," said Mats Pettersson, CEO of Biovitrum.
Haemophilia is a rare hereditary disorder in which the ability of patients' blood to clot is impaired.
As a result, the patient suffers from excessive bleeding and uncontrolled internal bleeding, leading to pain and eventual permanent damage to joints and muscles. One form, Haemophilia B results from mutations that impair the production of Factor IX.
Increasingly, the normal mode of treatment for younger patients is a prophylaxis regimen where patients are infused two or three times per week to maintain a better circulating level of coagulation factor. Long term studies in Sweden and elsewhere demonstrate that such regimens greatly reduce if not eliminate progressive joint deterioration.
Wyeth Pharmaceuticals, a division of Wyeth, is just one of the main pharmaceutical companies who already produce a recombinant coagulation Factor IX.
Its BeneFIX Coagulation Factor IX (Recombinant) and ReFacto Antihemophilic Factor (Recombinant), is indicated for the control and prevention of hemorrhagic episodes in patients with haemophilia B (congenital factor IX deficiency or Christmas disease), including control and prevention of bleeding in surgical settings.
Likewise Avigen is conducting research on the application of its treatment to haemophilia B.
AV513 represents the first non-biological (i.e. non-gene therapy, non-protein) approach for the treatment of haemophilia and is the first such agent to treat multiple bleeding disorders (Haemophilias A and B and potentially FVII deficiency & severe von Willebrand's disease).
In additon, it has the potential to be the first therapy for haemophilia to be delivered orally. Concept and preliminary safety validations have been achieved utilising stringent haemophilic animal models with clinically relevant endpoints.
AV513 has been tested and demonstrated to significantly improve clotting time in both large and small animal models of haemophilia.