ProFactor Pharma Ltd. (PFP), a UK biotech company, is pleased to announce it has been awarded a SMART: SCOTLAND grant from Scottish Enterprise. This grant has been awarded to perform a preclinical Proof-of-Concept study of a novel approach to significantly improve the half-life of recombinant human Factor VIII (rhFVIII), the clotting factor used to treat the rare genetic bleeding disorder haemophilia A.
Replacement therapy with (rh)FVIII is the primary treatment option for patients with haemophilia A, who lack sufficient levels of FVIII in their blood. Currently available standard half-life FVIII products have an onerous, intravenous dosing frequency of several times per week when used prophylactically. Several enhanced half-life FVIII products are available today but most of these have only marginally reduced the dosing frequency, down to 1-2x per week. PFP has developed a novel concept for enhancing the half-life of Factor VIII that could potentially bring life changing improvements in dosing frequency versus the currently available products.
PFP’s pipeline also contains a standard half-life FVIII, that will soon be ready for further development in the clinic. This standard half-life rhFVIII is produced by a proprietary high expressing cell-line and high-yielding bio-manufacturing process, resulting in a low-cost rhFVIII. PFP believes that it can produce rhFVIII treatment at a significantly reduced cost, offering an affordable solution to underserved haemophilia A patients, ~70% of whom, mostly in low-middle income countries, remain untreated, with affordability being a key barrier for access to treatment.
It is expected that the same high-yielding, low-cost manufacture will be applicable to PFP’s enhanced half-life rhFVIII, thereby offering a rhFVIII that is both affordable and has a significantly reduced dosing frequency. PFP believes this disruptive innovation will address the needs of patients as well as healthcare providers in both developed and low-middle income countries.
About haemophilia A
Haemophilia A is a rare inherited bleeding disorder caused by deficiency of blood coagulation factor VIII (FVIII), affecting ~ 1 in 5,800 males. People with haemophilia bleed longer and more easily than other people; the frequency and severity of bleeds is dependent on the severity of the disease, which can vary from mild or moderate to severe. Replacement with (plasma/recombinant) human FVIII has been a standard of care treatment now for many years for haemophilia A. Worldwide, approximately 70% of all (prevalent) patients are untreated, according to the Annual Global Survey reports from the World Federation of Hemophilia (WFH).
About Scottish Enterprise
Scottish Enterprise (SE) is Scotland’s national economic development agency and a non-departmental public body of the Scottish Government. It supports businesses to innovate and scale to transform the Scottish economy by focusing on new market opportunities through targeted investment, innovation and internationalisation.
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