Scottish biotech business ProFactor Pharma Ltd: (PFP) has completed the optimisation of its process for the manufacture of recombinant Factor VIII, for the treatment of Haemophilia A.
In July 2020 we reported on the important development milestone that had been reached, enabling the release of the second tranche of the investment secured in 2019. Over the last 7 months, work has been focussed on optimising the process in collaboration with Ingenza Ltd, a significant investor in PFP and one of the UK’s leading industrial biotech companies. Modifications to the fermentation (upstream process – USP) have resulted in expression of recombinant human Factor VIII (rhFVIII) of up to 40 milligrams per Litre.
Jaymin Amin, the CEO of PFP said: “The results of our optimised process successfully produced yields far in excess of those expected at the beginning of the development programme. These results provide great confidence as we enter into pre-clinical toxicology studies next month.
Richard Cruse, the CFO & founder said: “The reason why PFP is able to produce so much more is down to its extremely robust, proprietary cell-line capable of supporting much higher cell densities in the fermentation process. The data available suggests the PFP expression level is probably more than 100 times more than the industry standard”
With this significant advantage, PFP projects a low cost of goods enabling their disruptive technology to address the sale of rhFVIII into the vast markets currently remaining unserved (according to the World Federation of Hemophilia (WFH), approximately 70% of sufferers are untreated)
PFP has developed a proprietary high yielding cell line which, when combined with a production process employing innovative, single use, disposable technology, can address a significant market opportunity with an affordable rhFVIII product for those markets locked out by high prices.
Haemophilia is an X-linked genetic disease associated with the dysfunction of the biological cascade leading to blood clotting. Coagulation factors are vital to the functioning of the mechanism that the human body uses to protect and repair itself from physical injury, by the formation of blood clots to seal ruptured blood vessels. Haemophilia patients have a reduced capacity to stop bleeding and are prone to bleeding internally, into joints and organs, and often require hospitalisation 3-4 times per year. Without proper treatment, haemophiliacs typically die from organ failure in their teens.Haemophilia A is treated with replacement therapy of a human blood protein, Factor VIII which is limited in supply – by the quantity of available plasma for fractionation and by the limited installed capacity for the production of recombinant Factor VIII. The WFH estimate that more than two thirds of haemophiliacs receive inadequate or NO treatment at all.