A SCOTTISH biotech company has completed an important development milestone in research that it says will transform the treatment of the blood clotting disorder Haemophilia A.

ProFactor Pharma (PFP) has received £1.1 million in the second tranche of support from its £2m funding round announced in September last year to advance its research and development programme.

The programme is moving closer to toxicology studies for its low-cost treatment, ahead of clinical trials and a fresh funding round next year.

According to the World Federation of Haemophilia, more than 400,000 have Haemophilia A globally but only 173,000 are currently treated, in part due to current high product costs. Even so, annual sales of rhFVIII are currently $6 billion.

PFP, which is based outside Edinburgh at the Easter Bush Campus, has already seen better-than-expected results from initial testing of the process it will use to produce its low-cost version of replacement therapy of human blood protein Factor VIII.

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The 2019 funding round was led by the investment syndicate Kelvin Capital and supported by Ingenza Ltd and the Scottish Investment Bank, the investment arm of Scottish Enterprise.

PFP says initial process optimisation testing has successfully produced yields far in excess of those expected at the beginning of the development programme.The company’s Jaymin Amin said: “The results of our optimised process are extremely positive. They are ahead of our expectations giving us greater confidence as we move towards toxicology studies in Q4 this year, prior to entering clinical trials and a Series A funding round next year.”

Series A investment is the first venture capital funding for a start-up.

Angus Hay from Kelvin Capital said: “These results are extremely exciting and reflective of the high-quality team at ProFactor Pharma. The company’s R&D activity continues to impress and exceed expectations which is very positive as we look towards a significant Series A investment in 2021.”

Kerry Sharp, director of the Scottish Investment Bank, said: “The test results are an incredibly encouraging development for the company, everyone active in the field of haemophilia research and the hundreds of thousands of people suffering the debilitating effects of the condition.

“It’s great to be able to support the development of a cost-effective treatment in Scotland that could have a massive global impact.”

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PFP says has developed a high-yielding cell line which, when combined with a production process employing innovative, disposable, single use technology, can address a significant market opportunity with a rhFVIII product that is competitively priced.

The Kelvin Capital syndicate, led by directors John McNicol and Angus Hay, represents private investors in the UK, Europe and the USA.

It initially invested in PFP in 2013 and has since led five rounds of follow-on funding.

Haemophilia is an X-linked genetic disease associated with dysfunction of the process leading to blood clotting. Patients are prone to bleeding internally, into joints and organs, and often require hospitalisation three or four times per year. Without proper treatment, they typically die from organ failure in their teens.