Scottish biotech company completes important milestone towards treatment of Haemophilia A.

Based outside Edinburgh at the Easter Bush Campus, the company has received £1.1million (the second tranche of funding from its £2m funding round announced in September 2019) to advance its R&D programme, which is moving closer to toxicology studies for its low cost recombinant human factor VIII (rhFVIII) for the treatment of Haemophilia A.

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.

As a key element of its product research and development programme, the initial process optimisation testing has successfully produced yields far in excess of those expected at the beginning of the development programme.

Jaymin Amin from PFP, said: “The results of our optimised process are extremely positive and 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.”

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, 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.”

According to the World Federation of Haemophilia, there are more than 400,000 sufferers of Haemophilia A worldwide but only ~173,000 are currently treated.  Annual sales of rhFVIII are currently $6 billion however there remains massive under-supply and significant unmet needs in other markets excluded from rhFVIII access due to current high product costs.

The company has developed a proprietary 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.