Midlothian biotechnology firm ProFactor Pharma seals 'game-changer' deal to broaden use of low-cost Haemophilia A treatment

Biotechnology company ProFactor Pharma (PFP) has inked an agreement it says could be “a real game-changer” for treating Haemophilia A globally, providing access to a market potentially worth more than £1.5 billion, as it prepares to embark on a funding round.

The Roslin-headquartered firm explains that the disorder involves not being able to make enough, or any, “Factor VIII” to stop bleeding. However, it says it has developed a proprietary cell line capable of producing Recombinant Human Factor FVIII (rhFVIII) at significantly higher yields, which when combined with a production process employing “innovative” disposable/single-use technology, delivers a low cost of goods.

It has now revealed the completion of a Product Development, Technology Transfer and Licence Agreement with PT Bio Farma (Persero), which will see Indonesia’s largest pharmaceutical company and manufacturer of vaccines and other biological products scale up PFP technology to industrial level and implement clinical trials in South-east Asia and India.

According to PFP, which in 2019 sealed a £2 million funding injection and counts Scottish venture capital firm Kelvin Capital as an investor, the tie-up opens an estimated $1.36bn (£1.52bn) total addressable market for treatment in India, Indonesia, Malaysia, Thailand, Philippines, Singapore, and Vietnam. The Scottish business added that under the deal, Bio Farma will make up-front and milestone payments of “several million” dollars to access its technology and royalties on sales, and comes as the Indonesian company already distributes products to 150 countries and will also supply PFP finished product for global sales.

From left: John McNicol of Kelvin Capital with PFP's CEO Jaymin Amin and board director Ian Fotheringham at the biotech firm's offices in Roslin. Picture: contributed.

PFP chief executive Jaymin Amin said: “This agreement has the potential to be a real game-changer for the treatment of Haemophilia A across the world, and significantly in the undersupplied markets in Asia, Middle East, Eastern Europe and South America that cannot afford to buy current treatments. There also is a huge undersupply of [rhFVIII] worldwide, which PFP is now well-placed to address. We will soon commence a major Series A funding round to begin human clinical trials, which will run over two to three years. Once concluded, we are confident that we will have a treatment that will be low-cost and accessible to every sufferer of Haemophilia A, no matter where they live.”

Also commenting was the firm’s chief financial officer Richard Cruse, who said: “This agreement demonstrates PFP is open to collaborations with pharma companies in these underserved markets to make treatment available not only via additional volumes, but also for those locked out by high prices.”

Honesti Basyir, president director of Bio Farma, said: “Collaboration with [PFP] will further maximise Bio Farma's innovation and performance, especially related to its vision and mission to produce quality life sciences products of international standard and affordable for people in various parts of the world.”

PFP cites research finding that there are 660,000-plus sufferers of Haemophilia A worldwide but only around 191,000 are currently treated, with a major challenge being a significant lack of provision in markets excluded from medications involving rhFVIII due to high prices.

Kerry Sharp, director of entrepreneurship and investment at Scottish Enterprise, said: “Scotland’s life sciences sector is internationally renowned… [PFP’s] agreement with Bio Farma demonstrates how Scottish companies can drive global innovation in the pharma sector and beyond. We look forward to continuing our partnership with the company as it delivers potential growth opportunities and medical advances.”