Low-cost Haemophilia A treatment deal unlocks world of opportunity for ProFactor Pharma

Low-cost Haemophilia A treatment deal unlocks world of opportunity for ProFactor Pharma

New agreement with Bio Farma opens estimated $1.36bn addressable Asian market

A new patented process to make low-cost treatment for Haemophilia A, developed by Scottish biotechnology company ProFactor Pharma Ltd (PFP), has taken an important step closer to market.

The successful completion of a Product Development, Technology Transfer and Licence Agreement with PT Bio Farma (Persero), will see Indonesia’s largest pharmaceutical company and manufacturer of vaccines and other biological products, undertake the scale-up of the PFP technology to industrial scale and implement clinical trials in Southeast Asia and India.

The agreement opens an estimated $1.36bn total addressable market for treatment in India, Indonesia, Malaysia, Thailand, Philippines, Singapore, and Vietnam. Bio Farma will make up-front and milestone payments of several million dollars to access PFP’s technology and royalties on sales. Bio Farma already distributes products to 150 countries around the world and will also supply PFP finished product for global sales under the agreement.

Haemophilia A is a genetic disorder where the Factor VIII gene is defective, meaning the patient does not make enough, or any, Factor VIII to stop bleeding. World Federation of Hemophilia research suggests the disorder affects 1/4,000 male births and that there are more than 660,000 sufferers of Haemophilia A worldwide but only around 191,000 are currently treated. A specific challenge is massive undersupply and significant unmet needs in markets excluded from proven treatments that involve Recombinant Human Factor VIII (rhFVIII), due to high prices.

PFP has developed a proprietary cell line capable of producing rhFVIII at significantly higher yields. This, when combined with a production process employing innovative disposable/single use technology, delivers a low cost of goods.

Jaymin Amin, CEO of ProFactorPharma, 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 Recombinant Human Factor VIII 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.”

Richard Cruse, CFO of ProFactor Pharma, 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 ProFactorPharma 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.”

Kerry Sharp, Director of Entrepreneurship & Investment at Scottish Enterprise, said: “Scotland’s life sciences sector is internationally renowned thanks to our incredible workforce, world-class universities and innovative companies that are located here.

“ProFactor Pharma’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.”