Thanks to meeting an important development milestone underlying a drive to absolutely transform the treatment of the blood clotting disorder Haemophilia A, Scotland-based ProFactor Pharma (PFP) recently received £1.1 million in the second tranche of support from a £2 million funding round announced in September 2019 to advance the development program. As it progresses towards toxicology, the Scottish venture has clinical trials in sight next year. The market needs a successful way to produce more recombinant Factor VIII at a lower cost to serve hundreds of thousands of underserved patients.
Haemophilia is a mostly inherited genertic disorder that impairs the body’s ability to actually make blood clots, the process that makes bleeding stop. Hence those that have this disease bleed for a longer time after an injury, bruising easily. Risks include bleeding inside of joints and in the brain. Mild cases are associated with symptoms only during an accident or during surgery. Stronger cases are very dangerous. The disease is often associated with hospitalization up to four times per year. Without the right treatment, patients typically die from organ failure by the teen years.
There are two main types of haemophilia, including 1) haemophilia A, which occurs due to low amounts of clotting factor VIII, and 2) haemophilia B, which is the result of low levels of clotting factor IX. Typically inherited from one’s parents via an X chromosome carrying a nonfunctioning gene, in rare occasions a new mutation could occur during early development or the disease could develop later in life due to antibodies forming against a clotting factor. Another type of the disease, haemophilia C, comes from not only low levels of factor XI but also parahaemophilia, a result of miniscule levels of factor V. There are cases of acquired haemophilia where a patient develops the disease in association with cancers, autoimmune disorders, and pregnancy.
The World Federation of Haemophilia claims that there are over 400,000 Haemophilia A patients worldwide and only 173,000 are presently in treatment. That high product costs precludes treatment is tragic for many, despite that those biotech companies selling rhFVIII generate a healthy $6 billion. Ideally existing companies would cut into their margin to potentially fund more treatment but often investors systematically push back on such a move. Hence disruption in the market from innovation is required, as better ways to treat this condition more economically could help save more children in what is a terribly underserved market.
The Investigational Product
Thus far, Jane Cassidy from The National of Scotland shares that the company, based on the edge of Edinburgh at the Easter Bush Campus, reports positive results from initial testing of the process it will harness to produce a low-cost version of replacement therapy of human blood protein Factor VIII. In fact, Ms. Cassidy further reports that the company’s initial process of optimization testing produced results far greater than what was expected.
Jaymin Amin, CEO and board member, shared, “The results of our optimized 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.”
The company came up with a way to make recombinant Factor VIII in a bioprocess delivering higher yields than the industry standard, reports the company on its website. They filed for patent protection and are driven, like many great Scottish initiatives over history, to drive superior yield with lower cost of goods via a combination of proprietary high expressing cell line (patented in Europe) with innovative single use disposable manufacturing technology. Add a patent pending, modern downstream purification technology, and when this comes together and works as intended, the goal is a high yield, high quality final product for the market.
In fact, they report on their website that based on expression data, their manufacturing model suggests a single 100L Single Use fermenter could produce 150 million International Units of rhFVIII per annum. Moreover, the process can scale as a 1,000L fermenter could yield 1.5 billion IUs. The net result: increase the world production of nearly 9 billion IUs. This will put competitors on notice.
Developing drugs and related biological products is a complex, risky and costly affair and but for investors—along with the scientific entrepreneurs—no progress would happen. In Scotland, thanks to an investment syndicate Kelvin Capital with the support Ingenza Ltd and the Scottish Investment Bank, the investment arm of the Scottish Enterprise, not only does the company have the capital to progress toward the next milestone (clinical trials), but those that would benefit form this new Factor VIII generating process have hope.
The Kelvin Capital syndicate includes private investors from the UK, Europe, and the United States. This Scottish investment group is committed to this cause. They commenced investment in ProFactor Pharma back in 2013 and have since then led five rounds of follow-on funding.
Lead investor, Kelvin Capital’s Angus Hay reported to The National, “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 commented as well on the positive outlook associated with this situation.
Call to Action: Those interested in the Haemophilia market should take note of ProFactor Pharma. Investor Watch may consider exploring participation, if possible, in the forthcoming A round in 2021.