ApcinteX logo

News & Media

ApcinteX starts clinical trial of novel haemophilia drug

22 October 2019, Cambridge, UK—ApcinteX Ltd, a biotech company spun out of the University of Cambridge in 2014, has begun dosing in a Phase I/II clinical trial of its investigational drug SerpinPC, a potential treatment for all types of haemophilia.

About 400,000 people worldwide have haemophilia, a genetic disorder that reduces the level of blood coagulation factors. Persons with haemophilia are therefore prone to bleeding that can be life-threatening if untreated. Currently 80% of haemophilia sufferers have limited access to treatment, without which life expectancy is about 10 years. The current standard of care for haemophilia is regular prophylactic intravenous infusion of the missing factor. The burden of care is compounded by breakthrough bleeds into joints and the development of damage similar to severe arthritis. In addition, about one third of those on factor replacement develop inhibitory antibodies that render treatment ineffective, greatly reducing care options and increasing the cost of care.

ApcinteX’s new drug, SerpinPC, is a protein designed to solve these problems. Instead of replacing the missing coagulation factor, SerpinPC helps the blood to clot by inhibiting an anticoagulant enzyme, activated protein C. The unique mode of action of SerpinPC results in a reduction in blood loss in animal models of haemophilia at low doses, and is well tolerated at high doses in normal animals.

The principal aim of the clinical trial protocol is to test the safety and tolerability of SerpinPC in healthy volunteers and persons with haemophilia. The first doses will be given to healthy volunteers, escalating to higher doses in patients with severe haemophilia. This will be followed by a six-month, multiple-dose study in haemophilia patients, with reduction in bleeding and factor usage as exploratory endpoints. The patient parts of the study will be preceded by an observational phase to establish baseline bleeding and factor use levels.

The company’s Chief Medical Officer, Trevor Baglin, said:
“We anticipate that a once monthly subcutaneous injection of SerpinPC will provide prophylaxis against bleeding for all forms of haemophilia, regardless of inhibitor status - a ‘one-size-fits-all’ treatment that is effective and safe.”


About ApcinteX

ApcinteX Ltd is a virtual biotech company (www.apcintex.com) which was spun out of the laboratory of Professor Jim Huntington at the University of Cambridge in 2014. It closed a £14 million series A financing round in 2016 which was led by Medicxi and IP Group. For enquiries, please contact info@apcintex.com.

About Medicxi

Medicxi is a European venture capital firm with the mission to create and invest in companies along the full drug development continuum. Medicxi was established by the former Index Ventures life sciences team, which has been active for over 20 years, and invests in both early and late-stage assets with a product vision that can fulfil a clear unmet need. GSK, Johnson & Johnson Innovation – JJDC, Inc., Novartis and Verily (an Alphabet company) have invested in Medicxi funds. Please see www.medicxi.com for more information. 

About IP Group

IP Group is a leading intellectual property commercialisation company which focuses on evolving great ideas, mainly from its partner universities, into world-changing businesses. The Group has pioneered a unique approach to developing these ideas and the resulting businesses by providing access to business building expertise, capital (through its 100%-owned FCA-authorised subsidiaries IP Capital and Parkwalk Advisors), networks, recruitment and business support. IP Group has a strong track record of success and its portfolio comprises holdings in early-stage to mature businesses across life sciences and technology. IP Group is listed on the Main Market of the London Stock Exchange under the code IPO. For more information, please visit our website at www.ipgroupplc.com.