Cambridge spin-out ApcinteX starts clinical trials of novel haemophilia drug

ApcinteX Ltd, a biotech company that 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 percent 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 osteoarthritis. In addition, about one third of those on factor replacement will 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.