ApcinteX advances disruption of $10bn haemophilia market
Cambridge University spin-out ApcinteX has started dosing haemophilia patients in the second phase of a clinical trial of its investigational drug, SerpinPC.
The move follows the successful completion of dose escalation in healthy volunteers.
Haemophilia affects approximately half-a-million people worldwide, the vast majority of whom have limited access to treatment. The unique mode of action of SerpinPC supports monthly subcutaneous prophylactic dosing and a favourable safety profile.
AP-0101, as the study is known, is a phase 1/2 clinical trial with the single ascending dose, which began in healthy volunteers (Part 1A) and is now continuing in patients with severe haemophilia at doses predicted to provide clinical benefit (Part 1B).
The primary purpose of the trial is to assess the safety and pharmacokinetic properties of SerpinPC, with reduction in bleeding episodes as an exploratory endpoint.
Four cohorts of three patients are planned in Part 1B, which is taking place in Moldova where most bleeds go untreated due to scarcity of replacement factor.
The trial is open to patients with severe haemophilia A and B, with or without inhibitors. Subjects who participate in Part 1B will have the option to continue into the six-month, multiple dosing Part 2 of the study. A second site in Georgia will be opened to support Part 2 which is scheduled to begin this summer.
Five cohorts of healthy volunteers were successfully dosed with SerpinPC in Part 1A of the study, which was conducted in the UK, four by IV infusion and one by subcutaneous injection. The final cohort finished dosing on February 5.
There were no SerpinPC related adverse events and no elevation in D-dimer levels – an early sign of excessive thrombin generation associated with other investigational haemophilia treatments.
ApcinteX chief medical officer, Trevor Baglin, said: “We are delighted that Part 1A is finished and that SerpinPC will now be administered to haemophilia patients, particularly in a setting where the case of need is palpable.
“It’s important to remember that although there are new treatments available, most people with haemophilia are not benefiting at the moment. It is our hope that SerpinPC will provide a once-monthly subcutaneous prophylactic treatment for all.”
ApcinteX is a virtual biotech company spun out of Professor Huntington’s Cambridge University with the support of Cambridge Enterprise, in 2014. It closed a £14 million series A financing round in 2016 which was led by Medicxi and IP Group.
The Uk technology business is seeking to disrupt a $10 billion haemophilia market by developing a drug that can be used in all patients, regardless of the type of haemophilia.
This novel treatment is based on the work of Professor Huntington and Dr Trevor Baglin (Cambridge University Hospitals) – both world-renowned experts in blood clotting disorders.