ApcinteX Ltd, a biotech company spun out of the University of Cambridge, announces the first dose of its investigational drug, SerpinPC, in a haemophilia patient following 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 the 5th of February this year. 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.
The company’s 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 benefitting at the moment. It is our hope that SerpinPC will provide a once-monthly subcutaneous prophylactic treatment for all.”
Tags: ApcinteX, haemophilia, Jim Huntington, SerpinPC, Trevor Baglin