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ApcinteX Ltd, a biotech company spun out of the University of Cambridge, announced today the results from Part 1 of its ongoing clinical trial, AP-0101, assessing the safety, tolerability and pharmacokinetics of its investigational drug SerpinPC in healthy volunteers and patients with severe haemophilia A and B. 

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 may provide safe and convenient prophylaxis for bleeding associated with all forms of haemophilia.

AP-0101 is a phase 1/2 clinical trial with a single ascending dose study (Part 1) followed by a six month multiple dose study (Part 2). Part 1 began in healthy volunteers (Part 1A) and was continued 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, tolerability and pharmacokinetic profile of SerpinPC, with reduction in bleeding episodes an exploratory endpoint. 

Five cohorts of healthy volunteers were successfully dosed in Part 1A in the UK, four by IV infusion and one by subcutaneous (SC) injection. The final cohort finished dosing on the 5th of February last year. There were no SerpinPC related adverse events (AEs), no injection site reactions and no increase in D-dimer levels. 

Four cohorts of three patients were dosed in Part 1B in Moldova  and Georgia, where all persons with haemophilia (PwH) are treated with replacement factor solely on demand when bleeds occur. Annualised Bleeding Rates (ABR) were calculated for each subject from prospective observation prior to exposure to SerpinPC (median 12 weeks, range 9 to 18 weeks). A total of 97 bleeds occurred in the pre-exposure observation period, with a median ABR of 35 (range 26 to 41). In the 8 weeks following a single SC injection of SerpinPC there was a 55% reduction in all bleeding and a 72% reduction in spontaneous joint and muscle bleeding. Five of the 12 subjects reported 0 spontaneous bleeds. Consistent with the mode of action of SerpinPC, no dose dependency of ABR reduction was observed. A total of 29 bleeds were recorded in the 8 week period following SerpinPC administration, all of which were effectively treated with factor concentrate, as per standard of care, without incident and without increase in D-dimer levels. No SerpinPC-related AEs, injection site reactions or anti-drug antibodies were observed.

Part 2 of the study is ongoing. Twenty-three subjects in three dose groups are receiving SerpinPC by monthly SC injection for six months. Results are due to read out in Q2 this year, and will be followed by a year-long open-label extension study at a flat monthly dose.

The company’s Chief Medical Officer, Trevor Baglin, who presented the results at the 14th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD2021) said: “It is early days, but the initial results are encouraging. We monitored patients’ bleeding in the months before dosing to enable them to roll seamlessly from Part 1B into Part 2, where reduction in ABR is an exploratory outcome, but also with the hope that we might see a hint of efficacy after a single dose of SerpinPC. The reduction in bleeding we observed in the two months following the dose surprised us, especially in this patient population with severe arthropathy and no history of prophylaxis. Although the safety signal is clear, the real effect of SerpinPC on ABR will take many months of exposure to ascertain.

About ApcinteX

ApcinteX Ltd is a virtual biotech company (www.apcintex.com) spun out of the laboratory of Professor Jim Huntington at the Cambridge Institute for Medical Research, University of Cambridge, with Cambridge Enterprise, in 2014. It closed a £14 million series A financing round in 2016 which was led by Medicxi and IP Group and a £9M series B round led by Medicxi in 2020. For enquiries, please contact [email protected].

About Cambridge Enterprise

Part of the University of Cambridge, Cambridge Enterprise supports academics, researchers, staff and students in achieving knowledge transfer and research impact. We do this by helping innovators, experts and entrepreneurs use commercial avenues to develop their ideas and expertise for the benefit of society, the economy, themselves and the University. For more information, please visit: www.enterprise.cam.ac.uk.