- Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients -

- Additional data will be presented in 5 poster presentations -

LONDON, June 29, 2020 (GLOBE NEWSWIRE) -- Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces that Professor Pratima Chowdary of the Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital UK and UCL Cancer Institute, and Chief investigator for the study, will present data in a late-breaking abstract session at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on behalf of co-authors, which will be held virtually 12-14 July 2020. In addition, Freeline has five posters at the conference and will be presenting data on the Factor IX Padua variant and health economics from its Adeno-Associated Virus (AAV)-based haemophilia gene therapy platform.

“We are pleased to have an abstract on our novel investigational gene therapy treatment for haemophilia B accepted as a late-breaking abstract at the upcoming ISTH conference,” said Theresa Heggie, CEO. “Results we have presented previously from our lead program suggest that FLT180a has the potential to create sustained FIX activity levels in the normal range in patients with severe haemophilia B and has the potential to address a significant unmet medical need and advance the standard of care.” 

Late breaking abstract:

  • Title: A novel adeno associated virus (AAV) gene therapy (FLT180a) achieves normal FIX activity levels in severe Hemophilia B (HB) patients (B-AMAZE study)
    Presenter: Prof. Pratima Chowdary
    Session: Late Breaking and CPVOD-19 Oral Communication Session
    Date & Time: Monday, July 13, 2020 at 11.45 AM – 13.00 PM ET

Additional five abstracts/poster presentations:

  • Title: Multi-centre field study of one-stage and chromogenic Factor IX assays in samples containing the Factor IX Padua variant
    Abstract No: A-1120-0035-00542

  • Title: Mechanistic evaluation of Factor IX-Padua activity in chromogenic FIX and thrombin generation assays
    Abstract No: A-1120-0035-00721

  • Title: Feasibility of Using Hospital Episode Statistics in England to Assess the Resource Use and Outcomes in Haemophilia
    Abstract No: A-1120-0033-02562

  • Title: The Relationship between Societal Costs Associated with Haemophilia and Disease Severity: A Regression Analysis Using CHESS II Data
    Abstract No: A-1120-0033-02935

  • Title: Modelling the Cost of Equivalent trough Level across Gene Therapy and Factor IX Replacement Therapy in Haemophilia B
    Abstract No:  A-1120-0033-02961

Founded in 1969, the ISTH is the leading worldwide not-for-profit organization dedicated to advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. ISTH is an international professional membership organisation with more than 5,000 clinicians, researchers and educators working together to improve the lives of patients in more than 100 countries around the world.

Further information
JW Communications
Julia Wilson
+44 (0) 7818 430877

About Freeline
Freeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the UK and has operations in Germany and the US.

About Haemophilia
Haemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In Haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in Haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be ‘carriers’ of the affected gene and may experience symptoms. Haemophilia A is the most common type of Haemophilia affecting about one in every 5,000 males, while Haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

About FLT180a
The Freeline Haemophilia B programme, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial, B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia.