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Freeline announces updated Factor IX data and expansion of its Haemophilia pipeline

- Freeline presents twelve-month clinical data for first cohort of two patients in FLT180a, an AAV gene therapy for Haemophilia B, at ISTH -

- Announces a new programme in Haemophilia A -


Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, presented long-term FLT180a data at the International Society on Thrombosis and Haemostasis (ISTH) congress in Melbourne Australia, 6-10 July 2019. In addition, the Company is pleased to announce that it is broadening its pipeline with a new preclinical programme, FLT210, to target Haemophilia A, a bleeding disorder caused by a deficiency in clotting factor VIII. 


New programme in Haemophilia A:


The new Freeline Haemophilia A programme leverages the company’s platform technology, including its proprietary adeno-associated viral (AAV) capsid, known as AAVS3. The programme is based on a proprietary approach and has yielded promising preclinical data which will be presented at a future scientific forum. By incorporating all aspects of the Freeline platform, the programme is designed to provide high and consistent expression at a relatively low dose. FLT210 will soon enter Investigational New Drug (IND) enabling studies.


Twelve-month clinical data update in Haemophilia B:

The Freeline Haemophilia B programme, FLT180a, uses the AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is in a Phase 1/2 trial known as B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia B. At ISTH Freeline presented data from the first cohort of two patients who were treated with the lowest study dose (4.5x1011vg/kg). Over the 12 months since infusion both patients’ liver enzymes remained within the normal range at all time points and the patients’ transgene expression was stable, with Factor IX levels of 40.5±4.5% at week 52, assessed by the one stage clotting assay using the SynthASilTM reagent. Following treatment, both patients were free of spontaneous bleeding episodes and did not require any Factor IX supplementation.

Freeline continues to enrol patients in the B-AMAZE trial, with the goal of optimising the dose to express Factor IX in the normal range of 50-150%, which would free patients from the symptoms of the disease.

Chris Hollowood, Executive Chairman of Freeline said:

“We are pleased to report updated data on the first two patients in our Haemophilia B trial. The results provide early validation for the potential durability and stability of our therapy at the lowest dose cohort. We continue to progress dose optimisation in additional patients and look forward to providing a further update as we seek to develop a functional cure for people with Haemophilia B.”

“We believe there is a significant opportunity for Freeline’s highly efficient capsid and manufacturing platform to offer gene therapies that substantially improve patients’ lives in a wide range of chronic systemic diseases, and we are seeking to leverage this same platform across Haemophilia A and B, Fabry and Gaucher Diseases, and ultimately into non-monogenic disorders.”

Poster & presentation for ISTH Congress were as follows:


Presenter: Jonathan Foley 

Session: Poster Networking Session

Abstract title: FLT180a: Next Generation AAV Vector for Haemophilia B - Long Term, Follow-up and

In-depth Analysis of Transgenic FIX Using One-stage, Chromogenic and Global Assays

Date: Sunday, 7 July 2019 from 6:30 – 7:30 pm

Location: Exhibition hall


Presenter: Amit Nathwani

Session: State-of-the-Art Session. ISTH-WFH Joint Session: Haemophilia Management

Presentation title: Gene Therapy

Date: Monday, 8 July 2019 from 8:00 – 8:25 am

Location: Plenary Hall SOA 08

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Last Updated: 09-Jul-2019