Freeline releases initial data for FLT180a

Syncona Limited

Freeline releases initial data for FLT180a

01 November 2018

Syncona Ltd, a leading healthcare company focused on investing in and building global leaders in life science, today announces that its portfolio company, Freeline, has released an abstract with initial data from the ongoing B-AMAZE Phase 1/2 clinical trial investigating a novel gene therapy, FLT180a for Haemophilia B.

Freeline is a leading, clinical stage AAV gene therapy company focused on liver expression for chronic systemic diseases. Syncona founded Freeline with Professor Amit Nathwani from University College London in 2015. FLT180a is a next generation gene therapy using a proprietary adeno-associated virus (AAV) capsid developed to better transduce human liver cells, and a codon optimised cassette. The trial is continuing.

The abstract notes that in the trial's first patient cohort:

·      Two patients with severe Haemophilia B received FLT180a at a single dose of 4.5 x 10¹¹ vector genomes/kg body weight, which was well-tolerated with no serious adverse events.

·      Within four weeks of infusion, FIX activity[1] in both participants rose to more than 30% and has since stabilised at 46% and 48%, respectively.[2]

Initial results will be presented by the Chief Scientific Officer of Freeline, UCL Professor Amit Nathwani, at the 60th annual meeting of the American Society of Hematology (ASH) December 2018 in San Diego. 

Syncona remains the sole institutional investor in Freeline alongside UCL Technology Fund. Syncona has an 80 per cent fully diluted stake in Freeline which is valued at cost and held at £63.5 million[3] following the first tranche of the Series B investment. 

ASH Annual Meeting Presentations Details:
PRESENTER:  Amit Nathwani, Chief Scientific Officer, Freeline Therapeutics
DATE: Monday, December 3, 2018
LOCATION: San Diego Convention Center, Room 30D
ABSTRACT TITLE: A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

[ENDS]

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[1] Level of Factor IX, an essential clotting protein

[2] The normal range of FIX activity in the general population's blood is between 50% and 150%

[3] As at 30 June 2018. Freeline is valued at cost. The business received £33.5 million in a Series A fund from Syncona prior to the Series B round. The first tranche of the Series B is £30.0 million.

Copies of this press release and other corporate information can be found on the company website at: www.synconaltd.com  

Forward-looking statements - this announcement contains certain forward-looking statements with respect to the portfolio of investments of Syncona Ltd. These statements and forecasts involve risk and uncertainty because they relate to events and depend upon circumstances that may or may not occur in the future. There are a number of factors that could cause actual results or developments to differ materially from those expressed or implied by these forward-looking statements and forecasts. Nothing in this announcement should be construed as a profit forecast. 

About Syncona:

Syncona is a leading FTSE250 healthcare company focused on investing in and building global leaders in life science. Our vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders.

We seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses.

We take a long-term view, underpinned by a deep pool of capital, and are established leaders in the development of genetic medicine, particularly gene and cell therapy. We focus on delivering dramatic efficacy for patients in areas of high unmet need.

About Freeline:

Freeline Therapeutics Ltd. is a privately-held clinical-stage biotechnology company focused on AAV based gene therapy targeting the liver. Our vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-time curative treatment. Freeline is headquartered in the UK and has operations in Germany and the US.

Our next generation gene therapy builds upon the pioneering work by the Freeline CSO, UCL Professor Amit Nathwani published in New England Journal of Medicine in 2011 and 2014.