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RNS Number : 2938J
Syncona Limited
04 December 2018
Syncona Limited
Freeline presents initial data at the ASH Annual Meeting
04 December 2018
Syncona Ltd, a leading healthcare company focused on founding,
building and funding global leaders in life science, notes the
announcement that its portfolio company, Freeline Therapeutics, has
presented on initial results from the ongoing B-AMAZE Phase 1/2
trial investigating FLT180a for Haemophilia B at the 60th Annual
meeting of the American Society of Hematology (ASH) in December
2018 in San Diego, USA.
The announcement can be accessed on Freeline's website at:
http://www.freelinetx.com and full text of the announcement from
Freeline is contained below.
[ENDS]
Enquiries
Syncona Ltd
Annabel Clay
Tel: +44 (0) 20 7611 2031
Tulchan Communications
Martin Robinson
Lisa Jarrett-Kerr
Tel: +44 (0) 207 353 4200
Copies of this press release and other corporate information can
be found on the company website at: www.synconaltd.com
About Syncona:
Syncona is a leading FTSE250 healthcare company focused on
founding, building and funding 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 gene and cell therapy. We focus on
delivering dramatic efficacy for patients in areas of high unmet
need.
Freeline Presents Data on AAV-based Gene Therapy for Haemophilia
B at the 60th ASH Annual Meeting
A single infusion of FLT180a resulted in sustained mean Factor
IX activity of 45%, with no toxicity or adverse effects, and
eliminated need for patient infusions of FIX replacements
LONDON, December 3, 2018 - Freeline, a clinical stage gene
therapy company focused on providing life-changing curative
treatments for chronic diseases, on December 3(rd) presented
initial results from the ongoing B-AMAZE Phase 1/2 trial
investigating FLT180a for Haemophilia B. After one infusion, the
two patients dosed in the lowest dose cohort achieved mean FIX
activity levels of 45%+/-5, which approaches the normal range of
50-150%.
The presentation was made by UCL Professor Amit Nathwani, Chief
Scientific Officer of Freeline, Director of the Katharine Dormandy
Haemophilia Centre at the Royal Free Hospital and a Senior NIHR
Investigator. The study was sponsored by University College
London.
Two patients with severe Haemophilia B received FLT180a at a
single dose of 4.5 x 10(11) vector genomes/kg body weight.
Enrolment criteria included FIX activity levels <2% and no
neutralising antibodies to AAVS3, the company's proprietary high
performing liver specific capsid, as measured by a transduction
inhibition assay. Within 4 weeks of infusion, FIX activity in both
participants rose to greater than 30%, and at 15 weeks stabilised
at 45% +/-5%. This level has now been sustained for six and nine
months, respectively.
FLT180a was well-tolerated in these patients, with no infusion
related reactions or late toxicities, no evidence of transaminitis
and no evidence of neutralising anti-FIX antibodies. Following gene
transfer, there were no spontaneous bleeding episodes and no need
for FIX concentrate usage.
Professor Amit Nathwani, Founder and Chief Scientific Officer of
Freeline and Professor of Haemophilia at UCL Cancer Institute
said:
"Every day, I meet with patients whose lives are severely
disrupted by Haemophilia B and who aspire to live free of the
effects of this disease. These clinical results show that the
Freeline technology has the potential to provide these patients
normalisation of FIX activity levels."
Anne Prener, Chief Executive Officer of Freeline, added:
"Freeline has been built on the ambition of providing patients a
gene therapy with high protein expression. I am excited about the
results, which support escalation to the next dose level with the
goal of developing a functional cure for people with Haemophilia B.
These data provide initial validation in humans of our proprietary
capsid and manufacturing platform and support our plan to quickly
progress our broad pipeline of programmes in a variety of
diseases."
- Ends -
Further information:
JW Communications
Julia Wilson
+44 (0) 781 8430877
juliawilsonuk@gmail.com
Notes for Editors:
About Freeline
Freeline 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
that Freeline CSO and University College London Professor Amit
Nathwani published in the New England Journal of Medicine in 2011
and 2014.
About Haemophilia B
Haemophilia B is an X-linked disease characterized by recurrent
spontaneous and traumatic bleeds in all tissues, including the
joints, muscles and central nervous system. Haemophilia B is caused
by mutations of the F9 gene encoding for coagulation factor IX
(FIX) which plays an essential role in normal blood coagulation.
Manifestations of haemophilia can have lasting effects e.g.,
chronic arthropathy, life-threatening soft tissue bleeds, and CNS
haemorrhages which can prove fatal. The life expectancy for
Haemophilia B patients receiving treatment is 63-75 years dependent
on severity of disease, and just 11 years for those without access
to treatment.
Current treatment is aimed at preventing and treating bleeds
through replacement of the missing blood coagulation factors given
intravenously, when needed, from once every two weeks to twice
weekly. Prophylactic coagulation factor can reduce arthropathy,
bleeding episodes and other risks, but is a short-acting treatment
requiring a life-long commitment. Even with treatment, FIX levels
are unstable; the surges and deteriorations of FIX levels means
breakthrough bleeds with their accompanying symptoms and risks can
still occur during low FIX levels. Gene therapy treatment for
Haemophilia B addresses the limitations of current treatment,
offering potential for a one-time curative treatment.
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END
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