TIDMSYNC
RNS Number : 3787C
Syncona Limited
07 February 2020
Syncona Limited
Freeline publishes further data for FLT180a
7 February 2020
Syncona Ltd, a leading healthcare company focused on founding,
building and funding a portfolio of global leaders in life science,
announces that its portfolio company, Freeline, is presenting
further data from the ongoing B-AMAZE Phase 1/2 clinical trial
investigating a novel gene therapy, FLT180a for Haemophilia B, at
The European Association for Haemophilia and Allied Disorders
(EAHAD) conference.
The presentation is entitled: "Phase 1/2 preliminary data from
B-AMAZE study of adeno associated virus (AAV) gene therapy
(FLT180a) confirms progress towards achieving Factor IX levels in
the normal range for patients with severe or moderately severe
Haemophilia B."
Chris Hollowood, Chief Investment Officer of Syncona and
Executive Chairman of Freeline, said: "We are highly encouraged by
the data being generated in our lead programme in Haemophilia B. We
believe that Freeline has developed a product that could be
best-in-class for patients living with Haemophilia B. A functional
cure was an aspiration of Freeline's when the business was founded
just under five years ago and this data takes an important step
towards that goal. We look forward to identifying the optimal dose
in the coming months and subsequently moving the product into a
pivotal trial.
The data also demonstrates the power of our next generation
capsid. The step change in performance delivered by the capsid will
allow Freeline to lead the systemic gene therapy space beyond
Haemophilia to diseases, such as Fabry Disease and Gaucher Disease,
where high expression levels will be needed for meaningful clinical
impact."
Trial background
Freeline is a systemic gene therapy company focused on liver
expression of proteins for a range of chronic diseases. Freeline
uses a novel capsid which is capable of driving high protein
expression. FLT180a is a next-generation AAV gene therapy for
people with Haemophilia B that leverages this next-generation
capsid. Freeline is seeking to identify the optimal dosing regimen
for FLT180a with the goal of delivering to patients Factor IX (FIX)
activity levels between 70 to 150 per cent, in the upper part of
the normal range, which no other treatment has been able to achieve
to date.
The normal range of FIX activity in the general population's
blood is between 50 per cent and 150 per cent and patients
diagnosed with severe and moderate Haemophilia B have FIX activity
below five per cent.
Update on data
Reportable data[1] is available for eight patients who have been
treated across four dose cohorts with FLT180a. The first dose
cohort (two patients) has follow-up over 78 weeks, the next two
dose cohorts (two patients in each) have data available over 26
weeks and the most recent dose cohort (two patients) have FIX
activity level readings available after three weeks:
-- Dose Level One: Two patients treated with the lowest dose,
4.5x10(11) vector genomes/kg body weight and had average FIX level
activity at 78 weeks of 43.5%
-- Dose Level Two: Two patients received a single dose at
1.5x10(12) vector genomes/kg body weight and had average FIX level
activity at 26 weeks of 160%
o One patient within the cohort experienced a rise in ALT (see
below) that was followed by a decline in expression at 26 weeks
-- Dose Level Three: Two patients received a single dose at
7.5x10(11) vector genomes/kg body weight and had average FIX
activity at 26 weeks of 32%
o One patient within the cohort experienced a rise in ALT that
was followed by a decline in expression at 26 weeks
-- Dose Level Four: Two patients received a single dose at
9.75x10(11) vector genomes/kg body weight and had average FIX
activity at three weeks of 109%
All patients had severe or moderate Haemophilia B with baseline
FIX activity levels prior to gene therapy of 2 per cent or
less.
In the low dose cohort, stable expression of FIX activity has
now been seen for over 18 months. Six patients have completed
follow-up for six months, and amongst those, three have FIX
activity levels over 50%. The two patients in dose level four
(9.75x10(11) ) have shown encouraging early expression and the
current focus is to expand this dose cohort to establish safety and
efficacy.
No patients in the trial have had any bleeding episodes
requiring treatment or required any FIX supplementation post gene
therapy.
Alanine aminotransferase (ALT) is an enzyme that is
predominantly found in the liver. Damage to liver cells can lead to
release of more ALT in the bloodstream and therefore ALT levels in
the blood can be used as a marker of liver damage or toxicity, and
risk of loss of FIX expression. Systemic gene therapy programmes
often show rises in ALTs between months one and six. Freeline has
been developing its immunosuppression regimen throughout the trial,
to seek to identify a regimen that consistently controls ALTs and
therefore provides patients receiving therapy with the maximum
confidence of normal activity. The business continues to evolve
this and has implemented a regimen for dose level four involving
prophylactic corticosteroids and tacrolimus[2]. Immunosuppression
appears to be effective in controlling ALTs and there were no
clinical symptoms associated with ALT rises. No ALT rises were
observed in the low dose cohort (4.5x10(11) ).
Further data is in the table below.
Next steps
Freeline continues to enrol Haemophilia B patients as part of
its Phase 1/2 dose-ranging trial in order to identify the optimal
dose to achieve normal FIX activity levels and will provide a
further update in H2 CY2020.
Valuation
In line with Syncona's valuation policy, we continue to hold our
investment in Freeline at fair value based on cost until material
clinical data is generated, or a third-party financing or other
factor occurs, that indicates that cost is no longer the
appropriate measure of fair value. Syncona anticipates that the
point at which Freeline selects the optimal dosing regimen for
FLT180a and indicates that it will move to a pivotal trial, it will
constitute material clinical data.
Syncona remains the sole institutional investor in Freeline
alongside UCL Technology Fund. Syncona has a 79 per cent fully
diluted stake in Freeline which as at 31 December 2019 is valued at
GBP148.6 million.
Data table
Dose cohort Age BMI (kg/m(2) Week 3* Week 26 FIX Week 52 Week 78
) FIX (%)[3] (%)(3) FIX (%)(3) FIX (%)(3)
1: 4.5x10(11) 32 21.2
----- ------------- ------------ ------------ ------------
1: 4.5x10(11) 25 21.4 24.5 40 37.5 43.5
----- ------------- ------------ -------------- ------------ ------------
2: 1.5x10(12) 27 27.0 130 160** -
----- ------------- ------------ -------------- --------------------------
2: 1.5x10(12) 67 32.1
----- ------------- ------------ ------------ ------------ ------------
3: 7.5x10(11) 48 23.0 25.5 32** -
----- ------------- ------------ -------------- --------------------------
3: 7.5x10(11) 29 26.5
----- ------------- ------------ ------------ ------------ ------------
4: 9.75x10(11) 39 29.5 109 - -
----- ------------- -------------- ------------ --------------------------
4: 9.75x10(11) 48 31.0
----- ------------- ------------ ------------ ------------ ------------
*Patients received steroids after week 3
**One patient within this cohort experienced a rise in ALT that
led to a decline in expression at 26 weeks
[S]
Enquiries
Syncona Ltd
Siobhan Weaver / Annabel Clay
Tel: +44 (0) 20 3981 7940
FTI Consulting
Ben Atwell / Natalie Garland-Collins
Tel: +44 (0) 20 3727 1000
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 Limited. 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. In particular, many companies
in the Syncona Limited portfolio are conducting scientific research
and clinical trials where the outcome is inherently uncertain and
there is significant risk of negative results or adverse events
arising. In addition, many companies in the Syncona Limited
portfolio have yet to commercialise a product and their ability to
do so may be affected by operational, commercial and other
risks.
About Syncona
Syncona is a leading FTSE250 healthcare company focused on
founding, building and funding a portfolio of global leaders in
life science. Our vision is to build a sustainable, diverse
portfolio of 15 - 20 companies focused on delivering
transformational treatments to patients in truly innovative areas
of healthcare, through which we are seeking to deliver strong
risk-adjusted 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 strategic capital base which
provides us with control and flexibility over the management of our
portfolio. We focus on delivering dramatic efficacy for patients in
areas of high unmet need.
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.
[1] As at cut-off date of 24 January 2020
[2] An immunosuppressive drug widely used in transplantation
surgery.
[3] Local FIX activity assay data
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END
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