TIDMSYNC
RNS Number : 6117N
Syncona Limited
30 September 2021
Syncona Limited
Gyroscope announces additional positive interim data from Phase
I/II FOCUS trial
30 September 2021
Syncona Ltd, a leading healthcare company focused on founding,
building and funding a portfolio of global leaders in life science,
notes the announcement that its portfolio company, Gyroscope
Therapeutics Holdings plc ("Gyroscope"), a clinical-stage gene
therapy company focused on diseases of the eye, today announced
positive interim data from the ongoing open-label Phase I/II FOCUS
clinical trial of its investigational one-time gene therapy, GT005,
in people with geographic atrophy (GA) secondary to age-related
macular degeneration (AMD):
-- GT005 continues to be well tolerated with encouraging
biomarker results in patients with GA secondary to AMD
-- New data from Phase I/II FOCUS clinical trial reflect
additional patients and longer follow-up
The full text announcement is contained below and can be
accessed on Gyroscope's website at:
https://www.gyroscopetx.com/press-releases/ .
[S]
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.
Enquiries
Syncona Ltd
Annabel Clay
Tel: +44 (0) 20 3981 7940
FTI Consulting
Ben Atwell / Natalie Garland-Collins / Tim Stamper
Tel: +44 (0) 20 3727 1000
About Syncona
Syncona's purpose is to invest to extend and enhance human life.
We do this by founding and building companies to deliver
transformational treatments to patients in areas of high unmet
need.
Our strategy is to found, build and fund companies around
exceptional science to create a dynamic portfolio of 15-20 globally
leading healthcare businesses for the benefit of all our
stakeholders. We focus on developing treatments for patients by
working in close partnership with world-class academic founders and
management teams. Our balance sheet underpins our strategy enabling
us to take a long-term view as we look to improve the lives of
patients with no or few treatment options, build sustainable life
science companies and deliver strong risk-adjusted returns to
shareholders.
Gyroscope Therapeutics Announces Presentation of Positive
Interim Phase I/II Data for Investigational Gene Therapy GT005 at
Retina Society Annual Scientific Meeting
GT005 Continues to be Well Tolerated with Encouraging Biomarker
Results in Patients with Geographic Atrophy (GA) Secondary to
Age-Related Macular Degeneration (AMD)
New Data from Phase I/II FOCUS Clinical Trial Reflect Additional
Patients and Longer Follow-Up
LONDON - 30 September 2021 - Gyroscope Therapeutics Holdings plc
("Gyroscope"), a clinical-stage gene therapy company focused on
diseases of the eye, announced positive interim data from the
ongoing open-label Phase I/II FOCUS clinical trial of its
investigational one-time gene therapy, GT005, in people with
geographic atrophy (GA) secondary to age-related macular
degeneration (AMD) were presented today in an oral session at the
Retina Society's 54th Annual Scientific Meeting.
Safety data from 28 patients showed GT005 continues to be well
tolerated with no treatment-related serious adverse events. There
was no evidence of clinically significant GT005-induced
inflammation. Biomarker data from 13 patients, ranging from
approximately seven months post treatment to nearly two years,
continued to demonstrate sustained increases in vitreous complement
factor I (CFI) levels compared to baseline in the majority of
patients, as well as sustained decreases in downstream proteins
associated with complement system activation. A new analysis showed
no increases in systemic CFI levels circulating in the blood,
suggesting GT005's effects remain localised to the eye as
intended.
These data are being presented today by Szilárd Kiss, M.D.,
Associate Professor in Ophthalmology and Director of Clinical
Research in the Department of Ophthalmology, Weill Cornell Medical
College.
"These data build on the positive interim results we shared
earlier this year, reinforcing GT005's potential to restore balance
to an overactive complement system and continuing to show an
encouraging safety profile," said Nadia Waheed, M.D., MPH, Chief
Medical Officer, Gyroscope Therapeutics. "It's an exciting time in
complement research, and we believe a one-time gene therapy has the
potential to be a long-term solution for people with geographic
atrophy. We continue to collect data from FOCUS and enrol our Phase
II clinical trials evaluating the potential of GT005 to slow
progression of the devastating vision loss that GA inflicts."
GA is a leading cause of permanent vision loss for people aged
55 and older, and there are no approved treatments. 1 (,2) An
overactive complement system is believed to be a key driver of AMD,
and CFI is a natural regulator of complement system
overactivity.
Updated Interim Data from Phase I/II FOCUS Trial
FOCUS [ NCT03846193 ] is an open-label Phase I/II clinical trial
evaluating the safety and dose response of three doses of GT005
given as a single subretinal injection to patients with GA
secondary to AMD. The trial is divided into several cohorts,
including dose escalation ( Cohorts 1, 2, 3, 5 and 6) and dose
expansion (Cohorts 4 and 7). GT005 is delivered to patients in
Cohorts 1 to 4 using the standard transvitreal procedure and in
Cohorts 5 to 7 using Gyroscope's proprietary Orbit(TM) subretinal
delivery system.
Data were reported from patients in Cohorts 1 to 4.
Safety data from 28 patients showed:
-- There were no dose-related trends in the frequency or type of
adverse events and no GT005-related serious adverse events.
-- As previously reported, there was one possible GT005-related
adverse event, which was a suspected choroidal neovascularization
of moderate severity at one patient's six-month follow-up. This was
successfully treated with anti-vascular endothelial growth factor
(VEGF) therapy.
-- There were 16 adverse events considered to be related to the
surgical procedure; the majority of these were mild (mild n=12;
moderate n=4).
Biomarker data were reported from 13 patients who had received
GT005 at least 29 weeks before analysis:
-- Eleven out of 13 patients treated with GT005 had increases in
CFI levels, with an average increase of 122% compared to baseline
(p=0.002).
-- Of the 11 patients with increased CFI levels, all showed
sustained increases compared to baseline at their most recent point
of analysis (week 29 or beyond), with two of these patients showing
sustained increases at nearly two years (one at 84 weeks and one at
100 weeks).
-- There were sustained decreases in the vitreous levels of key
proteins associated with complement activation (Ba and C3 breakdown
proteins: C3b and iC3b).
-- An average decrease of 46% was observed in levels of the Ba
protein compared to baseline (n=11; p=0.001); and, an average
decrease of 46% was also observed in the C3 breakdown proteins
compared to baseline (n=13; p=0.001).
-- Increases in CFI levels and decreases in Ba and the C3
breakdown proteins were observed in patients with rare variants in
the CFI gene as well as those in the broader GA population.
The presentation will be made available on Gyroscope's website
at https://www.gyroscopetx.com/publications/ .
About GT005
GT005 is designed as an AAV2-based, one-time investigational
gene therapy for GA secondary to AMD that is delivered under the
retina. GT005 aims to restore balance to an overactive complement
system, a part of the immune system, by increasing production of
the CFI protein. Complement overactivation can lead to inflammation
that damages healthy tissues, and it has been strongly correlated
with the development and progression of AMD. The CFI protein
regulates the activity of the complement system. It is believed
that increasing CFI production could reduce inflammation, with the
goal of preserving a person's eyesight.
In addition to FOCUS, Gyroscope is evaluating GT005 in two Phase
II clinical trials. EXPLORE [ NCT04437368 ] and HORIZON [
NCT04566445 ] are Phase II, multicentre, randomised, controlled,
assessor-outcomes masked trials evaluating the safety and
effectiveness of GT005 administered as a single subretinal
injection. The primary endpoint for both trials is progression of
GA over 48 weeks (measured by change in GA area from baseline).
EXPLORE is enrolling people who have GA secondary to AMD and carry
rare variants in their CFI gene associated with low levels of CFI.
HORIZON is enrolling a broader group of people who have GA
secondary to AMD.
About Dry Age-Related Macular Degeneration (AMD) and Geographic
Atrophy (GA)
Dry AMD is a leading cause of permanent vision loss in people
over the age of 55 and is a devastating diagnosis.[1] There are
currently no approved treatments for dry AMD, which is the most
common form, impacting approximately 85-90% of people with AMD.[2]
As dry AMD advances, it leads to GA, an irreversible degeneration
of retinal cells, causing a gradual and permanent loss of central
vision. This disease can severely impact a person's daily life as
they lose the ability to drive, read and even see the faces of
loved ones.
About Gyroscope: Vision for Life
Gyroscope Therapeutics is a clinical-stage gene therapy company
developing gene therapy beyond rare disease to treat diseases of
the eye that cause vision loss and blindness. Our lead
investigational gene therapy, GT005, is currently being evaluated
in Phase II clinical trials for the treatment of geographic atrophy
(GA) secondary to age-related macular degeneration (AMD), a leading
cause of blindness. GT005 has received Fast Track designation from
the U.S. Food and Drug Administration for the treatment of people
with GA.
Supported by leading life sciences investors, Gyroscope has
built a global organisation combining discovery, research, drug
development, a manufacturing platform and surgical delivery
capabilities. Headquartered in London with locations in
Philadelphia and San Francisco, our mission is to preserve sight
and fight the devastating impact of blindness.
For more information visit: https://www.gyroscopetx.com/ and
follow us on Twitter ( @GyroscopeTx ) and on LinkedIn .
# # #
Contact:
Kate Archer
Director, Corporate Affairs
Gyroscope Therapeutics
media@gyroscopetx.com
[1] National Eye Institute. Age-Related Macular Degeneration.
https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration
. Page last reviewed June 22, 2021. Accessed September 28,
2021.
[2] American Macular Degeneration Foundation. What is Macular
Degeneration? https://www.macular.org/what-macular-degeneration .
Accessed September 28, 2021.
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