TIDMSYNC
RNS Number : 0013P
Syncona Limited
12 February 2021
Syncona Limited
Gyroscope Therapeutics Announces Positive Interim Data from
Phase I/II FOCUS Trial
12 February 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 Limited, a clinical-stage gene therapy company focused
on diseases of the eye, today announced positive interim safety,
protein expression and biomarker data from the ongoing open-label
Phase I/II FOCUS clinical trial of its investigational gene
therapy, GT005, in patients with geographic atrophy (GA) secondary
to age-related macular degeneration (AMD):
-- Interim data from 10 patients showed GT005 was well tolerated
in patients with GA secondary to AMD
-- Nine out of 10 patients treated with GT005 had increases in
Complement Factor I (CFI) levels, with an average increase of 146%
compared to baseline
-- Interim data demonstrated potential for single administration
of GT005 to down-regulate overactive complement system, which has
been strongly correlated with the development and progression of
AMD
-- These results were observed both in GA patients who had rare
variants in their CFI gene as well as those who did not
-- The data were presented today at the Angiogenesis, Exudation,
and Degeneration 2021 virtual meeting by Nadia Waheed, M.D., MPH,
Chief Medical Officer, Gyroscope Therapeutics
Chris Hollowood, Chief Investment Officer of Syncona Investment
Management Limited and Chair of Gyroscope said : "We are highly
encouraged by the early data from Gyroscope's Phase I/II FOCUS
trial, which suggests that the administration of GT005 leads to
sustained expression of CFI and down-regulation of the complement
system - a part of the inflammatory system which has been strongly
linked as a cause of dry AMD.
In parallel with generating this data, Gyroscope is building a
scalable manufacturing platform and delivery technology and is led
by a world-class management team, which has demonstrated a strong
track record of execution, despite challenging conditions over the
last 12 months. This positions the business well for the next phase
of its growth as it continues to progress its ongoing phase II
clinical trials and seeks to deliver on its promise to bring gene
therapy beyond rare disease to help the millions of patients
globally with dry AMD."
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 strategic 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 Positive Interim Data from
Phase I/II FOCUS Trial of
Investigational Gene Therapy GT005
Interim Data Showed GT005 Was Well Tolerated in Patients with
Geographic Atrophy (GA) Secondary to Age-Related Macular
Degeneration (AMD)
Majority of Patients Treated with GT005 Had Increases in
Complement Factor I (CFI) Levels, with an Average Increase of 146%
Compared to Baseline
Interim Data Demonstrated Potential for Single Administration of
GT005 to Down-Regulate Overactive Complement System, a Key Driver
of AMD
LONDON - Friday, 12 February, 2021 - Gyroscope Therapeutics
Limited, a clinical-stage gene therapy company focused on diseases
of the eye, today announced positive interim safety, protein
expression and biomarker data from the ongoing open-label Phase
I/II FOCUS clinical trial of its investigational gene therapy,
GT005, in patients with geographic atrophy (GA) secondary to
age-related macular degeneration (AMD). Interim results showed
GT005 was well tolerated and resulted in sustained increases in
vitreous Complement Factor I (CFI) levels in the majority of
patients, as well as decreases in the downstream complement
proteins associated with over-activation of the complement system.
These results were observed both in GA patients who had rare
variants in their CFI gene as well as those who did not. The data
were presented today at the Angiogenesis, Exudation, and
Degeneration 2021 virtual meeting by Nadia Waheed, M.D., MPH, Chief
Medical Officer, Gyroscope Therapeutics.
"Our investigational gene therapy, GT005, is designed to restore
balance to an overactive complement system and reduce inflammation
by increasing production of the CFI protein. We are excited by
these early results from the FOCUS trial that showed GT005 has been
well tolerated to date, increased CFI levels in a durable manner
and caused down-regulation of an overactive complement system,"
said Dr. Waheed. "These results give us confidence that a one-time
treatment with GT005 may have the potential to slow progression of
geographic atrophy, and this is being evaluated in our ongoing
Phase II clinical trials."
"There is strong evidence that an overactive complement system
is a key driver of dry AMD," said Arshad Khanani, M.D., M.A.,
Director of Clinical Research at Sierra Eye Associates, Clinical
Associate Professor at the University of Nevada, Reno School of
Medicine, and an investigator in the FOCUS trial. "The recently
released data from the FOCUS trial suggest the potential of a
one-time gene therapy with GT005 to regulate an overactive
complement system. It is encouraging that GT005 generated sustained
increases in CFI in a majority of the patients, even in some
patients treated more than a year ago. We continue to look forward
to learning more about GT005 as a potential treatment for GA in the
ongoing Phase II clinical trial programme."
Interim Data from the 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).
Interim results were reported today from patients in Cohorts 1
to 4. The three doses of GT005 evaluated were well tolerated and
there were no signs of GT005-induced inflammation.
-- There were no dose-related trends in the frequency or type of
adverse events and no GT005-related serious adverse events.
-- There was one possible GT005-related adverse event, which was
a suspected choroidal neovascularization of moderate intensity at
the patient's six-month follow up. This was successfully treated
with anti-vascular endothelial growth factor (VEGF) therapy.
-- There were 12 adverse events considered to be related to the
surgical procedure; the majority of these were mild (mild n=9;
moderate n=3).
Interim results showed sustained increases in vitreous CFI
levels in the majority of patients, as well as decreases in the
vitreous levels of key proteins associated with complement
over-activation (Ba and C3 breakdown proteins: C3b, iC3b and
C3c).
-- Nine out of 10 patients treated with GT005 had increases in
CFI levels, with an average increase of 146% compared to baseline
(p=0.02).
-- Of the nine patients with increased CFI levels, eight showed
sustained increases at week 24 and beyond, with one showing a
sustained increase at 84 weeks. The most recent measurement for the
ninth patient with increased CFI levels was week 12.
-- Increases in CFI levels were observed in patients with rare
variants in the CFI gene as well as those who did not have rare
variants.
-- There was an average decrease of 41% in levels of the Ba
protein compared to baseline at weeks 24 to 56 (n=6; p=0.03), and
an average decrease of 42% in the C3 breakdown proteins compared to
baseline at weeks 24 to 56 (n=9; p=0.03). These decreases were
observed in patients with and without CFI rare variants.
-- There was a significant correlation between i ncreased CFI
levels and decreases in Ba levels (p=0.03).
Dr. Waheed's 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 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 dampen the system's
overactivity and reduce inflammation, with the goal of preserving a
person's eyesight.
As of December 2020, 22 patients had been dosed with GT005 in
the FOCUS trial across Cohorts 1 to 5. Dosing in Cohorts 1, 2, 3
and 5 is complete. Patients continue to be enrolled in the dose
expansion Cohort 4, which is planned to enrol up to 20 patients.
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.
Gyroscope is also evaluating GT005 in two Phase II clinical
trials. EXPLORE [ NCT04437368 ] and HORIZON [ NCT04566445 ] are
Phase II, multicentre, randomised, controlled 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. EXPLORE is enrolling people who
have GA secondary to AMD who have rare variants in their CFI gene.
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 50 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.
Syncona Ltd., our lead investor, helped us create a leading gene
therapy company 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:
www.gyroscopetx.com and follow us on Twitter ( @GyroscopeTx ) and
on LinkedIn .
# # #
Contact:
Charlotte Arnold
VP, 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 August 17, 2020. Accessed July 16, 2020.
[2] American Macular Degeneration Foundation. What is Macular
Degeneration? https://www.macular.org/what-macular-degeneration.
Page last reviewed December 20, 2017. Accessed February 11,
2021.
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
RNS may use your IP address to confirm compliance with the terms
and conditions, to analyse how you engage with the information
contained in this communication, and to share such analysis on an
anonymised basis with others as part of our commercial services.
For further information about how RNS and the London Stock Exchange
use the personal data you provide us, please see our Privacy
Policy.
END
PFUBUGDDRSBDGBD
(END) Dow Jones Newswires
February 12, 2021 10:49 ET (15:49 GMT)
Syncona (LSE:SYNC)
Historical Stock Chart
From Apr 2024 to May 2024
Syncona (LSE:SYNC)
Historical Stock Chart
From May 2023 to May 2024