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Silence Therapeutics PLC
19 May 2021
Silence Therapeutics Announces Positive Data from GEMINI Phase 1
Study of SLN124 in Healthy Volunteers
- Data showed SLN124, an siRNA which targets TMPRSS6, was safe
and effective in reducing plasma iron levels and had a long
duration of action
- Data support ongoing phase 1 study of SLN124 in patients with
thalassemia and myelodysplastic syndrome (MDS)
- First clinical data from Silence's proprietary mRNAi GOLD(TM)
platform - two more clinical data readouts anticipated this
year
19 May 2021
LONDON, Silence Therapeutics plc , AIM: SLN and Nasdaq: SLN ("
Silence " or " the Company "), a leader in the discovery,
development and delivery of novel short interfering ribonucleic
acid (siRNA) therapeutics for the treatment of diseases with
significant unmet medical need, today announced positive topline
data from the GEMINI phase 1 study of its wholly owned product
candidate, SLN124, in healthy volunteers. SLN124, an siRNA which
targets TMPRSS6, is in development for the treatment of
iron-loading anemia conditions, thalassemia and myelodysplastic
syndrome (MDS).
The GEMINI phase 1, randomized, double-blind,
placebo-controlled, single-ascending dose study evaluated the
safety and tolerability of SLN124 (1.0, 3.0 and 4.5 mg/kg doses) in
24 healthy volunteers (18 active and 6 placebo). Pharmacokinetic
parameters and pharmacodynamic biomarkers of iron metabolism were
also measured to assess reduction in iron.
Initial data from the study showed all doses of SLN124 were
generally well-tolerated with no serious or severe treatment
emergent adverse events (TEAEs) or TEAEs leading to withdrawal.
TEAEs did not appear to be dose dependent and the majority were
mild, including transient injection site reactions which resolved
without intervention.
Notably, up to an approximate four-fold increase in average
hepcidin and 50% reduction in plasma iron levels were also observed
after a single dose of SLN124. Effects on hepcidin and iron appear
to be dose dependent and were still observed at the end of the
8-week study at all dose levels, indicating a sustained and long
duration of action.
These clinical data support preclinical findings which
demonstrated SLN124 effectively improved red blood cell production
and reduced anemia by increasing levels of hepcidin - a key natural
regulator of iron balance and distribution in the body. The Company
expects to measure red blood cell production and effects on anemia
in the ongoing GEMINI II phase 1 study of SLN124 in people with
thalassemia and MDS, who unlike healthy volunteers have
significantly elevated iron levels.
Mark Rothera, President and CEO of Silence Therapeutics, said :
"These data represent the first clinical data from our mRNAi
GOLD(TM) platform and underscore the promising potential for our
technology to deliver precision medicines. We look forward to
further data in patients anticipated from both of our wholly owned
clinical programs later this year - the GEMINI II study of SLN124
for iron-loading anemia conditions and the APOLLO study of SLN360
for cardiovascular disease due to high lipoprotein(a)."
Giles Campion, M.D., EVP, Chief Medical Officer and Head of
Research & Development of Silence Therapeutics, said : "Today's
results confirm the strong preclinical profile of SLN124 in humans
- we observed excellent safety, robust gene knockdown expressed by
up to an approximate four-fold increase in average hepcidin along
with a 50% reduction in serum iron levels and a durable effect
which lasted throughout the study. We are encouraged by these data
in healthy volunteers and the opportunity for SLN124 to potentially
address iron-loading anemia conditions such as thalassemia and
MDS."
John Porter, M.D., Professor and Consultant Haematologist, Red
Cell Disorders Unit, University College London and University of
College London Hospitals , commented : "Despite advances in our
understanding of thalassemia and MDS, there are no existing
treatments that specifically target the underlying mechanisms of
these conditions as a way to improve the degree of anemia. There is
a major unmet need for a therapy that can provide safe and
continuous control of iron balance and distribution as a way to
improve the efficiency of red cell production. I'm encouraged by
data from the SLN124 study in healthy volunteers and look forward
to further clinical testing."
Silence expects to present full data from the GEMINI phase 1
study of SLN124 in healthy volunteers at an appropriate scientific
meeting later this year. In addition, the Company plans to report
data from the single-ascending dose portion of the ongoing GEMINI
II phase 1 study of SLN124 in people with thalassemia and MDS in
the second half of this year. SLN124 has Orphan Drug Designation
for both conditions and rare pediatric disease designation for beta
thalassemia.
Enquiries:
Silence Therapeutics plc Tel: +1 (646) 637-3208
Gem Hopkins, Head of IR and Corporate Communications
ir@silence-therapeutics.com
Investec Bank plc (Nominated Adviser and Tel: +44 (0) 20 7597
Broker) 5970
Daniel Adams/Gary Clarence
European PR Tel: +44 (0) 20 3709
Consilium Strategic Communications 5700
Mary-Jane Elliott/ Angela Gray / Chris
Welsh
silencetherapeutics@consilium-comms.com
About Thalassemia and Myelodysplastic Syndrome (MDS)
Thalassemia and MDS are both rare diseases that prevent a person
from producing enough healthy red blood cells. Low levels of
healthy red blood cells, known as anemia, result in less oxygen
being delivered to different parts of the body. This can cause
symptoms such as excessive tiredness and weakness. It can also lead
to other serious health problems, such as heart disease. People
living with thalassemia or MDS can also store too much iron in
their bodies, leading to a phenomenon called 'iron overload', which
damages organs such as the heart and liver.
Both conditions are typically treated with regular blood
transfusions, which add to the problem of iron overload. Iron
chelation therapy removes excess iron from the body using special
medicines. While it helps reduce the amount of iron in the blood
for people with thalassemia or MDS, it does not treat the
underlying cause of the condition or stop it from progressing.
There is, therefore, a need for therapies that directly address the
biological drivers of disease.
About SLN124
SLN124 is a gene 'silencing' therapy - one that is designed to
temporarily block a specific gene's message that would otherwise
trigger an unwanted effect. In this case, SLN124 aims to
temporarily 'silence' TMPRSS6, a gene that prevents the liver from
producing a particular hormone that controls iron levels in the
body - hepcidin. As hepcidin increases, it is hoped that iron
levels in the blood will decrease, which could in turn allow more
healthy red blood cells to be produced, thereby improving anemia.
In preclinical studies, SLN124 has shown positive effects on
improving levels of red blood cells and reducing harmful iron
levels.
SLN124 is now being studied in the GEMINI clinical trial
program. GEMINI II is a phase 1 study to investigate the effects of
SLN124 in people with thalassemia or myelodysplastic syndrome
(MDS), whose bodies produce fewer healthy red blood cells than
normal and who can store too much iron in their bodies. For more
information on the GEMINI II study, please click here .
About Silence Therapeutics
Silence Therapeutics is developing a new generation of medicines
by harnessing the body's natural mechanism of RNA interference, or
RNAi, to inhibit the expression of specific target genes thought to
play a role in the pathology of diseases with significant unmet
need. Silence's proprietary mRNAi GOLD(TM) platform can be used to
create siRNAs (short interfering RNAs) that precisely target and
silence disease-associated genes in the liver, which represents a
substantial opportunity. Silence's wholly owned product candidates
include SLN360 designed to address the high and prevalent unmet
medical need in reducing cardiovascular risk in people born with
high levels of lipoprotein(a) and SLN124 designed to address
iron-loading anemia conditions. Silence also maintains ongoing
research and development collaborations with AstraZeneca,
Mallinckrodt Pharmaceuticals, and Takeda, among others. For more
information, please visit https://www.silence-therapeutics.com/
.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking
statements within the meaning of the U.S. Private Securities
Litigation Reform Act of 1995 and other securities laws, including
with respect to the Company's clinical and commercial prospects and
the anticipated timing of data reports from the Company's clinical
trials. These forward-looking statements are not historical facts
but rather are based on the Company's current expectations,
estimates, and projections about its industry; its beliefs; and
assumptions. Words such as 'anticipates,' 'expects,' 'intends,'
'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions
are intended to identify forward-looking statements. These
statements are not guarantees of future performance and are subject
to known and unknown risks, uncertainties, and other factors, some
of which are beyond the Company's control, are difficult to
predict, and could cause actual results to differ materially from
those expressed or forecasted in the forward-looking statements,
including those risks identified in the Company's most recent
Admission Document and its amended Annual Report on Form 20-F filed
with the U.S. Securities and Exchange Commission on April 29, 2021.
The Company cautions security holders and prospective security
holders not to place undue reliance on these forward-looking
statements, which reflect the view of the Company only as of the
date of this announcement. The forward-looking statements
made in this announcement relate only to events as of the date
on which the statements are made. The Company will not undertake
any obligation to release publicly any revisions or updates to
these forward-looking statements to reflect events, circumstances,
or unanticipated events occurring after the date of this
announcement except as required by law or by any appropriate
regulatory authority.
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