Silence Therapeutics Announces Publication in Blood Demonstrating Role for Iron Regulation in Polycythemia Vera
April 04 2023 - 7:00AM
Business Wire
– Study provides therapeutic rationale for
SLN124, Silence’s clinical candidate for polycythemia vera and
other hematological disorders
Silence Therapeutics plc, Nasdaq: SLN (“Silence” or “the
Company”), an experienced and innovative biotechnology company
committed to transforming people’s lives by silencing diseases
through precision engineered medicines, today announced publication
of human genomic and in vivo preclinical data linking iron
regulation to polycythemia vera (PV) in the latest issue of Blood,
the medical journal of the American Society of Hematology (ASH),
linked here.
The paper is authored by senior medical and scientific experts
from Silence and leading population health and hematology
researchers from WEHI (Walter and Eliza Hall Institute) in
Melbourne, Australia as well as Cambride UK. The research, led by
WEHI’s Dr Cavan Bennett and Professor Sant-Rayn Pasricha,
identified links between PV and variants of the iron-regulating
gene HFE in a genomewide association study (GWAS) of 440 PV cases
and over 400,000 healthy controls. The study further demonstrated
in a mouse model of PV that hepcidin, a master regulator of iron
availability whose expression is influenced by HFE, governs the red
blood cell (erythroid) phenotype in PV.
“The phenotype of PV involves the over-production of red blood
cells, leading to a range of adverse symptoms and a high risk of
life-threatening cardiovascular events,” said Dr. Ute Schaeper,
Drug Discovery Project Leader at Silence and a co-author on the
paper. “The results we reported in Blood provide a genetic and
biological rationale for treating PV with SLN124 by raising
hepcidin to control systemic iron levels, thus reducing red blood
cell count. This therapeutic approach could potentially prevent the
need for patients to undergo periodic blood withdrawals to treat
their disease and reduce their risk of cardiovascular events
without the fluid shifts and severe iron deficiency associated with
phlebotomies.”
SLN124, an siRNA (short interfering RNA) targeting TMPRSS6, is
currently being studied in the SANRECO phase 1/2 study in adults
with PV. SLN124 is also being studied in other hematological
disorders such as beta thalassemia. SLN124 works by silencing
TMPRSS6, a gene that negatively regulates hepcidin expression, to
increase production of hepcidin in the liver.
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, iron levels
in the blood are expected to decrease. which could in turn allow
more healthy red blood cells to be produced, thereby improving
anemia in conditions of anemia with ineffective erythropoiesis and
iron overload, like β-thalassemia. In conditions of red cell
overproduction, like polycythemia vera, it is expected that SLN124
will attenuate the red cell hyperproliferation by hepcidin mediated
iron restriction. SLN124 has demonstrated proof of concept in
healthy volunteers and is currently being evaluated in the GEMINI
II phase 1 study in adults with thalassemia and the SANRECO phase
1/2 study in adults with PV. To learn more about the GEMINI II
study, please click here. To learn more about the SANRECO study,
please click here. SLN124 has rare pediatric disease and orphan
drug designations for beta-thalassemia as well as orphan drug
designation for PV. SLN124 also has FDA Fast Track Designation for
PV.
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™ 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 zerlasiran (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 hematological diseases. Silence also maintains ongoing
research and development collaborations with AstraZeneca,
Mallinckrodt Pharmaceuticals, and Hansoh Pharma, among others. For
more information, please visit
https://www.silence-therapeutics.com/.
About WEHI (Walter and Eliza Hall Institute of Medical
Research) WEHI is where the world’s brightest minds collaborate
and innovate to make life-changing scientific discoveries that help
people live healthier for longer. Our medical researchers have been
serving the community for more than 100 years, making
transformative discoveries in cancers, infectious and immune
diseases, developmental disorders, and healthy ageing.
WEHI brings together diverse and creative people with different
experience and expertise to solve some of the world’s most complex
health problems. With partners across science, health, government,
industry, and philanthropy, we are committed to long-term
discovery, collaboration, and translation. At WEHI, we are brighter
together. Find out more at www.wehi.edu.au.
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 cash
runway and forecast operating cash flow, the Company’s clinical and
commercial prospects, regulatory approvals of the Company’s product
candidates, potential partnerships or collaborations or payments
under new and existing collaborations, the initiation or completion
of the Company’s clinical trials and the anticipated timing or
outcomes 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 assumptions, beliefs, expectations,
estimates and projections about its industry. Words such as
“anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,”
“estimate,” 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 Annual Report
on Form 20-F filed with the U.S. Securities and Exchange Commission
on March 15, 2023. 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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Enquiries: Silence Therapeutics plc Gem Hopkins,
VP, Head of IR and Corporate Communications Tel: +1 (646) 637-3208
ir@silence-therapeutics.com
Silence Media Relations MKC Strategies Mary Conway
Tel: +1 (516) 606-6545 mconway@mkcstrategies.com
WEHI Media enquiries M: +61 475 751 811 E:
communications@wehi.edu.au
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