New divesiran data continue to show substantial
reduction in phlebotomy frequency and lowering of hematocrit levels
in PV patients
Silence also announces first subject dosed in
Phase 2 study of divesiran in PV patients
Company reinforces commitment to prioritize
divesiran development as the first-in-class siRNA in PV
Silence Therapeutics plc (“Silence” or the “Company”) (Nasdaq:
SLN), a global clinical-stage company developing novel siRNA (short
interfering RNA) therapies, today announced additional results from
the Phase 1 open label portion of the SANRECO study of divesiran, a
siRNA targeting TMPRSS6, in patients with polycythemia vera (PV)
were presented at the American Society of Hematology (ASH) Annual
Meeting being held in San Diego, California.
“Additional divesiran data presented at ASH continue to support
a compelling profile in PV and highlight the broad potential of our
siRNA platform to target both rare and common genetic diseases,”
said Craig Tooman, President and CEO at Silence. “Based on these
very encouraging results, we are committed to advancing divesiran
development as the first-in-class siRNA in PV and will prioritize
our resources to ensure we are well positioned to progress this
very promising program. To this end, we are pleased to have dosed
the first subject in the Phase 2 portion of the SANRECO study,
which is currently underway.”
Initial results from the SANRECO Phase 1 study were presented in
June 2024 and showed that all doses of divesiran substantially
reduced phlebotomy frequency and lowered hematocrit (HCT) in 16
phlebotomy dependent PV patients regardless of baseline HCT levels.
Additional data presented at ASH further support those findings and
included 19 PV patients with a combined history of 79 phlebotomies
prior to enrolment. Following divesiran dosing, only five
phlebotomies occurred during the 18-week treatment period – all
were in patients who entered the study with high baseline HCT
levels (over 45%). Two phlebotomies occurred in the 16-week
follow-up period following the last administered dose.
Consistent with results reported in June, there was a sustained
reduction in HCT during the treatment period and favorable effects
on indices of iron metabolism. Hepcidin levels increased and were
sustained within physiological levels in all dose groups,
demonstrating consistent target engagement. Importantly, divesiran
continues to be well tolerated to-date with no dose-limiting
toxicities.
“PV patients could benefit from a novel treatment option that
effectively manages their condition without causing serious adverse
effects,” said Marina Kremyanskaya, MD, PhD, Associate Professor of
Medicine, Hematology and Medical Oncology, at the Icahn School of
Medicine at Mount Sinai. “In the Phase 1 portion of the SANRECO
study, divesiran substantially reduced the need for phlebotomy and
lowered hematocrit levels following infrequent dosing in a range of
PV patients. I’m particularly impressed by the long duration of
effect and clean safety/tolerability profile. These data are very
exciting and support further development of divesiran in PV.”
The ASH presentation is available on the Company’s website,
linked here.
The Phase 1 portion of the SANRECO study has enrolled 21
patients and is ongoing until all patients complete follow-up which
is expected to conclude in February 2025. Silence also announced
today the first subject has been dosed in the Phase 2 portion of
the SANRECO Study. Divesiran has FDA Fast Track and Orphan Drug
designation in the US.
SANRECO Phase 1 Study Design
The Phase 1 portion of SANRECO is a 34-week, open-label study
evaluating divesiran (3 mg/kg, 6 mg/kg and 9 mg/kg) administered
subcutaneously every 6 weeks for four doses, with a 16-week
follow-up period following the date of the last administered dose
in 21 PV patients. Key inclusion criteria include a PV diagnosis
and a history of requiring at least three phlebotomies in the last
six months or five in the last year prior to screening. Patients
are allowed to be on stable doses of cytoreductive agents. Given
the exploratory nature of this Phase 1 study, both well-controlled
patients - defined as those with HCT levels at 45% or less – as
well as those with HCT levels greater than 45% at baseline on
current standard of care treatment were enrolled.
About PV
PV is a rare, myeloproliferative neoplasm – a type of blood
cancer - characterized by the excessive production of red blood
cells, often resulting in elevated hematocrit levels. Elevated
hematocrit above 45-percent is associated with a four-times higher
rate of death from cardiovascular or thrombotic events. PV is
associated with a range of burdensome symptoms including fatigue,
cognitive disturbance and pruritis and additionally, longer term
can transform to myelofibrosis and Acute Myeloid Leukemia. The aim
of treatment is to maintain hematocrit less than 45%, a level that
is associated with a reduced incidence of thrombosis and
CV-associated death. The current standard of care includes repeated
phlebotomies to reduce hematocrit and/or cytoreductive agents to
reduce red blood cell production. There are currently no approved
therapies that specifically target red blood cells and
hematocrit.
About Divesiran
Divesiran is Silence’s wholly owned siRNA product candidate
developed from its proprietary mRNAi GOLD™ platform that “silences”
TMPRSS6 expressed almost exclusively in the liver. TMPRSS6 is a
negative regulator of hepcidin, the body's master regulator of iron
metabolism including its absorption, distribution, and storage. By
silencing TMPRSS6 in PV patients, divesiran aims to increase
hepcidin production and release by liver hepatocytes, leading to
the restriction of iron to the bone marrow and, thus, reducing the
excessive production of red blood cells, a process dependent on
availability of iron.
About Silence Therapeutics
Silence Therapeutics is a global clinical stage biotechnology
company committed to transforming people’s lives by silencing
diseases through precision engineered medicines created with
proprietary siRNA (short interfering RNA) technology. Silence
leverages its mRNAi GOLD™ platform to create innovative siRNAs
designed to precisely target and silence disease associated genes
in the liver, which represents a substantial opportunity. Silence
focuses on areas of high unmet medical need with programs advancing
in cardiovascular disease, hematology and rare diseases. Silence
also maintains research and development collaborations with
AstraZeneca and Hansoh Pharma, among others. For more information,
please visit https://www.silence-therapeutics.com/.
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the safe-harbor provisions of the Private Securities
Litigation Reform Act of 1995. These statements may be identified
by words such as “aims,” “anticipates,” “believes,” “could,”
“estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,”
“plans,” “possible,” “potential,” “seeks,” “will” and variations of
these words or similar expressions that are intended to identify
forward-looking statements, although not all forward-looking
statements contain these words. All statements in this press
release, other than statements of historical facts, are
forward-looking statements. Forward-looking statements in this
press release include, but are not limited to, statements
regarding: the Company’s clinical development activities and
timelines for divesiran, including the continued advancement of
Phase 2 clinical trial; expected clinical benefits, efficacy and
safety of divesiran and the potential to produce clinically
meaningful outcomes in PV patients; and the Company’s business
strategy and plans to focus on the development of divesiran as the
first-in-class siRNA product candidate for treatment of PV. Any
forward-looking statements are based on management’s current
expectations and beliefs of future events and are subject to a
number of risks and uncertainties that could cause actual events or
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements, many of which are
beyond the Company’s control. These risks and uncertainties
include, but are not limited to: the impact of worsening
macroeconomic conditions, including the conflict in Ukraine and the
conflict between Israel and Hamas, heightened inflation and
uncertain credit and financial markets, on the Company’s business,
clinical trials and financial position; the risk that success in
preclinical testing and earlier clinical trials is not replicated
in later clinical trials; the delay of any current or planned
clinical trials, whether due to patient enrollment delays or
otherwise; the Company’s ability to successfully demonstrate the
safety and efficacy of its product candidates and gain approval of
its product candidates on a timely basis, if at all; unexpected
safety or efficacy data observed during preclinical studies or
clinical trials; clinical trial site activation or enrollment rates
that are lower than expected; the Company’s ability to realize the
benefits of its collaborations and license agreements; changes in
expected or existing competition; changes in the regulatory
environment; the uncertainties and timing of the regulatory
approval process; and unexpected litigation or other disputes.
These and other risks and uncertainties are identified in the
section titled "Risk Factors" in the Company’s most recent Annual
Report on Form 20-F for the year ended December 31, 2023 filed with
the U.S. Securities and Exchange Commission (the “SEC”) on March
13, 2024 as updated by the section titled “Risk Factors” in the
Company’s Report on Form 6-K filed with the SEC on November 14,
2024, as well as its other documents subsequently filed with or
furnished to the SEC. The Company expressly disclaims any
obligation to update any forward-looking statements contained
herein, whether as a result of any new information, future events,
changed circumstances or otherwise, except as otherwise required by
law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241209815468/en/
Silence Therapeutics plc Gem Hopkins, VP, Head of IR and
Corporate Communications +1 (646) 637-3208
ir@silence-therapeutics.com
Media Relations MKC Strategies Mary Conway +1
(516) 606-6545 mconway@mkcstrategies.com
Silence Therapeutics (NASDAQ:SLN)
過去 株価チャート
から 11 2024 まで 12 2024
Silence Therapeutics (NASDAQ:SLN)
過去 株価チャート
から 12 2023 まで 12 2024
