Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102
May 07 2024 - 7:00AM
Verve Therapeutics, a clinical-stage biotechnology company
pioneering a new approach to the care of cardiovascular disease
with single-course gene editing medicines, today announced that the
first patient has been dosed with VERVE-102 in the Heart-2 Phase 1b
clinical trial. The Heart-2 trial is enrolling adult patients with
heterozygous familial hypercholesterolemia (HeFH) or premature
coronary artery disease (CAD), two patient populations who require
deep reductions of low-density lipoprotein cholesterol (LDL-C)
levels in the blood for an extended period of time. Patients living
with HeFH have an inherited disorder characterized by elevated
blood levels of LDL-C starting early in life. Patients living with
premature CAD experience cholesterol-driven blockage of coronary
arteries early in life and are at high risk of further
complications. A lack of durable control of LDL-C levels in both
HeFH and premature CAD patients carries high lifetime risks for
cardiovascular events, including heart attack and sudden death.
“Dosing the first patient in the Heart-2 Phase 1b clinical trial
for VERVE-102 is an important step in the continued progress of our
pipeline,” said Sekar Kathiresan, M.D., co-founder and chief
executive officer of Verve Therapeutics. “We are focused on
transforming the care of atherosclerotic cardiovascular disease
(ASCVD) through gene editing medicines that can lead to sustained
reductions in blood cholesterol after a single course of treatment.
VERVE-102 is designed to turn off the PCSK9 gene with a goal of
durably lowering LDL-C in patients living with either HeFH or
premature CAD, and we look forward to evaluating the safety and
preliminary efficacy data of VERVE-102 in the Heart-2 trial. With
Clinical Trial Applications cleared in Canada and the United
Kingdom, we are actively enrolling patients in the Heart-2 trial
and expect to provide a data update on our PCSK9 program in
2025.”
VERVE-102 is a novel, investigational gene editing medicine
designed to be a single course treatment that permanently turns off
the PCSK9 gene in the liver to reduce disease-driving LDL-C.
VERVE-102 consists of messenger RNA expressing an adenine base
editor and an optimized guide RNA targeting the PCSK9 gene. In
addition, VERVE-102 uses a proprietary GalNAc-LNP delivery
technology which allows lipid nanoparticles to access and deliver
the gene editing medicine to liver cells using either the
asialoglycoprotein receptor (ASGPR) or the low-density lipoprotein
receptor (LDLR).
Heart-2 is an open-label Phase 1b clinical trial designed to
evaluate the safety and tolerability of VERVE-102 administration in
adult patients with HeFH or premature CAD who require additional
lowering of LDL-C. Endpoints also include pharmacokinetics and
changes in blood PCSK9 protein and LDL-C levels. The trial is a
single-ascending dose study that has an adaptive design. Heart-2 is
expected to include four dose cohorts each comprised of three to
nine patients with either HeFH or premature CAD. HeFH is diagnosed
based on high LDL-C levels, a personal or family history of ASCVD,
physical exam features, and/or mutations identified in certain
genes. Premature CAD is defined as evidence of ASCVD occurring in
men aged 50 years old or women 60 years old. Clinical Trial
Applications for the Heart-2 trial have been cleared in Canada and
the United Kingdom. For more information, please
visit https://clinicaltrials.gov/.
About Verve Therapeutics Verve
Therapeutics, Inc. (Nasdaq: VERV) is a clinical-stage genetic
medicines company pioneering a new approach to the care of
cardiovascular disease, potentially transforming treatment from
chronic management to single-course gene editing medicines. The
company’s lead programs – VERVE-101, VERVE-102, and VERVE-201 –
target genes that have been extensively validated as targets for
lowering low-density lipoprotein cholesterol (LDL-C), a root cause
of atherosclerotic cardiovascular disease (ASCVD). VERVE-101 and
VERVE-102 are designed to permanently turn off
the PCSK9 gene in the liver and are being developed
initially for heterozygous familial hypercholesterolemia (HeFH) and
ultimately to treat patients with established ASCVD who continue to
be impacted by high LDL-C levels. VERVE-201 is designed to
permanently turn off the ANGPTL3 gene in the liver and is
initially being developed for homozygous familial
hypercholesterolemia (HoFH) and for refractory hypercholesterolemia
where patients still have high LDL-C despite treatment with
maximally-tolerated standard of care therapies. For more
information, please visit www.VerveTx.com.
Cautionary Note Regarding Forward Looking
StatementsThis press release contains “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995 that involve substantial risks and
uncertainties, including statements regarding expectations for the
company’s Heart-2 trial, including the trial design and the
company’s ability to enroll patients; the timing and availability
of data for the PCSK9 program; and the potential advantages and
therapeutic potential of VERVE-102. All statements, other than
statements of historical facts, contained in this press release,
including statements regarding the company’s strategy, future
operations, future financial position, prospects, plans and
objectives of management, are forward-looking statements. The words
“anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,”
“intend,” “may,” “plan,” “potential,” “predict,” “project,”
“should,” “target,” “will,” “would” and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Any
forward-looking statements are based on management’s current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in, or implied by,
such forward-looking statements. These risks and uncertainties
include, but are not limited to, risks associated with the
company’s limited operating history; the company’s ability to
timely submit and receive approvals of regulatory applications for
its product candidates; advance its product candidates in clinical
trials; initiate, enroll and complete its ongoing and future
clinical trials on the timeline expected or at all; correctly
estimate the potential patient population and/or market for the
company’s product candidates; replicate in clinical trials positive
results found in preclinical studies and/or earlier-stage clinical
trials of VERVE-101, VERVE-102, and VERVE-201; advance the
development of its product candidates under the timelines it
anticipates in current and future clinical trials; obtain, maintain
or protect intellectual property rights related to its product
candidates; manage expenses; and raise the substantial additional
capital needed to achieve its business objectives. For a discussion
of other risks and uncertainties, and other important factors, any
of which could cause the company’s actual results to differ from
those contained in the forward-looking statements, see the “Risk
Factors” section, as well as discussions of potential risks,
uncertainties and other important factors, in the company’s most
recent filings with the Securities and Exchange Commission and in
other filings that the company makes with the Securities and
Exchange Commission in the future. In addition, the forward-looking
statements included in this press release represent the company’s
views as of the date hereof and should not be relied upon as
representing the company’s views as of any date subsequent to the
date hereof. The company anticipates that subsequent events and
developments will cause the company’s views to change. However,
while the company may elect to update these forward-looking
statements at some point in the future, the company specifically
disclaims any obligation to do so.
Investor ContactJen RobinsonVerve Therapeutics,
Inc.jrobinson@vervetx.com
Media ContactAshlea
Kosikowski1ABashlea@1abmedia.com
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