Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-101 in Patients with Heterozygous Familial Hypercholesterolemia
23 Ottobre 2023 - 12:30PM
Verve Therapeutics, Inc., a clinical-stage biotechnology company
pioneering a new approach to the care of cardiovascular disease
with single-course gene editing medicines, today announced the
lifting of the clinical hold and clearance of its Investigational
New Drug (IND) application by the U.S. Food and Drug Administration
(FDA) to conduct a clinical trial in the United States evaluating
VERVE-101 for the treatment of heterozygous familial
hypercholesterolemia (HeFH). VERVE-101 is an investigational, in
vivo base editing medicine designed to be a single-course treatment
that inactivates the PCSK9 gene in the liver to durably
lower blood low-density lipoprotein cholesterol (LDL-C). HeFH is a
prevalent and life-threatening inherited disease characterized by
lifelong elevations in blood LDL-C and accelerated atherosclerotic
cardiovascular disease (ASCVD).
Verve submitted interim clinical data from the
dose-escalation portion of the ongoing heart-1 Phase 1b clinical
trial and addressed the FDA’s preclinical questions in its response
to the clinical hold. The heart-1 trial is evaluating the safety,
tolerability, pharmacokinetic and pharmacodynamic profile of
VERVE-101 in patients with HeFH, and is currently being conducted
at sites in the United Kingdom and New Zealand. Verve is scheduled
to report interim clinical data from the heart-1 trial in a
late-breaking science presentation at the American Heart
Association’s (AHA) Scientific Sessions 2023 on November 12, 2023
at 3:30pm E.T. in Philadelphia.
“The clearance of our IND application by the FDA is
a significant milestone in our effort to offer patients living with
HeFH a transformative alternative to the chronic care model of
disease management. This clearance, for the first time, enables
clinical development of an in vivo base editing product
candidate in the United States,” said Andrew Bellinger, M.D.,
Ph.D., chief scientific officer of Verve. “Our interactions with
the FDA have been valuable, and we plan to incorporate our
learnings from this regulatory process to execute a global
regulatory strategy across the rest of our pipeline. With the
clearance of this IND, we plan to begin the process of activating
U.S. clinical trial sites for the heart-1 clinical trial.”
About heart-1heart-1 is an
open-label Phase 1b clinical trial in patients with heterozygous
familial hypercholesterolemia (HeFH) who have established
atherosclerotic cardiovascular disease (ASCVD) to evaluate the
safety and tolerability of VERVE-101 administration, with
additional analyses for pharmacokinetics and pharmacodynamic
reductions in blood PCSK9 protein and low-density lipoprotein
cholesterol (LDL-C). Interim clinical data from the dose escalation
portion of the heart-1 clinical trial including safety parameters
and changes in blood PCSK9 protein and blood LDL-C levels are
expected to be presented at the American Heart Association's
Scientific Sessions 2023. For more information, please visit
clinicaltrials.gov.
About VERVE-101VERVE-101 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 low-density lipoprotein
cholesterol (LDL-C). VERVE-101 is being developed initially as a
treatment for patients with heterozygous familial
hypercholesterolemia (HeFH), a prevalent and potentially
life-threatening inherited subtype of atherosclerotic
cardiovascular disease (ASCVD). VERVE-101 consists of an adenine
base editor messenger RNA and an optimized guide RNA targeting the
PCSK9 gene packaged in an engineered lipid nanoparticle. By making
a single A-to-G change in the DNA genetic sequence of PCSK9,
VERVE-101 aims to inactivate the target gene. Inactivation of the
PCSK9 gene has been shown to up-regulate LDL receptor expression,
which leads to lower LDL-C levels, thereby reducing the risk for
ASCVD.
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 initial three 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 cardiovascular disease, in order to durably reduce
blood LDL-C levels. 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 atherosclerotic
cardiovascular disease (ASCVD) patients not at goal on oral
therapy. 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
ultimately to treat patients with refractory hypercholesterolemia.
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
the enrollment of patients in the ongoing heart-1 clinical trial,
the timing and availability of clinical trial data from the heart-1
clinical trial, and the potential advantages and therapeutic
potential of the company’s programs, including VERVE-101. 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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