Verve Therapeutics, a clinical-stage biotechnology company
pioneering a new approach to the care of cardiovascular disease
with single-course gene editing medicines, today reported pipeline
updates and financial results for the quarter ended March 31, 2024.
“Our accomplishments in the first quarter bring us closer to
realizing our mission of protecting patients from cardiovascular
disease through single-course gene editing medicines. We are
excited to investigate VERVE-102 in the Heart-2 clinical trial,
having recently announced the first patient dosing in the trial,”
said Sekar Kathiresan, M.D., co-founder and chief executive officer
of Verve Therapeutics. “We also remain on track to initiate the
Phase 1b clinical trial for VERVE-201 targeting ANGPTL3 in the
second half of the year and are pleased with the progress we’re
making on our Lp(a) collaboration with Eli Lilly. Following these
recent achievements and program updates, we are focused on
continued execution across our pipeline as we develop our clinical
and preclinical programs addressing a robust set of validated
targets in areas of high unmet need.”
First Patient Dosed with VERVE-102 in Heart-2 Clinical
Trial
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
low-density lipoprotein cholesterol (LDL-C). VERVE-102 is being
developed initially as a treatment for patients with heterozygous
familial hypercholesterolemia (HeFH) or premature coronary artery
disease (CAD). VERVE-102 consists of messenger RNA expressing an
adenine base editor and an optimized guide RNA targeting
the PCSK9 gene, identical to VERVE-101. However,
VERVE-102 uses a different delivery system than VERVE-101, which
includes a different ionizable lipid and Verve’s proprietary GalNAc
liver-targeting ligand, which allows the lipid nanoparticle (LNP)
to access liver cells using either the asialoglycoprotein receptor
(ASGPR) or the low-density lipoprotein receptor (LDLR).
Verve recently dosed its first patient in the Heart-2 Phase 1b
clinical trial. Heart-2 is an open-label Phase 1b clinical trial
designed to evaluate the safety and tolerability of VERVE-102 in
adult patients with HeFH or premature CAD who require additional
lowering of LDL-C, with additional analyses for pharmacokinetics
and changes in blood PCSK9 protein and LDL-C levels. The company
received clearances of its Clinical Trial Applications (CTAs) in
Canada and the U.K. Verve expects to provide a data update on the
PCSK9 program in 2025.
Ongoing Analysis of Heart-1 Clinical Trial of
VERVE-101
Enrollment remains paused in the Heart-1 trial as Verve
continues to investigate the observed laboratory abnormalities
which Verve believes are attributable to the LNP delivery system.
As the company continues to work with regulatory authorities to
define a potential path forward, the VERVE-101 Investigational New
Drug Application (IND) in the U.S. and CTAs in the U.K. and New
Zealand remain active.
VERVE-201 on Track for Clinical Trial Initiation in
Second Half of 2024
VERVE-201, an in vivo base editing medicine delivered as a
one-time intravenous infusion, is designed to inactivate the
ANGPTL3 gene in liver cells, turning off liver production of blood
ANGPTL3 protein and thereby durably reducing blood LDL-C and
triglyceride-rich lipoproteins. For VERVE-201, Verve is utilizing
its proprietary GalNAc-LNP delivery technology. VERVE-201 is being
developed for the treatment of patients living with homozygous
familial hypercholesterolemia (HoFH), a rare and often fatal
inherited subtype of premature accelerated atherosclerotic
cardiovascular disease (ASCVD) characterized by extremely high
blood LDL-C. VERVE-201 aims to reduce the heavy treatment burden
associated with available therapies for HoFH, including the
requirement for multiple oral, injectable, and intravenous
infusions, often administered over decades. VERVE-201 is also being
developed for ASCVD patients with refractory hypercholesterolemia,
who have high LDL-C despite treatment with maximally-tolerated
standard of care therapies.
Verve has completed preclinical studies to support regulatory
submissions for clinical development. Verve expects to initiate the
VERVE-201 Phase 1b clinical trial in the second half of 2024,
subject to regulatory clearances.
First Milestone Achieved in Global Collaboration with
Eli Lilly
Verve achieved its first research and development milestone
related to its exclusive research collaboration with Eli Lilly
focused on advancing Verve’s research stage in vivo gene editing
program targeting LPA. Elevated Lp(a) is an established and
genetically validated independent risk factor for ASCVD, ischemic
stroke, and aortic stenosis.
Under the terms of the collaboration established in June 2023,
Verve will advance the research and development of the Lp(a)
program through the completion of Phase 1 clinical development.
Lilly will be responsible for subsequent development,
manufacturing, and commercialization of the Lp(a) program. Verve is
eligible to receive up to $465 million in research, development,
and commercial milestones, as well as tiered royalties on global
net sales. Following the completion of Phase 1 clinical trials,
Verve has the right to opt-in to co-fund and share margins globally
on the Lp(a) program (in lieu of receipt of milestones and
royalties).
Upcoming Medical Meeting Presentations
American Society of Gene and Cell Therapy (ASGCT)
27th Annual
Meeting: Verve will present an overview
of off-target analyses for VERVE-101.Presentation
title: Characterization of Guide RNA Site Consistency
Across Ancestries and the Potential for Off-Target Editing with the
Clinical-Stage Base Editing Medicine,
VERVE-101Track: Base Editing and Prime Editing
IIDate/time: Saturday, May 11, 2024, 10:30 – 10:45
a.m. ETLocation: Ballroom 3, Baltimore Convention
Center, Baltimore, MD
TIDES 2024: Verve will present
previously disclosed nonclinical and clinical data from the
company’s PCSK9 program.Presentation title:
Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9
Gene and Lower LDL-Cholesterol in HumansTrack:
Genome Editing Technology and
ApplicationsDate/time: Friday, May 17, 2024, 11:15
– 11:45 a.m. ETLocation: Hynes Convention Center,
Boston, MA
Upcoming Investor Event
Verve plans to participate in a fireside chat at the RBC Global
Healthcare Conference on Wednesday, May 15, 2024, at 8:00 a.m. ET
in New York.
A live webcast will be available in the investor section of the
company’s website at www.vervetx.com. The webcast will be archived
for 30 days following the presentation.
First Quarter 2024 Financial Results
Cash Position: Verve ended the first quarter of
2024 with $606.4 million in cash, cash equivalents, and marketable
securities. Verve continues to expect its capital position to be
sufficient to fund its operations into late 2026.
Collaboration Revenue: Collaboration revenue
was $5.7 million for the first quarter of 2024, compared to $1.4
million for the first quarter of 2023. The increase was primarily
due to an increase in research services performed under the
company’s collaboration agreements and cost reimbursements.
Research & Development (R&D) Expenses:
R&D expenses were $48.4 million for the first quarter of 2024,
compared to $47.1 million for the first quarter of 2023.
Stock-based compensation expense included in R&D expenses was
$5.6 million and $4.5 million for the first quarter of 2024 and
2023, respectively.
General & Administrative (G&A)
Expenses: G&A expenses were $14.2 million for the
first quarter of 2024, compared to $12.6 million for the first
quarter of 2023. Stock-based compensation expense included in
G&A expenses was $4.7 million and $3.5 million for the first
quarter of 2024 and 2023, respectively.
Net Loss: Net loss was $48.7 million, or $0.59
basic and diluted net loss per share, for the first quarter of
2024, compared to $52.0 million, or $0.84 basic and diluted net
loss per share, for the first quarter of 2023.
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 the company’s ongoing
Heart-2 clinical trial; the timing and availability of data for the
PCSK9 program; expectations for the company’s Heart-1 clinical
trial, including the company’s assessment of the laboratory
abnormalities observed in the trial and the company’s interactions
with regulatory authorities regarding VERVE-101; the receipt of
regulatory clearances and expected timing of initiating the
clinical trial of VERVE-201; its research and development plans;
the potential advantages and therapeutic potential of the company’s
programs; the potential milestone payments and potential royalties
on future sale under the Lilly collaboration; the potential co-fund
and margin share arrangement under the Lilly collaboration; and the
period over which the company believes that its existing cash, cash
equivalents and marketable securities will be sufficient to fund
its operating expenses. 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
Verve Therapeutics, Inc.Selected Condensed
Consolidated Financial Information(in thousands,
except share and per share
amounts)(unaudited) |
|
|
|
Three months ended March 31, |
|
Condensed consolidated statements of
operations |
|
|
2024 |
|
|
|
2023 |
|
|
|
|
|
|
|
|
Collaboration revenue |
|
$ |
5,695 |
|
|
$ |
1,404 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
48,376 |
|
|
|
47,110 |
|
General and administrative |
|
|
14,163 |
|
|
|
12,553 |
|
Total operating expenses |
|
|
62,539 |
|
|
|
59,663 |
|
Loss
from operations |
|
|
(56,844 |
) |
|
|
(58,259 |
) |
Other
income: |
|
|
|
|
|
|
Change in fair value of success payment liability |
|
|
78 |
|
|
|
738 |
|
Interest and other income, net |
|
|
8,136 |
|
|
|
5,546 |
|
Total other income, net |
|
|
8,214 |
|
|
|
6,284 |
|
Loss
before provision for income taxes |
|
|
(48,630 |
) |
|
|
(51,975 |
) |
Provision for income taxes |
|
|
(106 |
) |
|
- |
|
Net
loss |
|
$ |
(48,736 |
) |
|
$ |
(51,975 |
) |
Net loss
per common share, basic and diluted |
|
$ |
(0.59 |
) |
|
$ |
(0.84 |
) |
Weighted-average common shares used in net loss per share, basic
and diluted |
|
|
83,132,960 |
|
|
|
61,787,403 |
|
Condensed consolidated balance sheet data |
|
March 31,2024 |
|
December 31,2023 |
|
Cash, cash equivalents and marketable securities |
|
$ |
606,367 |
|
|
$ |
623,950 |
|
Total assets |
|
$ |
732,357 |
|
|
$ |
752,688 |
|
Total liabilities |
|
$ |
149,288 |
|
|
$ |
153,186 |
|
Total stockholders' equity |
|
$ |
583,069 |
|
|
$ |
599,502 |
|
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