Verve Therapeutics Highlights Recent Company Progress and Reports Second Quarter 2023 Financial Results
2023年8月10日 - 7:30PM
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 reported recent
company progress and financial results for the second quarter of
2023.
“We are dedicated to bringing life-changing,
once-and-done medicines to patients with cardiovascular disease,”
said Sekar Kathiresan, M.D., co-founder and chief executive officer
of Verve. “Our primary focus is on developing medicines targeting
three pillars of cardiovascular risk: low-density lipoproteins
(LDL), triglyceride-rich lipoproteins (TRL) and lipoprotein(a)
(Lp(a)). We are making meaningful progress across the portfolio,
and we expect to report initial clinical data from the heart-1
Phase 1b clinical trial of VERVE-101, our first-in-class base
editor targeting PCSK9, in the fourth quarter of this year. In
addition, we are focused on completing the preclinical activities
and executing the clinical operations activities necessary to start
clinical trials for our second PCSK9 program, VERVE-102, and our
ANGPTL3 program, VERVE-201, next year. Finally, we are thrilled to
advance our Lp(a) program in collaboration with Lilly, an industry
leader in cardiometabolic disease. As Verve continues to evolve
with multiple assets in the clinic and a robust pipeline, our
efforts are supported by a strong financial position with a cash
runway extending into 2026.”
VERVE-101 Progressing in heart-1 Clinical
Trial
- VERVE-101, an in vivo base editing medicine delivered as a
one-time intravenous infusion, is designed to inactivate the PCSK9
gene in liver cells, turning off liver production of blood PCSK9
and thereby durably reducing LDL-C. VERVE-101 is being developed
initially for the treatment of heterozygous familial
hypercholesterolemia (HeFH).
- VERVE-101 is being evaluated in the Phase 1b heart-1 clinical
trial. Enrollment efforts are ongoing in New Zealand and the United
Kingdom.
- heart-1 is designed to evaluate the safety and tolerability of
VERVE-101 initially with dose escalation in cohorts of the highest
risk HeFH patients. In addition, the trial is designed to measure
PCSK9 protein changes in participants, and thereby assess early
proof of concept of the ability to base edit in the liver. In the
fourth quarter of 2023, Verve expects to report the initial safety,
pharmacodynamic, PCSK9, and LDL-C data for the four cohorts in the
dose-escalation portion of the heart-1 clinical trial.
- Verve continues to work with the U.S. Food and Drug
Administration to resolve the hold on its Investigational New Drug
Application for VERVE-101. Based on the progress of the heart-1
clinical trial, the company expects enrollment to be completed
outside the United States.
VERVE-102 On-Track for Clinical Trial
Initiation in First Half of 2024
- VERVE-102 is an in vivo base editing medicine that aims to
inactivate the PCSK9 gene in a similar way to VERVE-101. VERVE-101
and VERVE-102 share an identical guide RNA targeting PCSK9 as well
as similar mRNA expressing an adenine base editor; however,
VERVE-102 is delivered using the company’s proprietary GalNAc-LNP
delivery technology. Preclinical studies in mice and non-human
primates using VERVE-102 with the company’s GalNAc-LNP delivery
technology demonstrated effective in vivo liver gene editing and
significant PCSK9 protein reduction.
- Preclinical development to support a regulatory submission for
VERVE-102 began in early 2022, and, following regulatory clearance,
Verve expects to initiate a Phase 1b clinical trial with VERVE-102
for patients with HeFH in the first half of 2024.
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 and thereby durably reducing LDL-C and TRLs. VERVE-201 is
being developed initially for the treatment of homozygous familial
hypercholesterolemia (HoFH), a rare and often fatal genetic subtype
of premature 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.
- Preclinical data supporting a regulatory submission for the
initiation of Phase 1b clinical trial of VERVE-201 will be
presented at the European Society of Cardiology (ESC) 2023
Congress. Details of the oral presentation are as follows:
- Title: An investigational in vivo base editing
medicine targeting ANGPTL3, VERVE-201, achieves potent and
LDLR-independent liver editing in mouse models
- Session: What’s new in lipid lowering
- Data and Time: August 27, 2023 from
10:25-10:35 a.m. CEST
- Preclinical studies to support a regulatory submission for
clinical development of VERVE-201 are ongoing, and, following
regulatory clearance, Verve expects to initiate a Phase 1b clinical
trial with VERVE-201 in the second half of 2024.
Business Development
- In June 2023, Verve and Eli Lilly and Company (Lilly) entered
into a global research collaboration focused on advancing Verve’s
preclinical stage in vivo gene editing program targeting Lp(a).
Elevated Lp(a) is an established and genetically validated,
independent risk factor for ASCVD, ischemic stroke, thrombosis, and
aortic stenosis. Under the terms of the agreement, Verve received a
combined upfront payment and equity investment totaling $60
million. Research program costs through Phase 1 clinical trials
will be funded by Lilly. Verve is also eligible to receive up to
$465 million in research, development, and commercial milestones,
as well as tiered royalties on global net sales. In addition,
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). The
collaboration became effective in July following expiration of the
waiting period under the Hart-Scott-Rodino Antitrust Improvements
Act, and Verve expects research activities associated with the
Lp(a) collaboration to commence in the third quarter.
Upcoming Investor Events Verve
plans to participate in the following upcoming events:
- Canaccord Genuity 43rd Annual Growth Conference, August 10,
Boston, MA
- Stifel Biotech Executive Summit, August 15, Newport, RI
Second Quarter 2023 Financial
Results
- Cash Position: Verve ended the second quarter
of 2023 with $462.5 million in cash, cash equivalents, and
marketable securities. Verve expects its existing cash, cash
equivalents, and marketable securities, including the additional
$60.0 million upfront payment and equity investment from Lilly in
August 2023, to be sufficient to fund its operations into
2026.
- Collaboration Revenue: Collaboration revenue
was $2.1 million for the second quarter of 2023, which was related
to the collaboration agreement with Vertex Pharmaceuticals
Incorporated. There was no collaboration revenue in the second
quarter of 2022.
- Research & Development (R&D) Expenses:
R&D expenses were $47.3 million for the second quarter of 2023,
compared to $33.1 million for the second quarter of 2022.
- General & Administrative (G&A)
Expenses: G&A expenses were $13.4 million for the
second quarter of 2023, compared to $9.1 million for the second
quarter of 2022.
- Net Loss: Net loss was $54.0 million, or $0.87
basic and diluted net loss per share, for the second quarter of
2023, compared to $40.9 million, or $0.84 basic and diluted net
loss per share, for the second quarter of 2022.
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
Statements This 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 ability
to enroll patients in its ongoing heart-1 trial; the timing and
availability of clinical data from its heart-1 trial; the company’s
expectations related to the clinical hold on the IND for VERVE-101;
the expected timing of initiating clinical trials of VERVE-102 and
VERVE-201; the company’s research activities under the Lilly
collaboration; its research and development plans; the potential
advantages and therapeutic potential of the company’s programs,
including VERVE-101, VERVE-102, and VERVE-201; 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 Contact Jen Robinson
Verve Therapeutics, Inc. jrobinson@vervetx.com
Media Contact Ashlea Kosikowski
1AB ashlea@1abmedia.com
|
Verve Therapeutics, Inc. |
Selected Condensed Financial Information |
(in thousands, except share and per share
amounts) |
(unaudited) |
|
|
Three months ended June 30, |
|
Six months ended June 30, |
|
Condensed consolidated statements of
operations |
|
2023 |
|
|
2022 |
|
2023 |
|
2022 |
|
Collaboration revenue |
$ |
2,093 |
|
$ |
— |
|
$ |
3,497 |
|
$ |
— |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
47,260 |
|
|
33,125 |
|
|
94,370 |
|
|
57,614 |
|
General and administrative |
|
13,416 |
|
|
9,067 |
|
|
25,969 |
|
|
16,503 |
|
Total operating expenses |
|
60,676 |
|
|
42,192 |
|
|
120,339 |
|
|
74,117 |
|
Loss from operations |
|
(58,583 |
) |
|
(42,192 |
) |
|
(116,842 |
) |
|
(74,117 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
Change in fair value of success payment liability |
|
(662 |
) |
|
938 |
|
|
76 |
|
|
2,615 |
|
Interest and other income, net |
|
5,438 |
|
|
308 |
|
|
10,984 |
|
|
390 |
|
Total other income, net |
|
4,776 |
|
|
1,246 |
|
|
11,060 |
|
|
3,005 |
|
Loss before provision for income taxes |
|
(53,807 |
) |
|
(40,946 |
) |
|
(105,782 |
) |
|
(71,112 |
) |
Provision for income taxes |
|
(176 |
) |
|
- |
|
|
(176 |
) |
|
- |
|
Net loss |
$ |
(53,983 |
) |
$ |
(40,946 |
) |
$ |
(105,958 |
) |
$ |
(71,112 |
) |
Net loss per common share, basic and diluted |
$ |
(0.87 |
) |
$ |
(0.84 |
) |
$ |
(1.71 |
) |
$ |
(1.46 |
) |
Weighted-average common shares used in net loss per share, basic
and diluted |
|
61,953,992 |
|
|
48,674,873 |
|
|
61,871,158 |
|
|
48,623,330 |
|
|
|
|
|
|
|
|
|
|
Condensed consolidated balance sheet data |
June 30, 2023 |
|
December 31, 2022 |
|
Cash, cash equivalents and marketable securities |
$ |
462,476 |
|
$ |
554,808 |
|
Total assets |
$ |
589,131 |
|
$ |
679,223 |
|
Total liabilities |
$ |
123,909 |
|
$ |
128,291 |
|
Total stockholders' equity |
$ |
465,222 |
|
$ |
550,932 |
|
|
|
|
|
|
Verve Therapeutics (NASDAQ:VERV)
過去 株価チャート
から 5 2024 まで 6 2024
Verve Therapeutics (NASDAQ:VERV)
過去 株価チャート
から 6 2023 まで 6 2024