Verve Therapeutics, a clinical-stage company developing a new class
of genetic medicines for cardiovascular disease, today reported
pipeline updates and financial results for the quarter ended
September 30, 2024.
“In the third quarter, we made considerable progress towards our
mission to develop a new class of genetic medicines where a
one-time treatment leads to lifelong lowering of blood
cholesterol,” said Sekar Kathiresan, M.D., co-founder and chief
executive officer of Verve Therapeutics. “We continue to execute on
the Heart-2 clinical trial and are pleased to share that as of
October 29, 2024, seven participants have been dosed. VERVE-102 has
been well-tolerated, with no serious adverse events and no
clinically significant laboratory abnormalities observed. We look
forward to providing initial data from the Heart-2 clinical trial
and an update on the PCSK9 program in the first half of 2025.”
Dr. Kathiresan continued, “In addition, we are excited to
announce that the first participant in the Pulse-1 Phase 1b
clinical trial for our ANGPTL3 product candidate, VERVE-201, was
recently dosed. With cash runway through 2026, we are
well-positioned to execute additional important milestones across
our pipeline and advance our early-stage programs, including our
program targeting LPA. With two product candidates being tested in
the clinic, we expect 2025 to be an eventful year for Verve as we
develop a new approach for the treatment of cardiovascular
disease.”
PCSK9 ProgramEnrollment Ongoing in Heart-2
Phase 1b Clinical Trial Evaluating VERVE-102
- 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 and durably reduces
disease-driving low-density lipoprotein cholesterol (LDL-C).
VERVE-102 consists of messenger RNA expressing an adenine base
editor and an optimized guide RNA targeting
the PCSK9 gene identical to VERVE-101, the company’s
initial PCSK9 product candidate that showed proof-of concept for
this mechanism. However, compared to VERVE-101, VERVE-102 uses a
different lipid nanoparticle (LNP) delivery system, which includes
a different ionizable lipid and Verve’s proprietary GalNAc
liver-targeting ligand, designed to allow the LNP to access liver
cells using either the low-density lipoprotein receptor (LDLR) or
the asialoglycoprotein receptor (ASGPR).
- VERVE-102 is being evaluated in the Heart-2 open-label Phase 1b
clinical trial in two patient populations who require deep and
durable reductions of LDL-C levels in the blood: adults living with
heterozygous familial hypercholesterolemia (HeFH) and adults living
with premature coronary artery disease (CAD). The Heart-2 clinical
trial is expected to include four dose cohorts, each comprised of
three to nine patients with either HeFH or premature CAD.
- As of October 29, 2024, dosing has been completed in seven
participants in the first two dose cohorts, 0.3 mg/kg and 0.45
mg/kg, in the Heart-2 clinical trial. VERVE-102 has been
well-tolerated. No serious adverse events and no clinically
significant laboratory abnormalities have been observed. Following
the standard review from the independent data and safety monitoring
board (DSMB), the company expects to continue the dose escalation
portion of the clinical trial.
- Verve recently received clearance of its Clinical Trial
Applications (CTAs) for the Heart-2 clinical trial in Israel and
New Zealand. Enrollment remains ongoing in Australia, Canada, and
the U.K.
- In November 2024, Verve plans to present a moderated digital
poster at the American Heart Association (AHA) Scientific Sessions
describing the design of the Heart-2 Phase 1b clinical trial.
- Verve expects to provide initial data from the Heart-2 clinical
trial and an update on the PCSK9 program in the first half of 2025.
The company also plans to initiate the Phase 2 clinical trial for
the PCSK9 program in the second half of 2025.
Analyses of Heart-1 Phase 1b Clinical Trial of VERVE-101
- Verve recently reported updated durability data from the
Heart-1 clinical trial of VERVE-101 at the European Society of Gene
and Cell Therapy (ESGCT) 2024 Congress and the American Society of
Nephrology Kidney Week 2024 Meeting. Mean, time-averaged PCSK9
protein reductions of greater than 60% were observed in each of the
two higher dose cohorts (0.45 mg/kg and 0.6 mg/kg), and mean,
time-averaged LDL-C reductions of 42% at 0.45 mg/kg (n=6) and
time-averaged LDL-C reduction of 57% at 0.6 mg/kg (n=1) were
observed. In the single participant in the highest dose cohort,
LDL-C reduction has now been sustained out to 18 months after the
single dose. No new treatment related adverse events have been
reported since March 2024. Verve believes that these new durability
data further support the potential of once-and-done gene editing
medicines for the treatment of cardiovascular disease.
- Verve has completed a series of nonclinical studies as part of
its investigation into the previously disclosed laboratory
abnormalities observed with VERVE-101. In order to isolate the role
of the LNP and determine whether the laboratory abnormalities
observed in the Heart-1 clinical trial were due to the LNP delivery
system, these studies used a version of VERVE-101 with a
non-targeting guide RNA designed to preclude base editing. Data
from these studies continue to support Verve’s understanding that
the LNP in VERVE-101 is likely the primary driver of the laboratory
abnormalities observed in the Heart-1 clinical trial. The Heart-1
clinical trial will remain paused during the dose escalation
portion of the Heart-2 clinical trial evaluating VERVE-102.
ANGPTL3 ProgramFirst Participant Dosed with
VERVE-201 in the Pulse-1 Phase 1b Clinical Trial
- VERVE-201 is a novel, investigational gene editing medicine
designed to be a single course treatment that permanently turns off
the ANGPTL3 gene in the liver to reduce disease-driving LDL-C as
well as remnant cholesterol and utilizes Verve’s proprietary
GalNAc-LNP delivery technology. VERVE-201 is being developed in two
patient populations: patients with refractory hypercholesterolemia
(RH), defined as those who are unable to achieve adequate LDL-C
reduction with maximally tolerated standard of care therapies,
potentially including PCSK9 inhibitors, and patients living with
homozygous familial hypercholesterolemia (HoFH), a rare and often
fatal inherited cause of premature ASCVD characterized by extremely
high blood LDL-C. The aim of this medicine is to reduce the heavy
treatment burden associated with available therapies, including the
requirement for multiple oral, injectable, and intravenous
infusions, often administered over decades.
- Verve announced today that the first participant has been dosed
with VERVE-201 in its Pulse-1 Phase 1b clinical trial.The Pulse-1
clinical trial is designed to evaluate the safety and tolerability
of VERVE-201 administration in adult patients with RH who require
additional lowering of LDL-C despite treatment with maximally
tolerated standard of care therapies, potentially including PCSK9
inhibitors. Endpoints also include pharmacokinetics and changes in
blood ANGPTL3 protein and LDL-C levels. The Pulse-1 clinical trial
is a single-ascending dose study that has an adaptive design. The
Pulse-1 clinical trial is expected to include four dose cohorts
each comprised of three to nine patients with RH.
- Verve recently received regulatory clearances to initiate the
Pulse-1 clinical trial in Australia, Canada, and the U.K.
Upcoming Investor EventsVerve plans to
participate in fireside chats during the following upcoming
investor events:
- Guggenheim's Inaugural Healthcare Innovation Conference,
November 11 at 4:00 PM ET, Boston, MA
- Stifel 2024 Healthcare Conference, November 18 at 3:00 PM ET,
New York, NY
- Jefferies London Healthcare Conference, November 20 at 4:00 PM
GMT, London, UK
Upcoming Medical Meeting PresentationsVerve
plans to present a moderated digital poster at the American Heart
Association (AHA) Scientific Sessions in Chicago, IL. Details of
the poster session are as follows:
Title: Design of Heart-2: a phase 1b clinical
trial of VERVE-102, an in vivo base editing medicine delivered by a
GalNAc-LNP and targeting PCSK9 to durably lower LDL
cholesterolSession: Genomic Therapies for
Cardiovascular DiseaseDate and Time: November 16
at 10:35 AM CT
Third Quarter 2024 Financial Results
Cash Position: Verve ended the third quarter of
2024 with $539.9 million in cash, cash equivalents, and marketable
securities. Verve expects its capital position to be sufficient to
fund its operations through 2026.
Collaboration Revenue: Collaboration revenue
was $6.9 million for the third quarter of 2024, compared to $3.1
million for the third quarter of 2023. The increase was primarily
due to an increase in research services performed under the
company’s collaboration agreements.
Research & Development (R&D) Expenses:
R&D expenses were $49.9 million for the third quarter of 2024,
compared to $43.8 million for the third quarter of 2023.
Stock-based compensation expense included in R&D expenses was
$5.4 million and $4.9 million for the third quarter of 2024 and
2023, respectively.
General & Administrative (G&A)
Expenses: G&A expenses were $13.8 million for the
third quarter of 2024, compared to $11.7 million for the third
quarter of 2023. Stock-based compensation expense included in
G&A expenses was $5.4 million and $3.9 million for the third
quarter of 2024 and 2023, respectively.
Net Loss: Net loss was $50.1 million, or $0.59
basic and diluted net loss per share, for the third quarter of
2024, compared to $45.8 million, or $0.72 basic and diluted net
loss per share, for the third quarter of 2023.
About Verve Therapeutics Verve
Therapeutics, Inc. (Nasdaq: VERV) is a clinical-stage company
developing a new class of genetic medicines for cardiovascular
disease with the potential to transform 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
refractory hypercholesterolemia, where patients still have high
LDL-C despite treatment with maximally tolerated standard of care
therapies, and homozygous familial hypercholesterolemia (HoFH). 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 and Pulse-1 clinical trial; the timing and
availability of data for the Heart-2 trial, PCSK9 program and
Pulse-1 trial; expectations for the company’s Heart-1 clinical
trial; its research and development plans; the potential advantages
and therapeutic potential of the company’s programs; 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 September 30, |
|
Nine months ended September 30, |
|
| Condensed consolidated
statements of operations |
|
2024 |
|
2023 |
|
2024 |
|
2023 |
|
|
Collaboration revenue |
|
$ |
6,865 |
|
$ |
3,117 |
|
$ |
19,252 |
|
$ |
6,614 |
|
| Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
49,938 |
|
|
43,765 |
|
|
149,299 |
|
|
138,135 |
|
|
General and administrative |
|
|
13,837 |
|
|
11,686 |
|
|
42,546 |
|
|
37,655 |
|
|
Total operating expenses |
|
|
63,775 |
|
|
55,451 |
|
|
191,845 |
|
|
175,790 |
|
| Loss from operations |
|
|
(56,910 |
) |
|
(52,334 |
) |
|
(172,593 |
) |
|
(169,176 |
) |
| Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Change in fair value of success payment liability |
|
|
(6 |
) |
|
802 |
|
|
1,743 |
|
|
878 |
|
|
Interest and other income, net |
|
|
6,887 |
|
|
5,841 |
|
|
22,452 |
|
|
16,825 |
|
|
Total other income, net |
|
|
6,881 |
|
|
6,643 |
|
|
24,195 |
|
|
17,703 |
|
| Loss before provision for
income taxes |
|
|
(50,029 |
) |
|
(45,691 |
) |
|
(148,398 |
) |
|
(151,473 |
) |
| Provision for income
taxes |
|
|
(104 |
) |
|
(67 |
) |
|
(276 |
) |
|
(243 |
) |
| Net loss |
|
$ |
(50,133 |
) |
$ |
(45,758 |
) |
$ |
(148,674 |
) |
$ |
(151,716 |
) |
| Net loss per common share,
basic and diluted |
|
$ |
(0.59 |
) |
$ |
(0.72 |
) |
$ |
(1.77 |
) |
$ |
(2.43 |
) |
| Weighted-average common shares
used in net loss per share, basic and diluted |
|
|
84,632,952 |
|
|
63,211,849 |
|
|
83,999,797 |
|
|
62,322,965 |
|
|
Condensed consolidated balance sheet data |
|
September 30,2024 |
|
December 31,2023 |
|
|
Cash, cash equivalents and marketable securities |
|
$ |
539,920 |
|
$ |
623,950 |
|
|
Total assets |
|
$ |
663,906 |
|
$ |
752,688 |
|
|
Total liabilities |
|
$ |
155,355 |
|
$ |
153,186 |
|
|
Total stockholders' equity |
|
$ |
508,551 |
|
$ |
599,502 |
|
Verve Therapeutics (NASDAQ:VERV)
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Verve Therapeutics (NASDAQ:VERV)
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De Jan 2024 à Jan 2025
