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 fourth quarter and year ended
December 31, 2023.
“2023 was a momentous year for Verve as we made significant
strides towards our mission of protecting the world from
cardiovascular disease. Our pipeline, including clinical,
preclinical, and discovery programs, captures a robust set of
targets with compelling human genetics validation in areas of high
unmet need and significant market opportunities, including PCSK9,
ANGPTL3, and LPA. We are incredibly proud of the accomplishments
we’ve made to date, particularly with the Heart-1 clinical trial of
VERVE-101 in patients with HeFH, which demonstrated the first
proof-of-concept data for in vivo base editing in humans,” said
Sekar Kathiresan, M.D., co-founder and chief executive officer of
Verve Therapeutics. “Looking ahead in 2024, we expect to dose the
first patient in the United States, complete enrollment in the
ongoing Phase 1b trial of VERVE-101, and provide a data update. We
also expect to initiate the Heart-2 Phase 1b trial for VERVE-102
targeting PCSK9 in the first half of this year, followed by
initiation of a Phase 1b trial for VERVE-201 targeting ANGPTL3 in
the second half of the year. We are excited about the potential to
have three in vivo gene editing programs in the clinic in 2024,
supported by a substantial cash position that provides an estimated
runway into late 2026, and a collaborator with expertise in
cardiometabolic disease as well as late-stage development in Eli
Lilly.”
Interim Data from Ongoing Heart-1 Phase 1b Clinical
Trial Demonstrating First Human Proof-of-Concept for
In Vivo Base Editing
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 PCSK9 protein
and thereby durably reducing low-density lipoprotein cholesterol
(LDL-C). VERVE-101 is being developed initially for the treatment
of patients living with heterozygous familial hypercholesterolemia
(HeFH), a life-threatening inherited disease characterized by
lifelong elevations in blood LDL-C and accelerated atherosclerotic
cardiovascular disease (ASCVD).
VERVE-101 is being evaluated in the Heart-1 Phase 1b clinical
trial with endpoints of safety and tolerability as well as changes
in blood PCSK9 protein and LDL-C levels.
In November 2023, Verve presented interim data from the ongoing
Heart-1 trial of VERVE-101 in a late-breaking science presentation
at the American Heart Association (AHA) Scientific Sessions
highlighting first human proof-of-concept data for in vivo base
editing. Treatment with VERVE-101 led to dose-dependent reductions
of disease-causing LDL-C in patients living with
HeFH. Time-averaged LDL-C reductions of up to 55% and blood
PCSK9 protein reductions of up to 84% were observed after a single
infusion of VERVE-101 at potentially therapeutic doses. These
results suggest successful editing at the intended genomic target.
Dose-dependent LDL-C reductions, a validated measure of clinical
efficacy for this patient population, were observed one month after
treatment at the 0.45 and 0.6 mg/kg dose cohorts, and the reduction
was sustained out to six months for the single patient in the
highest dose cohort. A review of the safety data of VERVE-101 by an
independent data and safety monitoring board supported the
continued development of VERVE-101 in the Heart-1 trial, and the
adverse events were consistent with expectations in the severe,
advanced ASCVD patient population enrolled in the trial.
The Heart-1 trial is enrolling patients in the 0.45 and 0.6
mg/kg cohorts of the single ascending dose portion, and Verve
expects to complete enrollment of the Heart-1 clinical trial in
2024. Verve plans to provide a data update from the Heart-1
clinical trial in the second half of 2024.
IND Clearance by U.S. FDA for VERVE-101
In October 2023, Verve announced the clearance of its
Investigational New Drug (IND) Application for VERVE-101 in
patients living with HeFH by the U.S. Food and Drug Administration
(FDA). Supplementing the active trial sites in the United Kingdom
and New Zealand, Verve is working to activate U.S. trial sites and
to dose the first patient with VERVE-101 in 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 messenger RNA
expressing an adenine base editor; however, VERVE-102 is delivered
using Verve’s proprietary GalNAc-LNP delivery technology which can
access liver cells using either the asialoglycoprotein receptor
(ASGPR) or the low-density lipoprotein receptor (LDLR).
Verve plans to pursue a regulatory strategy initially outside
the U.S. for the Heart-2 Phase 1b clinical trial. Verve expects to
initiate the Heart-2 trial with VERVE-102 for patients with HeFH or
premature coronary artery disease in the first half of 2024,
subject to regulatory clearances. Following an evaluation of
clinical data from the Heart-1 and the Heart-2 trials, Verve plans
to initiate a randomized, placebo-controlled Phase 2 clinical trial
of either VERVE-101 or VERVE-102 in 2025.
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 initially for the treatment of patients living with
homozygous familial hypercholesterolemia (HoFH), a rare and often
fatal inherited subtype of premature 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 also
plans to evaluate VERVE-201 in ASCVD patients with refractory
hypercholesterolemia, who have high LDL-C despite treatment with
maximally-tolerated standard of care therapies.
Preclinical studies to support regulatory submissions for
clinical development of VERVE-201 are ongoing. Verve plans to
pursue a regulatory strategy initially outside the U.S. for the
VERVE-201 Phase 1b clinical trial. Verve expects to initiate the
VERVE-201 Phase 1b clinical trial in the second half of 2024,
subject to regulatory clearances.
Corporate Update
In December 2023, Verve announced the closing of an underwritten
public offering of 14,375,000 shares of its common stock at a
public offering price of $10.00 per share. The total net proceeds
of the public offering were approximately $134.7 million, after
deducting underwriting discounts and commissions and offering
expenses payable by Verve. Concurrently, the company announced the
closing of its private placement of 2,296,317 shares of its common
stock to Eli Lilly and Company (Lilly), at a price per share equal
to the public offering price. The total net proceeds of the private
placement were approximately $23.0 million.
In October 2023, Verve announced the expansion of its
relationship with Lilly, which was previously established in June
2023. Lilly acquired certain product rights from Beam Therapeutics
to Verve’s cardiovascular in vivo gene editing programs targeting
PCSK9 and ANGPTL3, as well as a third cardiovascular disease
target. Under the collaboration agreement, Verve retains control of
the development and commercialization of all collaboration
products.
Fourth Quarter and Full Year 2023 Financial
Results
Cash Position: Cash, cash equivalents and
marketable securities were $624.0 million as of December 31, 2023,
compared with $554.8 million as of December 31, 2022. Based on
current operating plans, Verve expects its existing cash, cash
equivalents and marketable securities will enable the company to
fund its operating expenses and capital expenditure requirements
into late 2026.
Collaboration Revenue: Collaboration revenue
was $5.1 million for the quarter ended December 31, 2023, and $11.8
million for the year ended December 31, 2023 and related to
research services performed under the collaboration agreement with
Vertex Pharmaceuticals Incorporated (Vertex) and the Lp(a)
collaboration with Lilly. Collaboration revenue was $1.0 million
for the quarter ended December 31, 2022, and $1.9 million for the
year ended December 31, 2022 and related to research services
performed under the collaboration agreement with Vertex.
R&D Expenses: Research and development
(R&D) expenses were $46.8 million for the quarter ended
December 31, 2023, and $184.9 million for the year ended December
31, 2023, compared to $37.3 million for the quarter ended December
31, 2022, and $130.1 million for the year ended December 31,
2022.
G&A Expenses: General and administrative
(G&A) expenses were $12.3 million for the quarter ended
December 31, 2023, and $49.9 million for the year ended December
31, 2023, compared to $11.4 million for the quarter ended December
31, 2022, and $37.5 million for the year ended December 31,
2022.
Net Loss: Net loss was $48.4 million, or $0.69
basic and diluted net loss per share, for the quarter ended
December 31, 2023, and $200.1 million, or $3.12 basic and diluted
net loss per share, for the year ended December 31, 2023, compared
to a net loss of $41.1 million, or $0.67 basic and diluted net loss
per share, for the quarter ended December 31, 2022, and $157.4
million, or $2.91 basic and diluted net loss per share, for the
year ended December 31, 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 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 ability
to enroll patients in its ongoing Heart-1 trial and activate
clinical trial sites, and begin dosing, in the United States; the
timing and availability of clinical data from its Heart-1 trial;
the regulatory strategy for VERVE-102 and VERVE-201; the receipt of
regulatory clearances and expected timing of initiating the
clinical trials of VERVE-102 and VERVE-201; 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 December
31, |
|
Year ended December 31, |
|
Condensed consolidated statements of
operations |
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue |
|
$ |
5,143 |
|
$ |
1,012 |
|
$ |
11,758 |
|
$ |
1,941 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
46,811 |
|
|
37,283 |
|
|
184,946 |
|
|
130,095 |
|
General and administrative |
|
|
12,281 |
|
|
11,438 |
|
|
49,936 |
|
|
37,533 |
|
Total operating expenses |
|
|
59,092 |
|
|
48,721 |
|
|
234,882 |
|
|
167,628 |
|
Loss from operations |
|
|
(53,949 |
) |
|
(47,709 |
) |
|
(223,124 |
) |
|
(165,687 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
Change in fair value of success payment liability |
|
|
(713 |
) |
|
2,177 |
|
|
165 |
|
|
1,486 |
|
Interest and other income, net |
|
|
6,341 |
|
|
4,501 |
|
|
23,166 |
|
|
6,867 |
|
Total other income, net |
|
|
5,628 |
|
|
6,678 |
|
|
23,331 |
|
|
8,353 |
|
Loss before provision for
income taxes |
|
|
(48,321 |
) |
|
(41,031 |
) |
|
(199,793 |
) |
|
(157,334 |
) |
Provision for income
taxes |
|
|
(32 |
) |
|
(53 |
) |
|
(275 |
) |
|
(53 |
) |
Net loss |
|
$ |
(48,353 |
) |
$ |
(41,084 |
) |
$ |
(200,068 |
) |
$ |
(157,387 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.69 |
) |
$ |
(0.67 |
) |
$ |
(3.12 |
) |
$ |
(2.91 |
) |
Weighted-average common shares
used in net loss per share, basic and diluted |
|
|
69,671,255 |
|
|
61,464,731 |
|
|
64,175,137 |
|
|
54,023,653 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Condensed consolidated balance sheet data |
|
December 31,2023 |
|
|
December 31,2022 |
|
Cash, cash equivalents and marketable securities |
|
$ |
623,950 |
|
|
$ |
554,808 |
|
Total assets |
|
$ |
752,688 |
|
|
$ |
679,223 |
|
Total liabilities |
|
$ |
153,186 |
|
|
$ |
128,291 |
|
Total stockholders' equity |
|
$ |
599,502 |
|
|
$ |
550,932 |
|
|
|
|
|
|
|
|
Verve Therapeutics (NASDAQ:VERV)
Gráfica de Acción Histórica
De Abr 2024 a May 2024
Verve Therapeutics (NASDAQ:VERV)
Gráfica de Acción Histórica
De May 2023 a May 2024