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Verve Therapeutics Announces Clearance of First VERVE-101 Clinical Trial Application and Outlines Global Clinical Development Strategy; Reports First Quarter 2022 Financial Results Verve on Track to Begin Dosing HeFH Pat

Key Takeaway: Verve Therapeutics Announces Clearance of First VERVE-101 Clinical Trial Application and Outlines Global Clinical Development Strategy; Reports First Quarter 2022 Financial Results Track to Begin Dosing HeFH Patients in New Zealand in Mid-2022 in a Phase 1 Clinical Trial with I

Full Press Release Details

Verve Therapeutics Announces Clearance of First VERVE-101
Clinical Trial Application and Outlines Global Clinical Development Strategy; Reports First Quarter 2022 Financial Results
Track to Begin Dosing HeFH Patients in New Zealand in Mid-2022 in a Phase 1 Clinical Trial with Initial Clinical Data Expected in 2023
Preparing for Submission of United Kingdom and United States Regulatory Filings for VERVE-101 in
Cash, Cash Equivalents and Marketable Securities of $323.3 Million with Cash Runway into 2024
CAMBRIDGE, Mass. May 10, 2022 Verve Therapeutics, Inc., a biotechnology company pioneering a new approach to the care of
cardiovascular disease with single-course gene editing medicines, today outlined its global development strategy for its lead gene editing candidate, VERVE-101, and reported financial results for the first
quarter ended March 31, 2022. VERVE-101 is a novel gene editing medicine discovered and developed by Verve that is designed to permanently turn 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 subtype of atherosclerotic cardiovascular disease (ASCVD). The company ultimately plans to expand clinical development of VERVE-101 beyond HeFH for the treatment of patients with established ASCVD not at LDL-C goal on oral therapy.
VERVE-101 Global Clinical Development
Verve has received clearance of its first clinical trial application (CTA) for VERVE-101 in New Zealand and plans to
initiate a first-in-human Phase 1 clinical trial in patients with HeFH in mid-2022. This CTA is part of a global regulatory
strategy established by Verve for clinical development of VERVE-101, which also includes plans to submit both a CTA in the United Kingdom and an investigational new drug (IND) application in the United States
in the second half of 2022. The company anticipates initiating clinical trial sites and patient enrollment in the respective regions shortly following clearance of its applications, should they be accepted. Verve expects to report initial clinical
data from the VERVE-101 Phase 1 clinical trial in 2023.
This CTA clearance represents a tremendous
milestone in the evolution of Verve as we start our transition to a clinical-stage gene editing company. To support the global development of VERVE-101, we have worked diligently with both U.S. and
international regulatory authorities and key clinical advisors to define clear paths for our regulatory submissions, beginning first in New Zealand and then rapidly expanding to the U.K. and U.S. later this year, said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve. Our global clinical development strategy for VERVE-101 is based on extensive
non-human primate (NHP) data that demonstrate the ability of VERVE-101 to potently and durably edit PCSK9, leading to
LDL-C reductions sustained out to one year. These data form the basis for our CTAs for clinical development in New Zealand and the U.K. The remaining IND-enabling
studies are on track to be completed in the third quarter, and we are prepared to rapidly submit our IND upon completion. This is a momentous year as we continue our mission of transforming the treatment of cardiovascular disease from the current
chronic care model to that of single-course gene editing medicines.
ANGPTL3 Program Update
Verve s ANGPTL3 program is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride
metabolism, and is being developed for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic subtype of ASCVD characterized by extremely high blood LDL-C, as well as for patients
with ASCVD who have not achieved goal LDL-C lowering with oral therapy and a PCSK9 inhibitor. In recently presented data, a 96% reduction in blood ANGPTL3 protein from baseline was observed in NHPs (n=4)
treated with a Verve ANGPTL3 base editor, with follow-up out to 616 days. In addition, no long-term impacts were observed on markers of liver toxicity, as measured by alanine aminotransferase (ALT) and
bilirubin levels, following treatment administration. Verve expects to identify its base editing development candidate targeting ANGPTL3 and begin IND-enabling studies in the second half of 2022.
Beyond VERVE-101, we continue to advance our product-focused pipeline, leveraging our approach of going after
validated disease targets and determining the best technology to address them, said Andrew Bellinger, M.D., Ph.D., chief scientific officer of Verve. Recent data from our ANGPTL3 program support its continued development toward IND-enabling studies. 2022 represents a significant year of progress, and in addition to advancing our lead programs, we are also actively conducting research to identify new targets we may be able to address, as we
work toward our goal of providing better treatments for the millions of people living with cardiovascular disease around the globe.
Medical Meeting Presentations
First Quarter 2022 Financial Results
Verve Therapeutics, Inc. (Nasdaq: VERV) is a genetic medicines company pioneering a new approach to the care of cardiovascular
disease, transforming treatment from chronic management to single-course gene editing medicines. The company s initial two programs target PCSK9 and ANGPTL3, genes that have been extensively validated as targets for lowering blood lipids such
as low-density lipoprotein cholesterol (LDL-C), a root cause of cardiovascular disease. Verve s lead product candidate,
VERVE-101, is designed to permanently turn off the PCSK9 gene in the liver in order to disrupt blood PCSK9 protein production and thereby durably reduce blood
LDL-C levels, with the goal of reducing a patient s risk for cardiovascular disease. VERVE-101 is being developed initially for the treatment of patients with
heterozygous familial hypercholesterolemia, a potentially fatal genetic heart disease. For more information, please visit www.VerveTx.com.
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 initiation, and timing, of the company s regulatory submissions and future clinical trials, 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 timing of and the company s ability
to submit applications for, its product candidates; advance its product candidates in clinical trials; initiate and enroll 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 and its other product candidates; 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.
Verve Therapeutics, Inc.
Ten Bridge Communications
Verve Therapeutics, Inc.
Selected Condensed Financial Information
(in thousands, except share and per share amounts)
Condensed consolidated statements of operations Three months ended March 31,
2022 2021
Operating expenses:
Research and development $ 24,490 $ 11,345
General and administrative 7,435 2,716
Total operating expenses 31,925 14,061
Loss from operations (31,925 ) (14,061 )
Other income:
Change in fair value of antidilution rights liability 396
Change in fair value of success payment liability 1,677 382
Interest and other income, net 82 20
Total other income, net 1,759 798
Net loss $ (30,166 ) $ (13,263 )
Net loss per common share, attributable to common stockholders, basic and diluted $ (0.62 ) $ (4.99 )
Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted 48,571,214 2,656,278
Condensed consolidated balance sheet data March 31, 2022 December 31, 2021
Cash, cash equivalents and marketable securities $ 323,299 $ 360,442
Total assets 349,241 384,124
Total liabilities 17,851 26,772
Total stockholders equity 331,390 357,352
Last updated: May 10, 2022