CAMBRIDGE, Mass., Sept. 21, 2022 (GLOBE NEWSWIRE) — Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to treating cardiovascular disease with gene-editing drugs in is completing a course, announced today the approval of its clinical trial approval application (CTA) by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for VERVE-101 as a potential treatment for patients with heterozygous familial hypercholesterolaemia (HeFH). HeFH is a common and potentially life-threatening genetic subtype of atherosclerotic cardiovascular disease (ASCVD) characterized by extremely high blood levels of the disease-causing low-density lipoprotein cholesterol (LDL-C).
This CTA in the UK is part of a global regulatory strategy established by Verve for the clinical development of VERVE-101, which also includes an approved CTA in New Zealand and an anticipated investigational drug approval (IND) regulatory approval in the United States second half of 2022. Verve is currently enrolling patients in its Herz 1 clinical trial of VERVE-101 for HeFH in New Zealand and expects to begin enrolling patients in the UK shortly.
“This CTA marks the second regulatory approval for VERVE-101 as we execute our global strategy aimed at bringing a potential, single-course gene editing treatment to patients with ASCVD around the world, beginning with HeFH,” said Andrew Bellinger, MD, Ph .D., Chief Medical and Scientific Officer of Verve. “We believe that VERVE-101 has the potential to transform the care and treatment of cardiovascular disease and we are committed to ensuring that Heart-1 is efficiently further developed to better understand its impact in humans build up. Recruitment in New Zealand is progressing well and with this latest approval we are working diligently with our investigators in the UK to begin patient enrollment and dosing as soon as possible. We continue to expect an interim analysis of the clinical data in 2023.”
The Heart-1 clinical study is designed to enroll approximately 40 adult patients with heterozygous familial hypercholesterolaemia (HeFH) with established atherosclerotic cardiovascular disease (ASCVD) and to evaluate the safety and tolerability of VERVE-101 administration with additional analyzes of pharmacokinetics and PCSK9 reduction in the blood evaluate protein and low-density lipoprotein cholesterol (LDL-C). The study will have three parts – (A) a single dose escalating dose, followed by (B) an expansion single dose cohort in which additional participants will receive the selected potentially therapeutic dose, and (C) an optional second dose cohort in which eligible participants in lower dose cohorts in Part A will have the option to receive a second treatment to achieve the selected potentially therapeutic dose. Preliminary clinical data from the Heart-1 clinical trial, including safety parameters, blood PCSK9 levels and blood LDL-C levels, are expected in 2023. Visit clinicaltrials.gov for more information.
VERVE-101 is a novel investigational gene-editing drug developed as a one-course treatment that PCSK9 Gene in the liver to lower disease-causing low-density lipoprotein cholesterol (LDL-C). VERVE-101 is initially being developed for the treatment of patients with heterozygous familial hypercholesterolemia (HeFH), a prevalent and potentially life-threatening genetic subtype of atherosclerotic cardiovascular disease (ASCVD). VERVE-101 consists of an adenine base editor messenger RNA (licensed from Beam Therapeutics Inc.) and an optimized guide RNA targeting the PCSK9 Gene packaged in a genetically engineered lipid nanoparticle. Through a single A-to-G change in the genetic DNA sequence of PCSK9, VERVE-101 aims to inactivate the target gene. inactivation of PCSK9 The gene has been shown to upregulate LDL receptor expression, leading to lower levels of LDL-C and thereby reducing the risk of ASCVD.
About Verve Therapeutics
Verve Therapeutics, Inc. (Nasdaq: VERV) is a clinical-stage genetic medicines company developing a new approach to treating cardiovascular disease, potentially moving treatment from chronic disease management to gene-editing drugs in one changed course. The Company’s first two programs, VERVE-101 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 were used to permanently lower the LDL -C level in the blood. VERVE-101 is designed to turn that off permanently PCSK9 Gene in the liver and is being developed initially for heterozygous familial hypercholesterolaemia (HeFH) and eventually for the treatment of atherosclerotic cardiovascular disease (ASCVD), which is not effective with oral therapy. VERVE-201 is designed to turn that off permanently ANGPTL3 gene in the liver and is being developed first in homozygous familial hypercholesterolemia (HoFH) and eventually in patients with ASCVD who have failed to achieve target LDL-C with oral therapy and a PCSK9 inhibitor. Visit www.VerveTx.com for more information.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve significant risks and uncertainties, including statements regarding enrollment of patients in the ongoing Heart-1 clinical trial, timing and availability of clinical trial data of the Heart-1 clinical trial and the status and timing of the Company’s regulatory filings, its research and development plans and the potential benefits and therapeutic potential of the Company’s programs, including VERVE-101. All statements contained in this press release, other than statements of historical fact, including statements about the Company’s strategy, future business, future financial condition, 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,” “goal,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All forward-looking statements are based on management’s current expectations regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among other things, risks related to the Company’s limited operating history; the Company’s timing and ability to submit applications for its product candidates; advance its product candidates into clinical trials; to initiate, enroll and complete its ongoing and future clinical trials within the expected timeline or at all; correctly assess the potential patient population and/or market for the Company’s product candidates; replicate in clinical studies positive results found in preclinical studies and/or earlier stage clinical studies of VERVE-101 and VERVE-201; advance the development of its product candidates within expected timelines in current and future clinical studies; obtain, maintain or protect intellectual property rights relating to its product candidates; manage expenses; and raise the significant additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties and other important factors that could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the Risk Factors section and Discussions of Potential Risks, Uncertainties and Other Important Factors, in the company’s recent filings with the Securities and Exchange Commission and in other filings the company may file in the future with the Securities and Exchange Commission. In addition, the forward-looking statements contained in this press release represent the Company’s views as of the date of this release and should not be construed as representing the Company’s views as of any date after the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. Although the Company may elect to update these forward-looking statements at any time in the future, the Company expressly disclaims any obligation to do so.
Verve Therapeutics, Inc.