Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia

VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.

Start Date05 Jul 2022

Sponsor / Collaborator

Verve Therapeutics, Inc.

100 Clinical Results associated with VERVE-101

100 Translational Medicine associated with VERVE-101

100 Patents (Medical) associated with VERVE-101

Literatures (Medical) associated with VERVE-101

02 Aug 2024·EXPERT OPINION ON INVESTIGATIONAL DRUGS

VERVE-101, a CRISPR base-editing therapy designed to permanently inactivate hepatic PCSK9 and reduce LDL-cholesterol

Author: Burnett, John R ; Hooper, Amanda J ; Tang, Xuan L

A review.A review discussing CRISPR base-editing therapy is a promising technol., including off- target effects and uncertain long-term health and safety consequences, as demonstrated with the VERVE-101.It also discusses urrent PCSK9 inhi - bitors in use, which are generally well tolerated with low rates of adverse events.

23 Feb 2024·European heart journal. Cardiovascular pharmacotherapy

VERVE-101: a promising CRISPR-based gene editing therapy that reduces LDL-C and PCSK9 levels in HeFH patients

Article

Author: Horie, Takahiro ; Ono, Koh

17 Jan 2023·Circulation

Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models

Article

Author: Mazzola, Anne Marie ; Lee, Richard G. ; Khera, Amit V. ; Braun, Maurine C. ; Bellinger, Andrew M. ; Platt, Colin ; Vafai, Scott B. ; Rohde, Ellen ; Kathiresan, Sekar

Background::

VERVE-101 is an investigational in vivo CRISPR base-editing medicine designed to alter a single DNA base in the PCSK9 gene, permanently turn off hepatic protein production, and thereby durably lower low-density lipoprotein cholesterol. We test the efficacy, durability, tolerability, and potential for germline editing of VERVE-101 in studies of nonhuman primates and a murine F1 progeny study.

Methods::

Cynomolgus monkeys were given a single intravenous infusion of a vehicle control (n=10) or VERVE-101 at a dose of 0.75 mg/kg (n=4) or 1.5 mg/kg (n=22) with subsequent follow-up up to 476 days. Two studies assessed the potential for germline editing, including sequencing sperm samples from sexually mature male nonhuman primates treated with VERVE-101 and genotyping offspring from female mice treated with the murine surrogate of VERVE-101 (VERVE-101mu).

Results::

Liver biopsies 14 days after dosing noted mean PCSK9 editing of 46% and 70% in monkeys treated with VERVE-101 at 0.75 and 1.5 mg/kg, respectively. This translated into mean reductions in blood PCSK9 (proprotein convertase subtilisin/kexin type 9) of 67% and 83% and reductions of low-density lipoprotein cholesterol of 49% and 69% at the 0.75 and 1.5 mg/kg doses, respectively, assessed as time-weighted average change from baseline between day 28 and up to 476 days after dosing. Liver safety monitoring noted a transient rise in alanine aminotransferase and aspartate aminotransferase concentrations after infusion that fully resolved by day 14 with no accompanying change in total bilirubin. In a subset of monkeys necropsied 1 year after dosing, no findings related to VERVE-101 were identified on macroscopic and histopathologic assessment of the liver and other organs. In the study to assess potential germline editing of male nonhuman primates, sperm samples collected after VERVE-101 dosing showed no evidence of PCSK9 editing. Among 436 offspring of female mice treated with a saturating dose of VERVE-101mu, the PCSK9 edit was transmitted in 0 of 436 animals.

Conclusions::

VERVE-101 was well tolerated in nonhuman primates and led to 83% lower blood PCSK9 protein and 69% lower low-density lipoprotein cholesterol with durable effects up to 476 days after dosing. These results have supported the initiation of a first-in-human clinical trial in patients with heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease.

News (Medical) associated with VERVE-101

15 Oct 2024

·www.prnewswire.com

Shareholders that lost money on Verve Therapeutics, Inc.(VERV) should contact Levi & Korsinsky about pending Class Action - VERV

NEW YORK, Oct. 15, 2024 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Verve Therapeutics, Inc. ("Verve Therapeutics" or the "Company") (NASDAQ: VERV) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Verve Therapeutics investors who were adversely affected by alleged securities fraud between August 9, 2022 and April 1, 2024. Follow the link below to get more information and be contacted by a member of our team: VERV investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (1) defendants did not fully disclose the circumstances under which the Heart-1 Phase 1b clinical trial of VERVE-101, its investigational gene editing medicine, would be halted; (2) defendants overstated the potential benefits of its proprietary lipid nanoparticle delivery system; and (3) as a result, defendants' statements about its business, operations, and prospects, were materially false and misleading and/or lacked a reasonable basis at all relevant times. WHAT'S NEXT? If you suffered a loss in Verve Therapeutics during the relevant time frame, you have until October 28, 2024 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Patent Infringement

07 Oct 2024

·www.prnewswire.com

Verve Therapeutics, Inc. Class Action: The Gross Law Firm Reminds Verve Therapeutics Investors of the Pending Class Action Lawsuit with a Lead Plaintiff Deadline of October 28, 2024 - VERV

NEW YORK, Oct. 7, 2024 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Verve Therapeutics, Inc. (NASDAQ: VERV). Shareholders who purchased shares of VERV during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery. CONTACT US HERE: CLASS PERIOD: August 9, 2022 to April 1, 2024 ALLEGATIONS: The complaint alleges that during the class period, Defendants issued materially false and/or misleading statements and/or failed to disclose that: (1) defendants did not fully disclose the circumstances under which the Heart-1 Phase 1b clinical trial of VERVE-101, its investigational gene editing medicine, would be halted; (2) defendants overstated the potential benefits of its proprietary lipid nanoparticle delivery system; and (3) as a result, defendants' statements about its business, operations, and prospects, were materially false and misleading and/or lacked a reasonable basis at all relevant times. DEADLINE: October 28, 2024 Shareholders should not delay in registering for this class action. Register your information here: NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of VERV during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is October 28, 2024. There is no cost or obligation to you to participate in this case. WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes. CONTACT: The Gross Law Firm 15 West 38th Street, 12th floor New York, NY, 10018 Email: [email protected] Phone: (646) 453-8903 SOURCE The Gross Law Firm WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1

30 Sep 2024

·www.prnewswire.com

Investors in Verve Therapeutics, Inc. Should Contact The Gross Law Firm Before October 28, 2024 to Discuss Your Rights - VERV

NEW YORK, Sept. 30, 2024 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Verve Therapeutics, Inc. (NASDAQ: VERV). Shareholders who purchased shares of VERV during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery. CONTACT US HERE: CLASS PERIOD: August 9, 2022 to April 1, 2024 ALLEGATIONS: The complaint alleges that during the class period, Defendants issued materially false and/or misleading statements and/or failed to disclose that: (1) defendants did not fully disclose the circumstances under which the Heart-1 Phase 1b clinical trial of VERVE-101, its investigational gene editing medicine, would be halted; (2) defendants overstated the potential benefits of its proprietary lipid nanoparticle delivery system; and (3) as a result, defendants' statements about its business, operations, and prospects, were materially false and misleading and/or lacked a reasonable basis at all relevant times. DEADLINE: October 28, 2024 Shareholders should not delay in registering for this class action. Register your information here: NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of VERV during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is October 28, 2024. There is no cost or obligation to you to participate in this case. WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes. CONTACT: The Gross Law Firm 15 West 38th Street, 12th floor New York, NY, 10018 Email: [email protected] Phone: (646) 453-8903 SOURCE The Gross Law Firm WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Patent Infringement

100 Deals associated with VERVE-101

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Atherosclerosis	Phase 1	US	Verve Therapeutics, Inc.	05 Jul 2022
Atherosclerosis	Phase 1	NZ	Verve Therapeutics, Inc.	05 Jul 2022
Atherosclerosis	Phase 1	GB	Verve Therapeutics, Inc.	05 Jul 2022
Heterozygous familial hypercholesterolemia	Phase 1	US	Verve Therapeutics, Inc.	05 Jul 2022
Heterozygous familial hypercholesterolemia	Phase 1	NZ	Verve Therapeutics, Inc.	05 Jul 2022
Heterozygous familial hypercholesterolemia	Phase 1	GB	Verve Therapeutics, Inc.	05 Jul 2022

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASGCT2024	Not Applicable	-	-	VERVE-101	uyzgsszpud(mmtcvbjdac) = ulzpaoelsp iyzxlpqgai (tpwiblayvj )	-	07 May 2024
NCT05398029 (heart-1, Company_Website \| Corporate Publications) Manual	Phase 1	Heterozygous familial hypercholesterolemia	10	VERVE-101 0.1 mg/kg	zlbjrnjhfw(ktywczhcff) = VERVE-101 was well-tolerated in the two lower dose cohorts, with no treatment-related adverse events observed. In the two higher dose cohorts, treatment-related adverse events were observed, including transient, mild or moderate infusion reactions and transient, asymptomatic increases in liver transaminases with mean bilirubin levels below the upper limit of normal. All infusion reactions and liver transaminase elevations resolved without clinical sequelae. yqumptolex (buwlosaarn )	Positive	12 Nov 2023
	Phase 1	Heterozygous familial hypercholesterolemia	10	VERVE-101 0.3 mg/kg		Positive	12 Nov 2023

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