Verve Therapeutics Announces Pipeline Progress and Reports First Quarter 2025 Financial Results

Reported positive initial data from the Heart-2 Phase 1b clinical trial of VERVE-102 demonstrating dose-dependent decreases in blood LDL-C and PCSK9

Mean reduction in LDL-C of 53%, with a maximum reduction of 69%, observed after a single infusion of VERVE-102 in the 0.6 mg/kg dose cohort in the Heart-2 clinical trial

Pulse-1 Phase 1b clinical trial of VERVE-201 targeting ANGPTL3 continues to progress with a program update expected in the second half of 2025

Cash, cash equivalents, and marketable securities of approximately $500 million; cash runway into mid-2027

BOSTON , May 14, 2025 (GLOBE NEWSWIRE) -- 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 first quarter ended March 31, 2025.

"2025 is off to a strong start with both clinical and regulatory progress. Last month, we reported exciting initial data from the Heart-2 clinical trial, which demonstrated that a single infusion of VERVE-102, a base editing medicine targeting PCSK9, was well-tolerated and led to compelling, dose-dependent reductions in LDL-C. These data, along with ongoing durability data of up to two years from our Heart-1 clinical trial, suggest a product profile that could fundamentally transform the journey for patients living with cardiovascular disease from decades of chronic care to a one dose future," said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. "As today's LDL-C lowering options lead to transient reduction, frequent discontinuation, and thereby, inadequate efficacy, we believe VERVE-102 can become a highly competitive option compared to existing therapies for cardiovascular disease."

"With cash runway into mid-2027, we are well-positioned to achieve our goals. We expect to dose the first patient in the Phase 2 clinical trial of VERVE-102 in the second half of 2025," Dr. Kathiresan continued. "In addition, we look forward to providing further updates in the second half of 2025, including final data from the dose escalation portion of the Heart-2 trial, delivery of the opt-in package, and a decision from Eli Lilly on the PCSK9 program, as well as a program update for VERVE-201 targeting ANGPTL3."

PCSK9 Program

Positive Initial Data from Ongoing Heart-2 Phase 1b Clinical Trial of VERVE-102

   -- VERVE-102 is a novel, in vivo, investigational base 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 uses Verve's 
      proprietary GalNAc lipid nanoparticle (LNP) delivery technology, which 
      has demonstrated a potentially best-in-class safety profile and is 
      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/or adults living with premature coronary 
      artery disease $(CAD.UK)$. To date, the Heart-2 clinical trial has dosed 
      patients in four weight-based dose cohorts, with each cohort expected to 
      be comprised of three to nine participants. 
 
   -- In April 2025, Verve presented initial data from the Heart-2 clinical 
      trial with a data cutoff date of March 13, 2025 from 14 participants 
      across the first three cohorts (weight-based cohorts of 0.3 mg/kg, 0.45 
      mg/kg, and 0.6 mg/kg) with at least 28 days of follow-up for each 
      participant. A single infusion of VERVE-102 demonstrated: 
 
          -- A favorable safety profile, with no treatment-related serious 
             adverse events, no clinically significant laboratory abnormalities, 
             and no cardiovascular events observed. Across all 14 participants, 
             there was one infusion related reaction (Grade 2) which involved 
             transient symptoms that resolved with acetaminophen. 
 
          -- Dose-dependent decreases in blood LDL-C and PCSK9 protein levels, 
             with mean reductions in blood LDL-C of 53% and PCSK9 protein of 
             60% in four participants in the 0.6 mg/kg dose cohort. The maximum 
             reduction in blood LDL-C was 69% in a single participant in the 
             0.6 mg/kg dose cohort. Blood LDL-C and PCSK9 protein reductions 
             are quantified as percent change from baseline using the 
             time-average from day 28 through last available follow-up. 
 
   -- Verve also evaluated the pharmacodynamic $(PD)$ data across all 14 
      participants from the Heart-2 clinical trial of VERVE-102 by total RNA 
      dose in milligrams, which is emerging as a key driver of PD for 
      LNP-delivered in vivo gene editing medicines. 
 
          -- Three of the 14 participants received a total RNA dose between 50 
             and 60 mg, with an average dose received of 55 mg. In this group, 
             VERVE-102 demonstrated time-averaged mean reductions in blood 
             LDL-C of 59% and PCSK9 protein of 65%. Each of the three 
             participants who received a dose >= 50 mg achieved a > 50% 
             time-averaged reduction of LDL-C from baseline. 
 
   -- The Heart-2 clinical trial is enrolling participants in the fourth dose 
      cohort of 0.7 mg/kg in the United Kingdom, Canada, Israel, Australia, and 
      New Zealand. 
 
   -- In March 2025, Verve announced the clearance by the U.S. Food and Drug 
      Administration (FDA) of the investigational new drug $(IND.AU)$ application 
      for VERVE-102. 
 
   -- In April 2025, Verve announced the receipt of Fast Track designation for 
      VERVE-102 for the treatment of patient groups with hyperlipidemia and 
      high lifetime cardiovascular risk to reduce LDL-C from the FDA. Fast 
      Track designation is designed to facilitate the development and expedite 
      the review of drugs that are intended to treat serious or 
      life-threatening conditions and demonstrate the potential to address an 
      unmet medical need. 
 
   -- Verve expects to announce the final data from the dose escalation portion 
      of the Heart-2 clinical trial, deliver the opt-in package for the PCSK9 
      program to Eli Lilly and Company (Lilly), and receive a decision from 
      Lilly in the second half of 2025. 
 
   -- Subject to regulatory clearance, Verve plans to dose the first patient in 
      the Phase 2 clinical trial of VERVE-102 in the second half of 2025. 

ANGPTL3 Program

Pulse-1 Phase 1b Clinical Trial for VERVE-201 Continues to Progress

   -- VERVE-201 is a novel, in vivo, investigational base 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 
      triglycerides 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 atherosclerotic cardiovascular 
      disease (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-201 is currently being evaluated in the ongoing Pulse-1 open-label 
      Phase 1b clinical trial, which is designed to evaluate the safety and 
      tolerability of VERVE-201 in adult patients with RH. Endpoints also 
      include pharmacokinetics and changes in blood ANGPTL3 protein and LDL-C 
      levels. 
 
   -- Verve remains on track to provide a program update on VERVE-201 in the 
      second half of 2025. 

LPA Program

Development for VERVE-301 Ongoing

   -- VERVE-301 is a novel, in vivo, investigational gene editing medicine 
      designed to be a single course treatment that permanently turns off 
      the LPA gene in the liver to reduce blood lipoprotein (a) [Lp(a)] levels 
      and utilizes Verve's proprietary GalNAc-LNP delivery technology and a 
      novel gene editor. Verve has an exclusive research collaboration with 
      Lilly to advance this program to lower Lp(a) for the treatment of ASCVD. 
 
   -- Lp(a) is a genetically validated, independent risk factor for ASCVD, 
      ischemic stroke, thrombosis, and aortic stenosis. This increased risk is 
      most pronounced in individuals with very high Lp(a) concentrations (e.g., 
      >= 125 nmol/L). An estimated 1.4 billion people worldwide have an Lp(a) 
      concentration above this threshold. Lp(a) concentrations are determined 
      at birth. Unfortunately, lifestyle changes - such as diet and exercise - 
      as well as currently approved lipid-lowering therapies have minimal to no 
      impact on reducing Lp(a) levels. 
 
   -- Verve received a milestone payment from Lilly in the first quarter of 
      2025 in conjunction with the nomination of VERVE-301 as the development 
      candidate. Preclinical studies for VERVE-301 continue to advance. 

Upcoming Investor Events

Verve plans to participate in a fireside chat during the following investor event:

   -- RBC Capital Markets Global Healthcare Conference, May 21 at 3:35 p.m. EDT, 
      New York, NY 

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