Shares of Verve Therapeutics (VERV) surged 26.4% on Monday and continue to soar today after the company announced positive initial data from an early-stage study of its investigational candidate, VERVE-102, which is being developed for reducing cholesterol levels. VERVE-102 is a novel, in vivo, investigational base editing medicine.The patient population enrolled in the phase Ib Heart-2 study comprises individuals with heterozygous familial hypercholesterolemia and/or premature coronary artery disease — two ...

Verve Therapeutics (VERV 23.79%) stock is seeing explosive gains Tuesday. The biotech company's share price was up 24.5% as of 12:30 p.m. ET and had been up as much as 41% earlier in the session.Yesterday, Verve published promising data from its Heart-2 Phase 1b clinical trial of its VERVE-102 treatment for heterozygous familial hypercholesterolemia (HeFH) and premature coronary artery disease. Several analysts have now raised their price targets on the stock, and investors are buying up shares in response. ...

Verve Therapeutics Inc VERV on Monday released initial data from the Heart-2 Phase 1b trial of VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD), which requires reducing low-density lipoprotein cholesterol (LDL-C) levels in the blood.VERVE-102 was well-tolerated among 14 participants across three dose levels. No treatment-related serious adverse events (SAEs) and no clinically significant laboratory abnormalities were observed.A singl ...

Single infusion of VERVE-102 led to dose-dependent decreases in blood PCSK9 and LDL-C, with mean reduction in LDL-C of 53% and a maximum reduction of 69% observed in the 0.6 mg/kg dose cohort VERVE-102 was well-tolerated with no treatment-related serious adverse events and no clinically significant changes in ALT or platelets observed at any dose level among 14 participants VERVE-102 utilizes a proprietary GalNAc-LNP which has demonstrated a potentially best-in-class safety profile In the second half of 202 ...

Core Insights - Verve Therapeutics has received Fast Track designation from the FDA for its investigational drug VERVE-102, aimed at treating hyperlipidemia and reducing LDL-C levels in patients at high cardiovascular risk [1][2] - VERVE-102 is designed as a single-course treatment that permanently disables the PCSK9 gene in the liver, potentially transforming the management of atherosclerotic cardiovascular disease [1][5] - The company is currently conducting the Phase 1b Heart-2 clinical trial to assess the safety and tolerability of VERVE-102 in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease [1][3] Company Overview - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular diseases, with a goal to shift treatment from chronic therapies to single-course gene editing solutions [5] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, targeting key cholesterol drivers associated with atherosclerosis [5] - VERVE-201 aims to turn off the ANGPTL3 gene for patients with refractory hypercholesterolemia, while VERVE-301 targets the LPA gene to reduce Lp(a) levels, an independent risk factor for cardiovascular diseases [5] Clinical Trial Updates - Initial demographic and safety data from the Heart-2 clinical trial are expected to be announced in Q2 2025, including results from three dose cohorts [3] - The final data for the dose escalation portion of the Heart-2 trial is anticipated in the second half of 2025, along with the initiation of the Phase 2 clinical trial for the PCSK9 program [4]

Core Insights - Verve Therapeutics has received FDA clearance for its Investigational New Drug (IND) application for VERVE-102, aimed at treating patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) [1][2] - VERVE-102 is designed as a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower low-density lipoprotein cholesterol (LDL-C) levels [1][2] - The company aims to provide lifelong cholesterol lowering with its gene editing medicines, addressing the limitations of existing cholesterol-lowering therapies [2] Clinical Trial Progress - The ongoing Heart-2 Phase 1b clinical trial for VERVE-102 is evaluating safety and tolerability, with interim clinical data submitted to the FDA as of January 10, 2025 [2][3] - Initial safety and efficacy data from the Heart-2 trial is expected to be announced in the second quarter of 2025, with participants having at least 28 days of follow-up [3] - The final data for the dose escalation portion of the Heart-2 trial is anticipated in the second half of 2025, along with the initiation of the Phase 2 clinical trial for the PCSK9 program [4] Company Overview - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular disease, transitioning treatment from chronic therapies to single-course gene editing [5] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, targeting key cholesterol drivers of atherosclerosis [5] - VERVE-201 aims to turn off the ANGPTL3 gene for refractory hypercholesterolemia, while VERVE-301 targets the LPA gene to reduce Lp(a) levels, an independent risk factor for cardiovascular diseases [5]

Core Viewpoint - Verve Therapeutics reported a quarterly loss of $0.58 per share, which was better than the Zacks Consensus Estimate of a loss of $0.72, indicating a positive earnings surprise of 19.44% [1] Financial Performance - The company achieved revenues of $13.08 million for the quarter ended December 2024, surpassing the Zacks Consensus Estimate by 90.39%, compared to $5.14 million in the same quarter last year [2] - Over the last four quarters, Verve Therapeutics has exceeded consensus EPS estimates four times and topped consensus revenue estimates three times [2] Stock Performance - Verve Therapeutics shares have increased by approximately 11.9% since the beginning of the year, outperforming the S&P 500, which gained 1.3% [3] Future Outlook - The company's earnings outlook will be crucial for determining future stock performance, with current consensus EPS estimates at -$0.71 for the coming quarter and -$2.83 for the current fiscal year [4][7] - The estimate revisions trend for Verve Therapeutics is currently mixed, resulting in a Zacks Rank 3 (Hold), suggesting the stock is expected to perform in line with the market in the near future [6] Industry Context - The Medical - Drugs industry, to which Verve Therapeutics belongs, is currently in the top 50% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

Genetic Medicines Development - The company is developing a new class of genetic medicines for cardiovascular disease (CVD), targeting three lipoprotein pathways that drive atherosclerotic cardiovascular disease (ASCVD) [19] - The company aims to disrupt the chronic care model for CVD by providing single-course gene editing treatments, potentially reducing the burden on patients and healthcare systems [37] - The company is developing a pipeline of single-course gene editing treatments targeting genes associated with ASCVD, leveraging advancements in human genetic analysis and gene editing technologies [38] - The company aims to address additional cardiovascular disease indications beyond ASCVD, expanding its pipeline of gene editing treatments [39] Clinical Trials and Programs - The lead clinical-stage programs, VERVE-102 and VERVE-101, target the PCSK9 gene, aiming to lower LDL cholesterol (LDL-C) levels durably throughout patients' lifetimes [21] - VERVE-102 is currently in the Heart-2 trial, with dosing completed or ongoing in three cohorts (0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg) [22] - The company plans to announce initial safety and efficacy data from the Heart-2 trial in Q2 2025 and expects to report final data in the second half of 2025 [22][24] - VERVE-201, targeting ANGPTL3, is designed to treat ASCVD patients with refractory hypercholesterolemia and is currently in the Pulse-1 trial [25][26] - The Heart-1 clinical trial is evaluating VERVE-101 in patients with HeFH and established ASCVD, with 13 participants dosed in New Zealand and the UK [79] Safety and Efficacy - In the Heart-2 clinical trial, dosing has been completed or is ongoing in participants across three dose cohorts: 0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg, with no treatment-related serious adverse events reported as of February 13, 2025 [77] - The company expects to report initial safety and efficacy data from the Heart-2 trial in Q2 2025, with final data for the dose escalation portion expected in the second half of 2025 [78] - The safety profile of VERVE-101 was generally well-tolerated, with no treatment-related adverse events in lower dose cohorts, while higher doses experienced mild to moderate infusion reactions [80] Gene Editing Technologies - The gene editing approach aims for a potent and durable lowering of blood lipids, potentially reducing ASCVD risk throughout a patient's lifetime [38] - The gene editing technologies include base editing and CRISPR nucleases, allowing for precise modifications at the single base pair level [42] - The company is optimizing its internal LNP discovery platform to improve delivery technology for current and future therapeutic programs [53] Manufacturing and Collaboration - The company is focused on scalable manufacturing capabilities for its gene editing treatments, utilizing Good Manufacturing Practice (GMP) vendors to produce high-quality mRNA, gRNA, and LNP [40] - The company has entered into a collaboration with Lilly, acquiring rights to jointly commercialize PCSK9 and ANGPTL3 programs in the U.S., enhancing its strategic partnerships [39] - The company currently relies on third-party contract manufacturing organizations (CMOs) and suppliers for critical starting materials and drug products, with plans to expand its network of CMOs as manufacturing scales [103] Financial and Competitive Landscape - The competitive landscape includes major pharmaceutical and biotechnology companies with significantly greater financial resources and expertise, posing challenges for the company's product candidates [108] - Key competitive factors for the company's product candidates include efficacy, safety, convenience, price, and the level of generic competition [109] - The company holds a patent estate covering various aspects of its programs, including gene editing programs for PCSK9 and ANGPTL3, with patents set to expire between 2041 and 2044 [118] Regulatory Compliance - The company must comply with extensive government regulations regarding the research, development, and marketing of pharmaceutical products, which require substantial time and financial resources [171] - The FDA must approve any product candidate before it can be marketed in the United States, requiring a series of preclinical and clinical testing steps [173] - Clinical trials must comply with GCP and undergo independent ethics committee review to ensure data integrity and participant safety [191]

Financial Performance - Cash, cash equivalents, and marketable securities totaled $524.3 million as of December 31, 2024, down from $624.0 million as of December 31, 2023, providing a cash runway into mid-2027[9] - Collaboration revenue increased to $13.1 million for Q4 2024 and $32.3 million for the full year 2024, compared to $5.1 million and $11.8 million for the same periods in 2023, respectively[10] - R&D expenses were $55.0 million for Q4 2024 and $204.3 million for the full year 2024, compared to $46.8 million and $184.9 million for the same periods in 2023[12] - General and administrative expenses were $14.1 million for Q4 2024 and $56.6 million for the full year 2024, compared to $12.3 million and $49.9 million for the same periods in 2023[13] - Total operating expenses for Q4 2024 were $69.15 million, up from $59.09 million in Q4 2023, with annual operating expenses totaling $260.99 million compared to $234.88 million in 2023[20] - The net loss for Q4 2024 was $50.0 million, or $0.58 per share, and for the full year 2024 was $198.7 million, or $2.35 per share, compared to a net loss of $48.4 million, or $0.69 per share, for Q4 2023[14] - The net loss for Q4 2024 was $50.04 million, slightly higher than the net loss of $48.35 million in Q4 2023, with an annual net loss of $198.71 million compared to $200.07 million in 2023[20] - Basic and diluted net loss per share improved to $0.58 in Q4 2024 from $0.69 in Q4 2023, with annual net loss per share at $2.35 compared to $3.12 in 2023[20] - Total assets declined to $647.39 million in Q4 2024 from $752.69 million in Q4 2023[20] - Total stockholders' equity decreased to $493.40 million in Q4 2024 from $599.50 million in Q4 2023[20] - Total liabilities remained relatively stable at $153.99 million in Q4 2024 compared to $153.19 million in Q4 2023[20] Clinical Trials and Developments - Initial data from the Heart-2 Phase 1b clinical trial of VERVE-102 targeting PCSK9 is expected in the second quarter of 2025, with final dose escalation data anticipated in the second half of 2025[1] - VERVE-102 has been well-tolerated with no treatment-related serious adverse events reported as of February 13, 2025[3] - Enrollment is ongoing in the Pulse-1 Phase 1b clinical trial of VERVE-201 targeting ANGPTL3, with initial data expected in the second half of 2025[1] - The company plans to deliver the opt-in data package for the PCSK9 program to Eli Lilly and receive a decision in the second half of 2025[6] - VERVE-301 has been nominated as the development candidate targeting the LPA gene, with a milestone payment received from Lilly[11]

Core Insights - Verve Therapeutics is making significant progress in its clinical trials for cardiovascular disease treatments, with key data expected in 2025 [1][2][3] Pipeline Updates - Initial data from the Heart-2 Phase 1b clinical trial of VERVE-102, targeting PCSK9, is anticipated in the second quarter of 2025, with final dose escalation data expected in the second half of 2025 [1][3] - VERVE-102 has shown good tolerability, with no serious adverse events or significant laboratory abnormalities reported [1][3] - Enrollment is ongoing for the Pulse-1 Phase 1b clinical trial of VERVE-201, which targets ANGPTL3 [1][4] Financial Position - As of December 31, 2024, the company reported cash, cash equivalents, and marketable securities totaling $524.3 million, providing a cash runway into mid-2027 [1][8] - Collaboration revenue for the fourth quarter of 2024 was $13.1 million, up from $5.1 million in the same quarter of 2023, indicating growth in research services [9] - Research and development expenses for the fourth quarter of 2024 were $55.0 million, compared to $46.8 million in the fourth quarter of 2023 [10] Leadership Changes - Dr. Frederick T. Fiedorek, Chief Medical Officer, will retire effective February 28, 2025, but will continue in an advisory role [2][7] Upcoming Events - Verve Therapeutics will present at the European Atherosclerosis Society Congress from May 4-7, 2025, in Glasgow, UK [5]