Summary of Verve Therapeutics (VERV) 2025 Conference Call Company Overview - **Company**: Verve Therapeutics - **Industry**: Biotechnology, specifically focusing on gene editing therapies for cholesterol reduction Key Points and Arguments Progress and Pipeline - Verve is developing one-time therapies aimed at lifelong cholesterol reduction through IV infusion targeting the PCSK9 gene [3][4] - Initial Phase 1 data shows well-tolerated safety and significant efficacy with LDL reductions of 50-60%, with one patient achieving a 69% reduction [4] - The company is also working on additional programs targeting ANGPTL3 and LPA, with updates expected in the second half of the year [5][6] Regulatory Environment - The new leadership at the FDA, particularly Dr. Prasad, is seen as an opportunity for Verve, aligning with their focus on meaningful clinical endpoints like LDL levels [10][11] - Verve's PCSK9 therapy is positioned to provide substantial value, potentially reducing coronary disease risk by 80-90% [12] Clinical Development - Transition from first-generation (Vrb 101) to second-generation (Vrb 102) therapies has improved safety profiles due to advancements in lipid nanoparticle delivery systems [15][16] - Ongoing dose escalation studies are expected to yield further efficacy data, with hopes of achieving higher LDL reduction percentages [19] Partnership with Eli Lilly - Verve has a partnership with Eli Lilly, which includes an opt-in decision expected in the second half of the year, where Lilly would cover a third of worldwide development costs and 50% of US commercialization expenses [21][22] - The partnership is focused on a multimodal pipeline for cardiovascular disease targets, including LPA and ANGPTL3 [23] Market Landscape and Competition - Despite the presence of oral medications for cholesterol reduction, Verve's one-time therapy addresses a significant unmet need, as many patients do not achieve treatment goals with existing options [29][30] - Patient and physician preferences indicate a strong demand for one-time therapies over daily pills [30][31] ANGPTL3 Target - The ANGPTL3 target is supported by strong genetic evidence and pharmacologic validation, with the potential to lower both LDL cholesterol and triglycerides [34] - Previous safety concerns with other ANGPTL3 therapies are attributed to specific drug-related issues rather than the target itself [36] Future Directions - Verve is optimistic about the upcoming data updates for both PCSK9 and ANGPTL3 programs, with plans to leverage learnings from previous studies to enhance study designs [40] - The company is also exploring monetization opportunities for its proprietary GalNAc LNP delivery technology, which has shown promising safety profiles [49][51] Competitive Landscape - The emergence of competitors like CRISPR in the gene editing space is acknowledged, with Verve focusing on differentiating its approach through established safety and efficacy data [38][53] Additional Important Insights - The ongoing trials and partnerships are critical for Verve's strategy to establish itself in the competitive landscape of cholesterol-lowering therapies [47][48] - The company remains confident in the potential of its therapies to address significant cardiovascular health challenges, particularly in light of the limitations of current treatment options [45][46]

Group 1: Earnings Performance - Verve Therapeutics reported a quarterly loss of $0.35 per share, significantly better than the Zacks Consensus Estimate of a loss of $0.71, representing an earnings surprise of 50.70% [1] - The company has surpassed consensus EPS estimates in all four of the last quarters [2] - The revenue for the quarter ended March 2025 was $32.98 million, exceeding the Zacks Consensus Estimate by 362.76%, compared to $5.7 million in the same quarter last year [2] Group 2: Stock Performance and Outlook - Verve Therapeutics shares have declined approximately 26.2% since the beginning of the year, while the S&P 500 has gained 0.1% [3] - The company's earnings outlook is mixed, with the current consensus EPS estimate for the upcoming quarter at -$0.70 on revenues of $8.29 million, and for the current fiscal year at -$2.73 on revenues of $29.74 million [7] - The Zacks Rank for Verve Therapeutics is currently 3 (Hold), indicating expected performance in line with the market in the near future [6] Group 3: Industry Context - The Medical - Drugs industry, to which Verve Therapeutics belongs, is currently in the top 29% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Another company in the same industry, Plus Therapeutics, is expected to report a quarterly loss of $0.17 per share, reflecting a year-over-year change of +77.3% [9]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____________ to ____________ Commission File Number: 001-40489 VERVE THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 82-48001 ...

[Company Highlights and Overview](index=1&type=section&id=Company%20Highlights) Verve Therapeutics reported strong Q1 2025 progress, highlighted by positive preliminary Heart-2 Phase 1b clinical trial data for VERVE-102, showing significant reductions in LDL-C and PCSK9 levels, maintaining approximately **$500 million** in cash, cash equivalents, and marketable securities, supporting operations until mid-2027, with further clinical and regulatory milestones anticipated in H2 2025 - Strong start to 2025 with clinical and regulatory advancements; preliminary Heart-2 clinical trial data showed single-infusion VERVE-102, a PCSK9-targeting base editor, was well-tolerated and led to dose-dependent reductions in LDL-C[2](index=2&type=chunk) - Cash runway extends to mid-2027, positioning the company to achieve its goals; dosing of the first patient in the Phase 2 clinical trial for VERVE-102 is expected in the second half of 2025[2](index=2&type=chunk) - Preliminary Heart-2 Phase 1b clinical trial data for VERVE-102 showed an average **53% reduction** in LDL-C and a maximum **69% reduction** in the 0.6 mg/kg dose cohort, with dose-dependent reductions in blood LDL-C and PCSK9[1](index=1&type=chunk) [Pipeline Updates](index=1&type=section&id=Pipeline%20Updates) Verve Therapeutics provided updates on its three gene-editing programs—PCSK9 (VERVE-102), ANGPTL3 (VERVE-201), and LPA (VERVE-301)—all aimed at treating cardiovascular diseases, with encouraging preliminary clinical data for PCSK9 and ongoing progress for ANGPTL3 and LPA [PCSK9 Program (VERVE-102)](index=1&type=section&id=PCSK9%20Program) The PCSK9 program's lead asset, VERVE-102, a base editor designed for a single-treatment, permanent PCSK9 gene inactivation, showed favorable safety and significant LDL-C reduction in preliminary Heart-2 Phase 1b clinical trial data, achieving key regulatory milestones and setting the stage for Phase 2 [Heart-2 Phase 1b Clinical Trial Initial Data](index=1&type=section&id=Heart-2%20Phase%201b%20Clinical%20Trial%20Initial%20Data) Preliminary Heart-2 Phase 1b clinical trial data for VERVE-102 demonstrated a favorable safety profile with no treatment-related serious adverse events, and in the 0.6 mg/kg dose cohort, an average **53%** LDL-C reduction and **60%** PCSK9 protein reduction, with one participant achieving a maximum **69%** LDL-C reduction - VERVE-102 demonstrated a favorable safety profile with no treatment-related serious adverse events, clinically significant laboratory abnormalities, or cardiovascular events observed[3](index=3&type=chunk)[4](index=4&type=chunk) - In four participants in the 0.6 mg/kg dose cohort, blood LDL-C was reduced by an average of **53%** and PCSK9 protein by an average of **60%**, with dose-dependent reductions; one participant achieved a maximum blood LDL-C reduction of **69%**[7](index=7&type=chunk) - In three participants who received a total RNA dose between 50 and 60 mg, VERVE-102 demonstrated an average blood LDL-C reduction of **59%** and an average PCSK9 protein reduction of **65%**[7](index=7&type=chunk) [Regulatory Milestones & Future Plans (PCSK9)](index=3&type=section&id=Regulatory%20Milestones%20%26%20Future%20Plans%20(PCSK9)) VERVE-102 received U.S. FDA Investigational New Drug (IND) application clearance and Fast Track designation for hypercholesterolemia and high lifetime cardiovascular risk, with final Heart-2 dose-escalation data, Eli Lilly opt-in decision, and Phase 2 trial initiation anticipated in H2 2025 - In March 2025, Verve announced the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for VERVE-102[7](index=7&type=chunk) - In April 2025, Verve announced VERVE-102 was granted FDA Fast Track designation for the treatment of patients with hypercholesterolemia and high lifetime cardiovascular risk to reduce LDL-C[7](index=7&type=chunk) - Verve expects to report final data from the Heart-2 clinical trial dose-escalation portion, submit the PCSK9 program opt-in package to Eli Lilly and Company, and receive Lilly’s decision in the second half of 2025; Verve plans to dose the first patient in the Phase 2 clinical trial for VERVE-102 in the second half of 2025, subject to regulatory clearance[7](index=7&type=chunk) [ANGPTL3 Program (VERVE-201)](index=3&type=section&id=ANGPTL3%20Program) The ANGPTL3 program's VERVE-201, a base editor designed to permanently inactivate the ANGPTL3 gene, is currently being evaluated in the Pulse-1 Phase 1b open-label clinical trial for refractory hypercholesterolemia, with an update expected in H2 2025 - VERVE-201 is a novel in vivo base editor designed to permanently turn off the ANGPTL3 gene in the liver with a single treatment to lower atherogenic LDL-C and triglycerides[7](index=7&type=chunk) - VERVE-201 is currently being evaluated in the ongoing Pulse-1 Phase 1b open-label clinical trial, which is designed to assess the safety and tolerability of VERVE-201 in adults with refractory hypercholesterolemia[7](index=7&type=chunk) - Verve remains on track to provide an update on the VERVE-201 program in the second half of 2025[7](index=7&type=chunk) [LPA Program (VERVE-301)](index=3&type=section&id=LPA%20Program) The LPA program is developing VERVE-301 to permanently inactivate the LPA gene and reduce Lp(a) levels, advancing preclinical studies through an exclusive research collaboration with Eli Lilly, which included a milestone payment to Verve in Q1 2025 for VERVE-301's nomination as a development candidate - VERVE-301 is a novel in vivo gene editing medicine designed to permanently turn off the LPA gene in the liver with a single treatment to lower blood lipoprotein(a) [Lp(a)] levels[6](index=6&type=chunk)[8](index=8&type=chunk) - Verve has an exclusive research collaboration with Eli Lilly and Company to advance this program, aimed at lowering Lp(a) for the treatment of atherosclerotic cardiovascular disease (ASCVD)[8](index=8&type=chunk) - Verve received a milestone payment from Eli Lilly and Company in the first quarter of 2025 in recognition of VERVE-301 being nominated as a development candidate; preclinical studies for VERVE-301 are ongoing[17](index=17&type=chunk) [First Quarter 2025 Financial Results](index=4&type=section&id=First%20Quarter%202025%20Financial%20Results) Verve Therapeutics reported a Q1 2025 net loss of **$31.0 million**, an improvement from **$48.7 million** in Q1 2024, primarily driven by significant growth in collaboration revenue, while maintaining a strong cash position of **$497.1 million**, projected to support operations until mid-2027 [Cash Position](index=4&type=section&id=Cash%20Position) As of March 31, 2025, Verve's cash, cash equivalents, and marketable securities totaled **$497.1 million**, a decrease from **$524.3 million** on December 31, 2024, but still projected to fund operations until mid-2027 Cash, Cash Equivalents, and Marketable Securities (Millions of US Dollars) | Metric | March 31, 2025 | December 31, 2024 | | :----- | :------------------ | :------------------- | | Cash, cash equivalents, and marketable securities | $497.1 | $524.3 | - Verve expects its capital position to support its operations until mid-2027[11](index=11&type=chunk) [Collaboration Revenue](index=4&type=section&id=Collaboration%20Revenue) Q1 2025 collaboration revenue significantly increased to **$33.0 million** from **$5.7 million** in Q1 2024, primarily due to the recognition of remaining deferred revenue following the termination of the Vertex Pharmaceuticals collaboration and increased recognition from research services with Eli Lilly Collaboration Revenue (Millions of US Dollars) | Metric | Q1 2025 | Q1 2024 | Year-over-Year Change | | :----- | :------------- | :------------- | :------- | | Collaboration Revenue | $33.0 | $5.7 | +$27.3 (+478.9%) | - Revenue growth was primarily attributed to the recognition of remaining deferred revenue following the termination of the Vertex Pharmaceuticals collaboration and increased recognition from research services provided under the collaboration with Eli Lilly and Company[12](index=12&type=chunk) [Operating Expenses](index=4&type=section&id=Operating%20Expenses) Total operating expenses for Q1 2025 increased to **$69.6 million** from **$62.5 million** in Q1 2024, mainly driven by higher research and development and general and administrative expenses [Research and Development Expenses](index=4&type=section&id=R%26D%20Expenses) Q1 2025 R&D expenses increased to **$54.5 million** from **$48.4 million** in Q1 2024, with stock-based compensation remaining consistent at **$5.6 million** for both quarters Research and Development Expenses (Millions of US Dollars) | Metric | Q1 2025 | Q1 2024 | Year-over-Year Change | | :----- | :------------- | :------------- | :------- | | Research and Development Expenses | $54.5 | $48.4 | +$6.1 (+12.6%) | | Stock-based compensation expense (included in R&D expenses) | $5.6 | $5.6 | 0% | [General and Administrative Expenses](index=4&type=section&id=G%26A%20Expenses) Q1 2025 general and administrative expenses rose to **$15.2 million** from **$14.2 million** in Q1 2024, also reflecting an increase in stock-based compensation expense General and Administrative Expenses (Millions of US Dollars) | Metric | Q1 2025 | Q1 2024 | Year-over-Year Change | | :----- | :------------- | :------------- | :------- | | General and Administrative Expenses | $15.2 | $14.2 | +$1.0 (+7.0%) | | Stock-based compensation expense (included in G&A expenses) | $5.6 | $4.7 | +$0.9 (+19.1%) | [Net Loss](index=4&type=section&id=Net%20Loss) Q1 2025 net loss narrowed to **$31.0 million**, or **$0.35** per basic and diluted share, an improved financial performance compared to a net loss of **$48.7 million**, or **$0.59** per basic and diluted share, in Q1 2024 Net Loss (Millions of US Dollars) | Metric | Q1 2025 | Q1 2024 | Year-over-Year Change | | :----- | :------------- | :------------- | :------- | | Net Loss | $(31.0) | $(48.7) | +$17.7 (Improvement) | | Net loss per share, basic and diluted | $(0.35) | $(0.59) | +$0.24 (Improvement) | [Company Information](index=4&type=section&id=Company%20Information) This section outlines Verve Therapeutics' mission, its focus on developing gene medicines for cardiovascular diseases, details its lead programs and their targets, and provides information on upcoming corporate communication events [About Verve Therapeutics](index=4&type=section&id=About%20Verve%20Therapeutics) Verve Therapeutics is a clinical-stage company developing novel gene medicines to transform cardiovascular disease treatment from chronic care to single-course gene editing, with lead programs VERVE-102, VERVE-201, and VERVE-301 targeting LDL-C, triglycerides, and Lp(a) - Verve Therapeutics, Inc. is a clinical-stage company dedicated to developing a new class of gene medicines designed to transform the treatment of cardiovascular disease from chronic care to a single-course gene editing medicine[16](index=16&type=chunk) - The company’s lead programs—VERVE-102, VERVE-201, and VERVE-301—target three lipoprotein drivers of atherosclerosis: LDL-C, triglycerides, and Lp(a)[18](index=18&type=chunk) - VERVE-102 is designed to permanently turn off the PCSK9 gene in the liver; VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver; and VERVE-301 is designed to permanently turn off the LPA gene to lower Lp(a) levels[18](index=18&type=chunk) [Corporate Communications](index=4&type=section&id=Corporate%20Communications) Verve Therapeutics has scheduled participation in upcoming investor conferences and has presented or will present nonclinical data and gene editing advancements at medical meetings [Upcoming Investor Events](index=4&type=section&id=Upcoming%20Investor%20Events) Verve is scheduled to participate in a fireside chat at the RBC Capital Markets Global Healthcare Conference in New York on May 21 - RBC Capital Markets Global Healthcare Conference, May 21 at 3:35 p.m. EDT, New York[9](index=9&type=chunk) [Recent and Upcoming Medical Meeting Presentations](index=4&type=section&id=Recent%20and%20Upcoming%20Medical%20Meeting%20Presentations) Verve presented nonclinical data for VERVE-102 at the 93rd Congress of the European Atherosclerosis Society on May 5 and will present advancements in single-course gene editing for durable cholesterol reduction at TIDES USA 2025 on May 22 - 93rd Congress of the European Atherosclerosis Society: Presentation titled “Nonclinical data demonstrate effective and precise in vivo inactivation of hepatic PCSK9 by clinical-stage GalNAc base editor VERVE-102.” Date: May 5 at 4:55 p.m. BST[10](index=10&type=chunk) - TIDES USA 2025: Oligonucleotide and Peptide Therapeutics: Presentation titled “Single-course gene editing for durable reduction of harmful cholesterol.” Date: May 22 at 4:30 p.m. PDT[10](index=10&type=chunk) [Legal & Contact Information](index=6&type=section&id=Legal%20%26%20Contact%20Information) This section includes standard cautionary notes regarding forward-looking statements, clarifying inherent risks and uncertainties, and provides contact information for investor and media inquiries [Cautionary Note Regarding Forward-Looking Statements](index=6&type=section&id=Cautionary%20Note%20Regarding%20Forward%20Looking%20Statements) This press release contains "forward-looking statements" subject to substantial risks and uncertainties, including those related to clinical trial timelines, regulatory approvals, intellectual property, and capital needs, which could cause actual results to differ materially from expectations - 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 company’s ongoing Heart-2 clinical trial and Pulse-1 clinical trial; the timing and availability of data from the Heart-2 trial and the initiation of the Phase 2 clinical trial for VERVE-102; the timing of the submission of the PCSK9 program opt-in package and Eli Lilly’s decision; the timing of an update on the ANGPTL3 program; the company’s research and development programs; the potential benefits and therapeutic potential of the company’s programs; and the period for which the company believes its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses[19](index=19&type=chunk) - 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[19](index=19&type=chunk) - These risks and uncertainties include, but are not limited to, risks related to the company’s limited operating history; the company’s ability to timely submit and receive approvals for its product candidates from regulatory authorities; the company’s ability to advance its product candidates in clinical trials; the company’s ability to initiate, enroll and complete its ongoing and future clinical trials on the timelines it expects, or at all; the company’s ability to accurately estimate the potential patient population and/or market for its product candidates; the ability of positive results in the preclinical studies and/or early clinical trials of VERVE-101, VERVE-102 and VERVE-201 to be replicated in clinical trials; the company’s ability to advance the development of its product candidates in current and future clinical trials as anticipated; the company’s ability to obtain, maintain and protect its intellectual property; the company’s ability to manage expenses; and the company’s ability to raise the substantial additional capital needed to achieve its business objectives[19](index=19&type=chunk) [Investor and Media Contact](index=6&type=section&id=Investor%20and%20Media%20Contact) Contact information is provided for investor relations and media inquiries - Investor Contact: Jen Robinson, Verve Therapeutics, Inc., jrobinson@vervetx.com[20](index=20&type=chunk) - Media Contact: Ashlea Kosikowski, 1AB, ashlea@1abmedia.com[20](index=20&type=chunk) [Condensed Consolidated Financial Information](index=7&type=section&id=Condensed%20Consolidated%20Financial%20Information) This section presents unaudited condensed consolidated financial statements, including statements of operations for the three months ended March 31, 2025 and 2024, and consolidated balance sheet data as of March 31, 2025, and December 31, 2024 Condensed Consolidated Statements of Operations (Thousands of US Dollars) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Collaboration revenue | $32,976 | $5,695 | | Operating expenses: | | | | Research and development expenses | $54,458 | $48,376 | | General and administrative expenses | $15,154 | $14,163 | | Total operating expenses | $69,612 | $62,539 | | Operating loss | $(36,636) | $(56,844) | | Other income: | | | | Change in fair value of success payment liability | $223 | $78 | | Interest and other income, net | $5,459 | $8,136 | | Total other income, net | $5,682 | $8,214 | | Loss before income taxes | $(30,954) | $(48,630) | | Provision for income taxes | $(79) | $(106) | | Net loss | $(31,033) | $(48,736) | | Net loss per common share, basic and diluted | $(0.35) | $(0.59) | | Weighted-average common shares used in computing net loss per share, basic and diluted | 88,795,827 | 83,132,960 | Condensed Consolidated Balance Sheet Data (Thousands of US Dollars) | Metric | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Cash, cash equivalents and marketable securities | $497,077 | $524,281 | | Total assets | $614,163 | $647,392 | | Total liabilities | $140,650 | $153,992 | | Total stockholders’ equity | $473,513 | $493,400 |

Core Insights - Verve Therapeutics reported positive initial data from the Heart-2 Phase 1b clinical trial of VERVE-102, showing a mean reduction in LDL-C of 53% and a maximum reduction of 69% after a single infusion [1][2][3] - The company has a cash position of approximately $500 million, providing a runway into mid-2027 [1][2][8] - The Pulse-1 Phase 1b clinical trial of VERVE-201 targeting ANGPTL3 is ongoing, with updates expected in the second half of 2025 [1][4][2] Clinical Trial Updates - VERVE-102 is designed to permanently turn off the PCSK9 gene in the liver, leading to durable reductions in LDL-C levels [3] - Initial data from the Heart-2 trial included 14 participants, with a favorable safety profile and no serious adverse events reported [3] - The trial demonstrated dose-dependent decreases in LDL-C and PCSK9 protein levels, with a mean reduction of 60% in PCSK9 protein among participants in the 0.6 mg/kg cohort [3] Financial Performance - Collaboration revenue for the first quarter of 2025 was $33 million, a significant increase from $5.7 million in the same quarter of 2024, primarily due to the termination of a research collaboration with Vertex Pharmaceuticals [9] - Research and development expenses were $54.5 million for Q1 2025, compared to $48.4 million in Q1 2024 [10] - The net loss for the first quarter of 2025 was $31 million, or $0.35 per share, an improvement from a net loss of $48.7 million, or $0.59 per share, in Q1 2024 [12][18] Upcoming Developments - The company plans to dose the first patient in the Phase 2 clinical trial of VERVE-102 in the second half of 2025 [2][4] - Further updates on the Heart-2 trial and the VERVE-201 program are anticipated in the second half of 2025 [2][4] - Verve is also advancing the VERVE-301 program, which targets Lp(a) levels, with preclinical studies ongoing [5][13]

Company Overview - Verve Therapeutics is a clinical-stage biotech company focused on developing gene editing therapies, particularly VERVE-102, aimed at treating heterozygous familial hypercholesterolemia (HeFH) and premature coronary artery disease [2][4] - The company has a partnership with Eli Lilly, which provides financial backing and reduces the risk of funding issues [5] Clinical Progress - Verve Therapeutics recently announced positive results from a Phase 1b clinical trial for VERVE-102, showing a mean LDL-C reduction of 53% in patients after a single injection [3][4] - The potential target market for VERVE-102 includes approximately three million patients in the U.S. and the European Union, and 31 million globally [3] Market Expectations - Wall Street has set an average price target of $24.43 for Verve Therapeutics, suggesting a potential stock increase of 321% over the next 12 months [1] - Despite the promising clinical results, the stock only saw a 24% increase in one day following the announcement, indicating cautious market sentiment [4] Future Outlook - The company plans to start Phase 2 studies in the second half of the year, but it may take a couple of years before VERVE-102 enters Phase 3 studies [5][6] - There are concerns about potential clinical and regulatory setbacks that could impact the company's progress and stock performance [4][6]

Core Viewpoint - Verve Therapeutics experienced a significant stock price increase of 40% due to positive laboratory news and favorable analyst reports [1]. Company Developments - Verve is a clinical-stage biotech company focused on its drug pipeline, with recent encouraging results from a phase 1b clinical trial of VERVE-102, aimed at treating heterozygous familial hypercholesterolemia (HeFH) [2]. - The trial involved 14 patients and demonstrated efficacy in reducing low-density lipoprotein cholesterol (LDL-C) levels, with no serious adverse events reported, indicating a well-tolerated treatment [3]. Analyst Reactions - Following the trial results, analysts issued bullish notes on Verve, with several raising their price targets; Cantor Fitzgerald upgraded its recommendation to a buy [3]. - Despite the overall positive sentiment, JPMorgan Chase analyst Eric Joseph lowered his price target from $19 to $16 per share [4]. - BMO Capital's Kostas Biliouris reiterated an outperform (buy) recommendation, noting that the drug's performance exceeded expectations and its safety profile suggested a wide therapeutic window [4].

Core Viewpoint - Verve Therapeutics' shares increased by 26.4% following the announcement of positive initial data from the early-stage study of VERVE-102, a candidate aimed at reducing cholesterol levels [1] Group 1: Study Details - The phase Ib Heart-2 study includes patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease, both of which require significant reductions in low-density lipoprotein cholesterol (LDL-C) levels [2] - The data readout involved 14 patients from the first three cohorts who received doses of VERVE-102 at 0.3 mg/kg, 0.45 mg/kg, or 0.6 mg/kg, with each participant having at least 28 days of follow-up as of March 13, 2025 [3] Group 2: Efficacy Results - A single infusion of VERVE-102 led to dose-dependent reductions in blood PCSK9 protein levels and LDL-C, with an average LDL-C decrease of 53% and a maximum reduction of 69% in the 0.6 mg/kg cohort [4] - In cohort 1, patients receiving 0.3 mg/kg showed a 21% reduction in LDL-C and a 46% reduction in PCSK9; those receiving 0.45 mg/kg had a 41% reduction in LDL-C and a 53% reduction in PCSK9; and in cohort 3, the reductions were 53% in LDL-C and 60% in PCSK9 [5][7] Group 3: Safety and Next Steps - VERVE-102 was well-tolerated across all doses, with no serious adverse events reported and no significant changes in liver enzymes or other clinical parameters among participants [7] - The Heart-2 study is currently enrolling participants for a fourth dose cohort of 0.7 mg/kg, with early safety findings consistent with previous cohorts; final data from the dose escalation phase is expected in the second half of 2025 [8] - A phase II clinical study for VERVE-102 is planned to begin in the second half of 2025, following FDA clearance of its investigational new drug application [9] Group 4: Collaboration and Future Plans - Verve Therapeutics has a collaboration agreement with Eli Lilly for the PCSK9 program, where Lilly can opt in to share development costs and profits; Verve retains control over development and commercialization in the U.S. [11] - The company plans to submit the opt-in package to Eli Lilly and expects a decision in the second half of 2025 [11]

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 ...