Verve Therapeutics(VERV) - 2025 Q1 - Quarterly Results

Company Highlights and Overview 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] - 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] - 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] Pipeline Updates 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) 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 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][4] - 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] - 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] Regulatory Milestones & Future Plans (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] - 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] - 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] ANGPTL3 Program (VERVE-201) 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] - 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] - Verve remains on track to provide an update on the VERVE-201 program in the second half of 2025[7] LPA Program (VERVE-301) 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][8] - 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] - 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] First Quarter 2025 Financial Results 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 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] Collaboration Revenue 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] Operating Expenses 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 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 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 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 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 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] - 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] - 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] Corporate Communications 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 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] Recent and Upcoming Medical Meeting Presentations 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] - 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] Legal & Contact Information 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 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] - 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] - 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] Investor and Media Contact Contact information is provided for investor relations and media inquiries - Investor Contact: Jen Robinson, Verve Therapeutics, Inc., jrobinson@vervetx.com[20] - Media Contact: Ashlea Kosikowski, 1AB, ashlea@1abmedia.com[20] Condensed Consolidated Financial Information 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 |