Verve Therapeutics(VERV) - 2023 Q4 - Annual Results

Verve Therapeutics Q4 and Full Year 2023 Earnings Release Executive Summary Verve Therapeutics reported significant 2023 progress, highlighted by the first human proof-of-concept for in vivo base editing from the VERVE-101 Heart-1 trial, anticipating three clinical-stage programs in 2024, supported by $624 million in cash and an Eli Lilly collaboration - Announced the first human proof-of-concept data for in vivo base editing from the Heart-1 Phase 1b clinical trial of VERVE-101[1][2][5] - Expects to have three clinical-stage programs in 2024 targeting cardiovascular diseases by editing PCSK9 and ANGPTL3 genes[1][2] - Ended the year with a strong cash position of $624 million, providing a cash runway into late 2026[1][16] - Strengthened collaboration with Eli Lilly, an expert in cardiometabolic disease and late-stage development[2][15] Pipeline Progress and Clinical Updates Verve advanced its gene editing pipeline with VERVE-101 showing LDL-C reduction and FDA IND clearance, and plans to initiate Phase 1b trials for VERVE-102 and VERVE-201 in 2024 VERVE-101 (Heart-1 Trial & FDA Clearance) Interim data from the Heart-1 Phase 1b trial for VERVE-101 demonstrated successful in vivo base editing in humans, leading to significant LDL-C and PCSK9 reductions, with FDA IND clearance enabling U.S. trial expansion and expected enrollment completion in 2024 - VERVE-101 is an in vivo base editing medicine designed to inactivate the PCSK9 gene to treat HeFH by durably reducing low-density lipoprotein cholesterol (LDL-C)[3] VERVE-101 Heart-1 Interim Trial Results | Metric | Reduction | | :--- | :--- | | Time-averaged LDL-C | Up to 55% | | Blood PCSK9 protein | Up to 84% | - The company received Investigational New Drug (IND) application clearance from the U.S. FDA in October 2023, enabling the activation of U.S. trial sites[8] - Verve expects to complete enrollment of the Heart-1 clinical trial in 2024 and provide a data update in the second half of the year[7] VERVE-102 (Heart-2 Trial) VERVE-102, targeting PCSK9 with proprietary GalNAc-LNP delivery, plans to initiate its Heart-2 Phase 1b clinical trial outside the U.S. in the first half of 2024 for patients with HeFH or premature coronary artery disease - VERVE-102 targets the same PCSK9 gene as VERVE-101 but uses a proprietary GalNAc-LNP delivery technology[9] - The Heart-2 Phase 1b clinical trial is expected to be initiated in the first half of 2024, subject to regulatory clearances outside the U.S[10] - Following evaluation of data from both Heart-1 and Heart-2 trials, Verve plans to start a randomized, placebo-controlled Phase 2 trial of either VERVE-101 or VERVE-102 in 2025[10] VERVE-201 VERVE-201 is an in vivo base editing medicine designed to inactivate the ANGPTL3 gene to treat HoFH and refractory hypercholesterolemia, with a Phase 1b clinical trial expected to be initiated in the second half of 2024 - VERVE-201 is designed to inactivate the ANGPTL3 gene to treat patients with homozygous familial hypercholesterolemia (HoFH) and refractory hypercholesterolemia[11] - A Phase 1b clinical trial for VERVE-201 is expected to be initiated in the second half of 2024, with an initial regulatory strategy outside the U.S[12] Corporate Developments Verve significantly strengthened its financial position in late 2023 by raising approximately $157.7 million in net proceeds through a public offering and a concurrent private placement with Eli Lilly, while also expanding its collaboration with Lilly Recent Financing Activities (December 2023) | Financing Type | Shares Issued | Price Per Share | Net Proceeds | | :--- | :--- | :--- | :--- | | Public Offering | 14,375,000 | $10.00 | ~$134.7M | | Private Placement (Eli Lilly) | 2,296,317 | $10.00 | ~$23.0M | - Expanded its collaboration with Eli Lilly, which acquired certain rights to Verve's in vivo gene editing programs targeting PCSK9, ANGPTL3, and a third cardiovascular target[15] - Verve retains control of the development and commercialization of all collaboration products under the agreement with Lilly[15] Fourth Quarter and Full Year 2023 Financial Results Verve ended 2023 with a robust cash position of $624.0 million, with full-year collaboration revenue growing to $11.8 million, increased operating expenses, and a net loss of $200.1 million - Cash, cash equivalents, and marketable securities were $624.0 million as of December 31, 2023, expected to fund operations into late 2026[16] Full Year 2023 vs. 2022 Financial Performance (in millions) | Metric | FY 2023 | FY 2022 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $11.8 | $1.9 | +$9.9 | | R&D Expenses | $184.9 | $130.1 | +$54.8 | | G&A Expenses | $49.9 | $37.5 | +$12.4 | | Net Loss | ($200.1) | ($157.4) | +($42.7) | Q4 2023 vs. Q4 2022 Financial Performance (in millions) | Metric | Q4 2023 | Q4 2022 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $5.1 | $1.0 | +$4.1 | | R&D Expenses | $46.8 | $37.3 | +$9.5 | | G&A Expenses | $12.3 | $11.4 | +$0.9 | | Net Loss | ($48.4) | ($41.1) | +($7.3) | Selected Condensed Consolidated Financial Information Condensed consolidated financial statements detail increased collaboration revenue and operating expenses for Q4 and full year 2023, resulting in a larger net loss, while the balance sheet reflects a stronger year-end 2023 financial position with higher cash and total assets Condensed Consolidated Statements of Operations (in thousands) | | Year ended Dec 31, 2023 | Year ended Dec 31, 2022 | | :--- | :--- | :--- | | Collaboration revenue | $11,758 | $1,941 | | Total operating expenses | $234,882 | $167,628 | | Loss from operations | ($223,124) | ($165,687) | | Net loss | ($200,068) | ($157,387) | | Net loss per share | ($3.12) | ($2.91) | Condensed Consolidated Balance Sheet Data (in thousands) | | Dec 31, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $623,950 | $554,808 | | Total assets | $752,688 | $679,223 | | Total liabilities | $153,186 | $128,291 | | Total stockholders' equity | $599,502 | $550,932 |