FREQUENCY THERAP(FREQ) - 2025 Q1 - Quarterly Results

Executive Summary & Company Overview Introduction and Strategic Highlights Korro Bio reported Q1 2025 results, highlighting progress on KRRO-110 interim data in H2 2025 and its 3-2-1 strategy through 2027 - Korro Bio is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases[3] - The company remains on track to report interim data from its Phase 1/2a REWRITE clinical trial of KRRO-110 in the second half of 2025[4] - Korro is focused on executing its '3-2-1 strategy' through 2027, aiming to develop novel genetic medicines[4] Operational Streamlining and Workforce Reduction Korro is streamlining operations and reducing its workforce by 20% to advance key programs, incurring approximately $1.2 million in one-time charges - Korro is streamlining operations and reducing its workforce by approximately 20% to advance programs to key value inflection points[5] - The company estimates one-time restructuring charges of approximately $1.2 million, primarily for employee severance, benefits, and related termination costs, with the majority expected in Q2 2025[5] - The streamlining is considered essential for long-term success, prioritizing KRRO-110 development and focused investments in the OPERA platform[5] Pipeline and Business Updates KRRO-110 Clinical Trial Progress The KRRO-110 REWRITE clinical trial for AATD is expanding globally, with interim data expected in H2 2025 and trial completion in 2026 - Korro received regulatory approval to expand the REWRITE clinical trial in New Zealand and is progressing enrollment, site activation, and expansion into other geographies, including the United States[6][8] - An interim readout from the Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD is on track for the second half of 2025[7][8][12] - Completion of the REWRITE clinical trial is expected in 2026[5][8][12] Strategic Initiatives and Collaborations Korro is executing its 3-2-1 strategy to establish clinical programs and advancing two preclinical programs with Novo Nordisk for cardiometabolic diseases - Korro continues to execute its 3-2-1 strategy, aiming to establish three clinical-stage development programs targeting two tissue types by leveraging its OPERA™ platform[12] - The collaboration with Novo Nordisk is progressing, with Korro working to advance up to two programs through preclinical development for cardiometabolic diseases[5][12] - Korro's CNS programs are also continuing to progress[12] Leadership Team Enhancements Korro strengthened its leadership team with the appointments of Loïc Vincent, Ph.D., as CSO, and GaoZhong Zhu, Ph.D., as SVP of CMC - Loïc Vincent, Ph.D., was appointed Chief Scientific Officer, bringing over 20 years of experience in drug discovery and translational research[12] - GaoZhong Zhu, Ph.D., was appointed Senior Vice President of Chemistry, Manufacturing and Controls, with over 25 years of industrial experience in CMC strategies[12] Anticipated Upcoming Milestones Key milestones include KRRO-110 interim data in H2 2025, a second development candidate nomination by year-end, and continued Novo Nordisk partnership progress - Interim readout from Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD expected in the second half of 2025[12] - Korro expects to nominate a development candidate by the end of 2025 for its rare metabolic disorder program targeting the liver (GalNAc) with subcutaneous delivery[7][12] - Progress partnership with Novo Nordisk in cardiometabolic diseases with high prevalence[12] First Quarter 2025 Financial Results Cash Position Korro's cash, cash equivalents, and marketable securities decreased to $139.0 million in Q1 2025, projected to fund operations into 2027 Cash, Cash Equivalents and Marketable Securities | Date | Amount (in millions) | | :--- | :--- | | March 31, 2025 | $139.0 | | December 31, 2024 | $163.1 | | Change | ($24.1) | - Korro expects its cash, cash equivalents, and marketable securities as of March 31, 2025, will fund operating expenses and capital expenditure requirements into 2027[5][7][10] Collaboration Revenue Collaboration revenue significantly increased to $2.6 million in Q1 2025, primarily due to the Novo Nordisk agreement Collaboration Revenue (Three Months Ended March 31) | Year | Amount (in thousands) | | :--- | :--- | | 2025 | $2,550 | | 2024 | $0 | | Change | +$2,550 | - The increase in collaboration revenue was due to revenue earned in the first quarter of 2025 from the agreement with Novo Nordisk[11] Operating Expenses Total operating expenses increased to $27.6 million in Q1 2025, driven by higher R&D expenses, with G&A remaining stable Total Operating Expenses (Three Months Ended March 31) | Year | Amount (in thousands) | | :--- | :--- | | 2025 | $27,570 | | 2024 | $21,453 | | Change | +$6,117 | Research and Development (R&D) Expenses R&D expenses increased to $19.7 million in Q1 2025, primarily due to higher KRRO-110 external R&D, personnel, and pre-development candidate expenses R&D Expenses (Three Months Ended March 31) | Year | Amount (in thousands) | | :--- | :--- | | 2025 | $19,739 | | 2024 | $13,572 | | Change | +$6,167 | - The increase in R&D expenses was primarily driven by increases in KRRO-110 external research and development expenses, personnel expenses, and other research and pre-development candidate expenses[13] General and Administration (G&A) Expenses G&A expenses slightly decreased to $7.8 million in Q1 2025, mainly due to lower professional service and facilities fees G&A Expenses (Three Months Ended March 31) | Year | Amount (in thousands) | | :--- | :--- | | 2025 | $7,831 | | 2024 | $7,881 | | Change | -$50 | - The minor decrease in G&A expenses was primarily driven by a $0.1 million decrease in professional service fees and facilities expenses, partially offset by an increase in personnel expenses[14] Net Loss Korro's net loss widened to $23.4 million in Q1 2025, compared to $19.6 million in Q1 2024, reflecting increased operating expenses Net Loss (Three Months Ended March 31) | Year | Amount (in thousands) | | :--- | :--- | | 2025 | $(23,387) | | 2024 | $(19,557) | | Change | -($3,830) | Net Loss Per Share and Weighted-Average Shares | Metric | 2025 | 2024 | | :--- | :--- | :--- | | Net loss per share, basic and diluted | $(2.49) | $(2.44) | | Weighted-average shares used in computing net loss per share, basic and diluted | 9,384,266 | 8,019,626 | Balance Sheet Highlights Korro's total assets decreased to $202.2 million as of March 31, 2025, with total liabilities and stockholders' equity also decreasing Selected Condensed Consolidated Balance Sheet Data (in thousands) | Metric | March 31, 2025 | December 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $138,992 | $163,054 | $(24,062) | | Working capital | $108,354 | $116,572 | $(8,218) | | Total assets | $202,207 | $226,240 | $(24,033) | | Total liabilities | $63,250 | $65,825 | $(2,575) | | Total stockholders' equity | $138,957 | $160,415 | $(21,458) | Product and Company Information About REWRITE Clinical Trial The REWRITE clinical trial is a two-part, dose-escalating study for KRRO-110 in up to 64 participants, with interim data expected in H2 2025 - REWRITE is a two-part single and multiple dose-escalating study evaluating the safety and tolerability of KRRO-110[16] - The study will enroll up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype[16] - Interim data from Part 1 (single ascending doses) is expected in the second half of 2025, and completion of the study is anticipated in 2026[16] About AATD and KRRO-110 AATD is a genetic disorder caused by a SERPINA1 gene mutation; KRRO-110 aims to repair SERPINA1 RNA to restore AAT protein secretion - AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene, leading to pulmonary emphysema and/or hepatic cirrhosis[17] - KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro's proprietary OPERA™ platform[17] - KRRO-110 is designed to co-opt the ADAR enzyme to edit the 'A' variant on SERPINA1 RNA, repair an amino acid codon, and restore secretion of normal AAT protein, with potential to clear liver protein aggregates and preserve lung function[17] About Korro Bio Korro Bio is a clinical-stage biopharmaceutical company developing RNA editing genetic medicines for various diseases using its proprietary OPERA™ platform - Korro is a clinical-stage biopharmaceutical company developing genetic medicines based on editing RNA for rare and highly prevalent diseases[18] - The company's proprietary OPERA™ platform harnesses the body's natural RNA editing process for precise yet transient single-base edits[18] - By editing RNA instead of DNA, Korro aims to deliver increased precision, tunability, specificity, and improved long-term tolerability[18] Additional Information Investor Relations Korro will use its IR website, LinkedIn, and X (Twitter) as official channels for disclosing material nonpublic information and Regulation FD compliance - Korro intends to use its Investor Relations website, LinkedIn, and X (Twitter) for disclosing material nonpublic information and complying with Regulation FD[19] - Investors should monitor these channels in addition to press releases, SEC filings, public conference calls, presentations, and webcasts[19] Forward-Looking Statements This press release contains forward-looking statements about future expectations, subject to inherent uncertainties and risks that may cause actual results to differ - The press release contains forward-looking statements regarding future expectations, hopes, beliefs, intentions, or strategies of Korro[20] - Examples include timing of data readouts, trial completion, execution of the 3-2-1 strategy, timing of second development candidate nomination, cash runway, KRRO-110's potential, and benefits of workforce reduction and Novo Nordisk collaboration[20] - Forward-looking statements are based on current expectations and assumptions that are inherently uncertain, and actual results may differ materially due to various factors and risks outlined in SEC filings[20] Contact Information Contact details are provided for investor and media inquiries - Investor & Media Contact: IR@korrobio.com[21] Financial Statements Condensed Consolidated Statements of Operations and Comprehensive Loss This section presents unaudited condensed consolidated statements of operations and comprehensive loss for Q1 2025 and 2024, detailing revenue, expenses, and net loss Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share amounts) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Collaboration revenue | $2,550 | $— | | Research and development | $19,739 | $13,572 | | General and administrative | $7,831 | $7,881 | | Total operating expenses | $27,570 | $21,453 | | Loss from operations | $(25,020) | $(21,453) | | Other income, net | $1,633 | $1,913 | | Loss before provision for income taxes | $(23,387) | $(19,540) | | Provision for income taxes | $— | $(17) | | Net loss | $(23,387) | $(19,557) | | Other comprehensive income (loss): | | | | Unrealized gain on available-for-sale investments | $(3) | $— | | Foreign currency translation adjustments, net | $(1) | $— | | Comprehensive loss | $(23,391) | $(19,557) | | Net loss per share, basic and diluted | $(2.49) | $(2.44) | | Weighted-average shares used in computing net loss per share, basic and diluted | 9,384,266 | 8,019,626 | Selected Condensed Consolidated Balance Sheet Data This section provides selected unaudited condensed consolidated balance sheet data as of March 31, 2025, and December 31, 2024 Selected Condensed Consolidated Balance Sheet Data (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $138,992 | $163,054 | | Working capital | $108,354 | $116,572 | | Total assets | $202,207 | $226,240 | | Total liabilities | $63,250 | $65,825 | | Total stockholders' equity | $138,957 | $160,415 |