Korro Bio(KRRO) - 2025 Q2 - Quarterly Results

Business Overview and Highlights Korro reported significant progress in its Phase 1/2a REWRITE clinical trial for KRRO-110, achieving EMA Orphan Drug Designation and ending Q2 2025 with a strong cash position, with key milestones anticipated in H2 2025 Second Quarter 2025 Highlights Korro reported significant progress in its Phase 1/2a REWRITE clinical trial for KRRO-110, having dosed over 80% of healthy volunteers with no serious adverse events. The company received EMA Orphan Drug Designation for KRRO-110 and ended the second quarter of 2025 with a strong cash position of $119.6 million. Key milestones, including interim trial data and a new development candidate announcement, are expected in the second half of 2025 - The Phase 1/2a REWRITE clinical trial for KRRO-110 is progressing well, with an interim data readout expected in the second half of 2025[4][6] - Dosing has been completed for over 80% of planned healthy volunteers in the single ascending dose (SAD) cohorts, with no treatment-emergent serious adverse events (SAEs) or dose-limiting toxicities observed[6][7] - The European Medicines Agency (EMA) granted Orphan Drug Designation to KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)[6][7] - The company plans to announce its rare metabolic disorder development candidate by the end of 2025[4][6] - Ended the second quarter of 2025 with $119.6 million in cash, cash equivalents, and marketable securities[6] Pipeline and Strategic Updates Korro's KRRO-110 program is advancing with positive clinical trial results and regulatory designations, while the company progresses towards key milestones including new development candidates and strategic collaborations through 2027 KRRO-110 Program Update The Phase 1/2a REWRITE clinical trial for KRRO-110 in AATD is advancing well, with over 80% of healthy volunteers dosed across multiple single ascending dose (SAD) cohorts, showing a safe and well-tolerated profile. The drug also received Orphan Drug Designation from the European Medicines Agency (EMA), adding to its previous U.S. FDA designation, which provides significant development incentives - More than 80% of planned healthy volunteers have received KRRO-110 across multiple SAD cohorts, including dose levels expected to be pharmacologically relevant[7] - As of August 12, 2025, KRRO-110 continues to be safe and well-tolerated, with no treatment-emergent SAEs or dose-limiting toxicities observed[7] - The EMA granted orphan designation status to KRRO-110, which provides development incentives such as protocol assistance, reduced regulatory fees, and market exclusivity upon approval[7][8] - KRRO-110 also received orphan drug designation from the U.S. Food and Drug Administration in March 2025[8] Anticipated Upcoming Milestones Korro anticipates several key milestones through 2027, guided by its 3-2-1 strategy. Near-term events include an interim data readout from the KRRO-110 trial in H2 2025, the announcement of a new rare metabolic disorder candidate by year-end 2025, and the full completion of the REWRITE trial in 2026. The company is also progressing its central nervous system programs and its collaboration with Novo Nordisk - Interim data from the Part 1 SAD portion of the Phase 1/2a REWRITE clinical trial is expected in the second half of 2025[14] - Completion of the full REWRITE trial, including the Part 2 multiple ascending dose (MAD) portion, is expected in 2026[14] - A development candidate for its rare metabolic disorder program will be announced by the end of 2025[14] - The company continues to execute its 3-2-1 strategy, aiming for three clinical-stage programs and targeting two tissue types by the end of 2027[14] - Progress continues on the collaboration with Novo Nordisk to advance up to two preclinical programs for cardiometabolic diseases[14] Financial Performance Korro's second quarter 2025 financial results show increased collaboration revenue and operating expenses, resulting in a net loss, while maintaining a strong cash position expected to fund operations into 2027 Second Quarter 2025 Financial Results For the second quarter of 2025, Korro reported $1.5 million in collaboration revenue, a new stream compared to the prior year. Operating expenses increased to $28.7 million, driven primarily by higher R&D costs, resulting in a net loss of $25.8 million. The company maintained a solid financial position, ending the quarter with $119.6 million in cash and equivalents, which is expected to fund operations into 2027 Financial Metric Summary | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Cash & Marketable Securities | $119.6M (as of Jun 30) | $163.1M (as of Dec 31, 2024) | -$43.5M | | Collaboration Revenue | $1.5M | $0.0M | +$1.5M | | R&D Expenses | $21.0M | $17.1M | +$3.9M | | G&A Expenses | $7.6M | $7.0M | +$0.6M | | Net Loss | $25.8M | $21.8M | +$4.0M | - Cash, cash equivalents, and marketable securities of $119.6 million as of June 30, 2025, are expected to fund operating expenses and capital expenditure requirements into 2027[10] - The increase in collaboration revenue was due to revenue earned from the collaboration with Novo Nordisk[11] - The rise in R&D expenses was primarily driven by increases in personnel expenses and other research and pre-development candidate costs[12] Condensed Consolidated Statements of Operations This section provides the unaudited condensed consolidated statements of operations for the three and six months ended June 30, 2025, and June 30, 2024. It details revenue, operating expenses, other income, net loss, and comprehensive loss for these periods Condensed Consolidated Statements of Operations (in thousands) | (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue | $1,460 | $— | $4,010 | $— | | Research and development | $21,031 | $17,138 | $40,770 | $30,710 | | General and administrative | $7,631 | $6,987 | $15,462 | $14,868 | | Total operating expenses | $28,662 | $24,125 | $56,232 | $45,578 | | Loss from operations | ($27,202) | ($24,125) | ($52,222) | ($45,578) | | Other income, net | $1,433 | $2,329 | $3,066 | $4,242 | | Net loss | ($25,770) | ($21,826) | ($49,157) | ($41,383) | | Net loss per share | ($2.74) | ($2.43) | ($5.24) | ($4.87) | Selected Condensed Consolidated Balance Sheet Data This section presents selected unaudited condensed consolidated balance sheet data as of June 30, 2025, compared to December 31, 2024. It highlights key figures including cash, total assets, total liabilities, and total stockholders' equity Selected Condensed Consolidated Balance Sheet Data (in thousands) | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $119,626 | $163,054 | | Working capital | $85,461 | $116,572 | | Total assets | $180,425 | $226,240 | | Total liabilities | $65,322 | $65,825 | | Total stockholders' equity | $115,103 | $160,415 | Company and Program Information This section details the REWRITE clinical trial design, the genetic basis of AATD, KRRO-110's mechanism of action, and Korro Bio's proprietary RNA editing platform for genetic medicines About REWRITE Clinical Trial The REWRITE trial is a two-part, single and multiple dose-escalating study designed to evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including both healthy adults and AATD patients. The trial will also assess pharmacokinetic and pharmacodynamic parameters to guide future dose selection, with interim data from Part 1 expected in the second half of 2025 and full study completion anticipated in 2026 - REWRITE is a two-part study (Part 1: single ascending doses; Part 2: multiple ascending doses) that will enroll up to 64 participants, including healthy volunteers and AATD patients with the PiZZ genotype[16] - The primary endpoints are safety and tolerability, while secondary endpoints include pharmacokinetic and pharmacodynamic parameters to guide optimal dose selection[16] - Interim data from Part 1 is expected in the second half of 2025, and the study is anticipated to be completed in 2026[16] About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110 Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder caused by a mutation in the SERPINA1 gene, leading to severe lung and liver disease. Korro's lead candidate, KRRO-110, utilizes its proprietary OPERA® RNA editing platform to co-opt an endogenous enzyme (ADAR) to repair the mutated RNA. This process is designed to restore the secretion of normal AAT protein, potentially clearing harmful protein aggregates from the liver and protecting lung function - AATD is a genetic disorder most commonly caused by a single G-to-A missense mutation in the SERPINA1 gene, leading to pulmonary emphysema and/or hepatic cirrhosis[17] - KRRO-110 is an RNA editing oligonucleotide from the OPERA® platform designed to use the endogenous ADAR enzyme to edit the mutated SERPINA1 RNA[17] - The therapeutic goal is to repair an amino acid codon, restore normal AAT protein secretion, clear protein aggregates from the liver, and preserve lung function[17] About Korro Bio Korro Bio is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing for both rare and prevalent diseases. The company's proprietary OPERA® platform is designed to harness the body's natural RNA editing process to make precise, transient single-base edits. This approach aims to offer improved specificity and long-term tolerability compared to DNA-based genetic medicines - Korro is a clinical-stage biopharmaceutical company developing genetic medicines based on editing RNA[18] - The company's platform is designed to enable precise yet transient single base edits by harnessing the body's natural RNA editing process[18] - By editing RNA instead of DNA, Korro aims to expand the reach of genetic medicines with increased specificity and improved long-term tolerability[18] Legal Disclosures This section provides cautionary language regarding forward-looking statements, highlighting inherent risks and uncertainties in future projections and directing readers to SEC filings for comprehensive risk factors Forward-Looking Statements This section contains standard cautionary language regarding forward-looking statements within the press release. It advises investors that statements about future events, such as clinical trial timelines, data readouts, candidate announcements, and financial runway, are subject to significant risks and uncertainties and should not be unduly relied upon. Readers are directed to Korro's SEC filings for a more comprehensive discussion of potential risks - The press release includes forward-looking statements concerning the timing of clinical trial data, announcement of development candidates, KRRO-110's potential, and the company's cash runway[21] - Actual results may differ materially from current expectations due to various factors, including risks inherent in clinical trials, regulatory oversight, and general economic conditions[21] - Korro does not undertake any duty to publicly update or revise forward-looking statements and advises investors to consult its SEC filings for a full list of risk factors[21]