FREQUENCY THERAP(FREQ) - 2024 Q4 - Annual Results

Executive Summary and Business Highlights Introduction and CEO Commentary The company reported 2024 results and business updates, highlighting key achievements and strategic goals for its RNA editing platform - Korro Bio is a clinical-stage biopharmaceutical company focused on developing genetic medicines based on RNA editing for rare and prevalent diseases[2][17] - Key objectives for 2024 included initiating the Phase 1/2a REWRITE clinical study of KRRO-110, announcing a collaboration with Novo Nordisk, and advancing multiple product candidates[3] - KRRO-110 is anticipated to demonstrate best-in-class potential with interim data readout of REWRITE in H2 2025[4] - The company plans to announce its second development candidate by the end of 2025 and aims for three clinical-stage development candidates by the end of 2027[4] Key Achievements and Strategic Outlook The company advanced its lead candidate KRRO-110, secured key designations, and maintained a strong financial position - Completed dosing of the first two cohorts in the Phase 1/2a REWRITE Clinical Study of KRRO-110 for AATD, with interim readout expected in H2 2025[6][7] - KRRO-110 received U.S. FDA Orphan Drug Designation for AATD treatment[6][7][8] - Ended 2024 with $163.1 million in cash, cash equivalents, and marketable securities, providing a cash runway into H2 2026[6][9] - Executing a '3-2-1 strategy' to establish three clinical-stage development programs by the end of 2027[6][12] Pipeline and Business Updates KRRO-110 Clinical Development for AATD The company's lead candidate, KRRO-110, progressed in its Phase 1/2a study and received Orphan Drug Designation REWRITE Clinical Study Progress The REWRITE study completed dosing for its initial cohorts with no serious adverse events, targeting an interim readout in H2 2025 - Completed dosing of the first two single ascending dose cohorts in healthy adult volunteers in Phase 1/2a REWRITE clinical study of KRRO-110[6][7] - No serious adverse events observed through completion of dosing of the first two cohorts[7] - Interim readout from Phase 1/2a REWRITE clinical study of KRRO-110 for AATD expected in the second half of 2025, and study completion expected in 2026[6][7][12] Orphan Drug Designation KRRO-110 received Orphan Drug Designation from the U.S. FDA, providing key development incentives for treating AATD - KRRO-110 granted Orphan Drug Designation by the U.S. FDA for the treatment of AATD[6][7] - Orphan Drug designation provides various development incentives, including tax credits, user fee exemptions, and potential market exclusivity following FDA approval[8] Strategic Pipeline and Corporate Development The company is executing its '3-2-1 strategy' to expand its pipeline while advancing collaborations and strengthening its leadership team 3-2-1 Strategy and Future Candidates The '3-2-1 strategy' aims for three clinical programs by 2027, with a second development candidate nomination expected by the end of 2025 - Executing 3-2-1 strategy through end of 2027 with the goal of establishing three clinical-stage development programs, targeting two tissue types with a single RNA-editing platform[6][12] - Second development candidate expected to be announced in 2025[6] - Korro expects to nominate a development candidate for its rare metabolic disorder program targeting the liver by the end of 2025[12] Collaborations and Leadership Enhancements The collaboration with Novo Nordisk is progressing, and the company has strengthened its leadership with key appointments - Collaboration with Novo Nordisk continues to progress, leveraging Korro's OPERA platform for up to two disease targets[12] - Strengthened leadership team with the formation of Clinical Advisory Board and key additions to board and management team[6][12] - GaoZhong Zhu, Ph.D. joined Korro as Senior Vice President of Chemistry, Manufacturing and Controls (CMC) & Technical Operations[12] Full Year 2024 Financial Results Cash Position The company ended 2024 with $163.1 million in cash and equivalents, sufficient to fund operations into the second half of 2026 | Metric | As of Dec 31, 2024 | As of Dec 31, 2023 | Change | | :-------------------------------- | :------------------- | :------------------- | :----- | | Cash, cash equivalents and marketable securities | $163.1 million | $166.1 million | -$3.0 million | - Cash runway expected into the second half of 2026[6][9] Collaboration Revenue The company recognized its first collaboration revenue of $2.3 million in 2024 from its partnership with Novo Nordisk | Metric | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | Change | | :------------------- | :---------------------- | :---------------------- | :----- | | Collaboration revenue | $2.271 million | $0 | +$2.271 million | - Increase due to collaboration revenue earned in the fourth quarter of 2024 from the company's partnership with Novo Nordisk[10] Operating Expenses Total operating expenses increased to $94.2 million in 2024, driven by higher R&D and G&A costs Research and Development (R&D) Expenses R&D expenses rose to $63.6 million in 2024 due to increased costs for the KRRO-110 program, personnel, and facilities | Metric | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | Change | | :------------------- | :---------------------- | :---------------------- | :----- | | R&D expenses | $63.636 million | $57.250 million | +$6.386 million | - Increase driven primarily by increases in KRRO-110 external research and development expenses, personnel expenses, and facilities expenses[11] General and Administrative (G&A) Expenses G&A expenses increased to $30.5 million in 2024, primarily from higher IT, insurance, and professional fees | Metric | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | Change | | :------------------- | :---------------------- | :---------------------- | :----- | | G&A expenses | $30.545 million | $27.284 million | +$3.261 million | - Increase primarily due to increased information technology, insurance and other costs, as well as increased professional fees and facility costs[13] Net Loss The company's net loss slightly increased to $83.6 million for the year ended December 31, 2024 | Metric | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | Change | | :------- | :---------------------- | :---------------------- | :----- | | Net loss | $(83.581) million | $(81.172) million | $(2.409) million | | Metric | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | | :-------------------------------------------------- | :---------------------- | :---------------------- | | Net loss per share, basic and diluted | $(9.37) | $(53.08) | | Weighted-average shares used in computing net loss per share | 8,920,561 | 1,529,321 | Program and Company Information About REWRITE Study The REWRITE study evaluates the safety and tolerability of KRRO-110 in healthy adults and AATD patients - REWRITE is a two-part single and multiple dose-escalating study that will evaluate the safety and tolerability of KRRO-110 in up to 64 participants[15] - Secondary and exploratory endpoints include pharmacokinetic and pharmacodynamic parameters to guide optimal dose selection for later stage studies[15] - Interim data from Part 1 is expected in the second half of 2025, with study completion anticipated in 2026[15] About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110 AATD is a genetic disorder that KRRO-110 aims to treat by editing RNA to restore normal protein function - 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[16] - KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro's proprietary OPERA™ platform[16] - KRRO-110 is designed to edit the SERPINA1 RNA to restore secretion of normal AAT protein, potentially clearing liver aggregates and preserving lung function[16] About Korro Bio Korro Bio is a clinical-stage company developing a new class of genetic medicines based on its proprietary OPERA™ RNA editing platform - Korro 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[17] - Korro's proprietary RNA editing platform, OPERA™, is designed to harness the body's natural RNA editing process for a precise, transient single base edit[16][17] - By editing RNA instead of DNA, Korro aims to expand the reach of genetic medicines with increased specificity and improved long-term tolerability[17] Financial Statements Consolidated Statements of Operations and Comprehensive Loss The company reported its first collaboration revenue of $2.3 million and a net loss of $83.6 million in 2024 | Metric | Year Ended Dec 31, 2024 (in thousands) | Year Ended Dec 31, 2023 (in thousands) | | :------------------------------------ | :------------------------------------- | :------------------------------------- | | Collaboration revenue | $2,271 | $— | | Research and development | $63,636 | $57,250 | | General and administrative | $30,545 | $27,284 | | Total operating expenses | $94,181 | $84,534 | | Loss from operations | $(91,910) | $(84,534) | | Other income, net | $8,470 | $3,389 | | Net loss | $(83,581) | $(81,172) | | Net loss per share, basic and diluted | $(9.37) | $(53.08) | | Weighted-average shares used in computing net loss per share | 8,920,561 | 1,529,321 | Selected Condensed Consolidated Balance Sheet Data The company's balance sheet shows total assets of $226.2 million and total stockholders' equity of $160.4 million as of year-end 2024 | Metric | As of Dec 31, 2024 (in thousands) | As of Dec 31, 2023 (in thousands) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Cash, cash equivalents and investments | $163,054 | $166,150 | | Working capital | $116,572 | $153,245 | | Total assets | $226,240 | $221,663 | | Total liabilities | $65,825 | $51,752 | | Total stockholders' equity | $160,415 | $169,911 | Legal and Contact Information Forward-Looking Statements This section outlines the inherent risks and uncertainties associated with forward-looking statements contained in the report - Forward-looking statements are based on current expectations and assumptions that are inherently uncertain[20] - Factors that may cause actual results to differ materially include risks of conducting a clinical study, regulatory oversight, enrollment risks, and general economic conditions[20] - Readers should not place undue reliance on forward-looking statements in this press release, which speak only as of the date they are made[20] Investor & Media Contact This section provides contact details for investor and media inquiries and lists official disclosure channels - Investor & Media Contact: Tim Palmer, IR@korrobio.com[21] - Korro intends to use its Investor Relations website, LinkedIn, and X (Twitter) as means of disclosing material nonpublic information[18][19]