Actively recruiting clinical trials in neurology and rheumatology with KYV-101 No severe ICANS or CRS reported in first 36 autoimmune disease patients treated with KYV-101 Received FDA RMAT Designation for KYV-101 for treatment of patients with Stiff-Person Syndrome Received FDA RMAT Designation for KYV-101 for treatment of patients with Myasthenia Gravis Reported clinical experience with first KYV-101 patient disease-free at 1 year after treatment EMERYVILLE, Calif., Aug. 12, 2024 /PRNewswire/ -- Kyverna T ...

Company Overview - Kyverna Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing cell therapies for autoimmune diseases [4][14] - The company has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its product candidate KYV-101, aimed at treating refractory stiff-person syndrome [4][7] Product Development - KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate being evaluated in Phase 1/2 and Phase 2 trials in the U.S. and Germany [3][5] - The product is designed to improve tolerability and has been tested in a 20-patient Phase 1 trial in oncology, with results published in Nature Medicine [5] - KYV-101 is currently being evaluated for multiple indications, including stiff-person syndrome, multiple sclerosis, myasthenia gravis, systemic sclerosis, and lupus nephritis [9][6] Clinical Trials and Outcomes - The company has treated 50 patients with KYV-101 across more than 15 locations in Europe and the U.S. [8] - The ongoing KYSA-8 trial is anticipated to provide data that could significantly alter the treatment landscape for stiff-person syndrome [1][5] - The CEO expressed optimism about the potential for KYV-101 to offer durable, immunosuppressant-free remission for patients [5] Market Context - Stiff-person syndrome is a rare autoimmune disorder characterized by debilitating muscle stiffness, impacting mobility and quality of life [7] - The designation as RMAT will enable Kyverna to receive guidance from FDA officials on efficient drug development and the use of surrogate endpoints [7]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ________________ Commission File Number: 001-41947 Kyverna Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) Delaware ...

[Kyverna Therapeutics First Quarter 2024 Business Update and Financial Results](index=1&type=section&id=Kyverna%20Therapeutics%20First%20Quarter%202024%20Business%20Update%20and%20Financial%20Results) [Business Highlights](index=1&type=section&id=First%20Quarter%202024%20and%20Recent%20Business%20Highlights) The company advanced its lead CAR T-cell therapy, KYV-101, treating 30 patients across multiple indications and strengthened its financial position with a successful IPO - Advanced clinical development of its lead product candidate, KYV-101, a proprietary CD19 CAR T-cell therapy, in both the US and Europe[2](index=2&type=chunk)[6](index=6&type=chunk) - Cumulatively treated **30 patients** as of May 14, 2024, including 8 with myasthenia gravis, 7 with lupus nephritis, and 4 with multiple sclerosis[5](index=5&type=chunk)[6](index=6&type=chunk) - The U.S. FDA granted **Orphan Drug Designation** to KYV-101 for the treatment of myasthenia gravis[6](index=6&type=chunk) - Strengthened its balance sheet with approximately **$366.9 million in gross proceeds** from its initial public offering in February 2024[6](index=6&type=chunk) - Progressed the global KYSA clinical trial program across multiple indications: lupus nephritis (Phase 1/2), systemic sclerosis (Phase 1/2), myasthenia gravis (Phase 2), and multiple sclerosis (Phase 2)[6](index=6&type=chunk) [Financial Results](index=1&type=section&id=Financial%20Results%20for%20the%20Quarter%20Ended%20March%2031%2C%202024) The company reported a higher net loss of $26.7 million driven by increased R&D expenses but maintains a robust cash position of $369.8 million post-IPO [Financial Performance Summary](index=1&type=section&id=Financial%20Performance%20Summary) Net loss increased to $26.7 million in Q1 2024, while the company ended the quarter with a strong liquidity position of $369.8 million Key Financial Metrics | Financial Metric | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Net Loss | $26.7 million | $11.1 million | | Net Loss Per Share | $1.12 | $12.10 | | Net Cash Used in Operating Activities | $25.5 million | $11.5 million | - As of March 31, 2024, the company held **$369.8 million** in cash, cash equivalents, and available-for-sale marketable securities[4](index=4&type=chunk) - The cash position includes approximately **$336.2 million in net proceeds** from the initial public offering completed in February 2024[4](index=4&type=chunk) [Condensed Statements of Operations](index=3&type=section&id=Condensed%20Statements%20of%20Operations) Total operating expenses surged to $29.4 million, driven primarily by a significant year-over-year increase in R&D expenses to $22.5 million Selected Statement of Operations Data | (in thousands) | Three Months Ended March 31, 2024 | Three Months Ended March 31, 2023 | | :--- | :--- | :--- | | Research and development | $22,476 | $8,711 | | General and administrative | $6,882 | $2,734 | | **Total operating expenses** | **$29,358** | **$11,445** | | Loss from operations | $(29,358) | $(11,445) | | Interest income | $2,735 | $349 | | **Net loss** | **$(26,693)** | **$(11,143)** | [Condensed Balance Sheets](index=4&type=section&id=Condensed%20Balance%20Sheets) Total assets grew to $390.0 million and stockholders' equity became positive at $361.0 million, reflecting the impact of the recent IPO Selected Balance Sheet Data | (in thousands) | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $224,287 | $34,647 | | Available-for-sale marketable securities | $145,507 | $22,896 | | **Total current assets** | **$375,488** | **$60,664** | | **Total assets** | **$389,984** | **$75,195** | | **Liabilities & Equity** | | | | Total current liabilities | $21,865 | $19,859 | | **Total liabilities** | **$28,993** | **$26,018** | | **Total stockholders' equity (deficit)** | **$360,991** | **$(131,397)** | [Upcoming Milestones](index=1&type=section&id=Upcoming%20Milestones) The company anticipates releasing interim patient data at major medical conferences and providing updates on its manufacturing process and pipeline in 2024 - Interim patient data releases and symposia are planned for **EULAR in Q2 2024, ECTRIMS in Q3 2024, and ACR in Q4 2024**[6](index=6&type=chunk) - Anticipates ongoing regulatory progress in rheumatology and neurology in both the US and Europe[6](index=6&type=chunk) - Expects to provide updates on its **Ingenui-T manufacturing process** and its allogeneic CD19 CAR T-cell product candidate, **KYV-201**[6](index=6&type=chunk) [About Kyverna Therapeutics](index=2&type=section&id=About%20Kyverna%20Therapeutics) Kyverna is a clinical-stage biopharmaceutical company developing CAR T-cell therapies for autoimmune diseases, with its lead candidate KYV-101 in multiple trials - Kyverna is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for autoimmune diseases[7](index=7&type=chunk) - The lead CAR T-cell therapy candidate, **KYV-101**, is advancing in clinical trials for rheumatology and neurology, including Phase 2 trials for multiple sclerosis and myasthenia gravis, and Phase 1/2 trials for systemic sclerosis and lupus nephritis[8](index=8&type=chunk) - The pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats, designed for B cell-driven autoimmune diseases[9](index=9&type=chunk)

Financial Performance - The company reported a net loss of $60.4 million for the year ended December 31, 2023, compared to a net loss of $28.9 million for the previous year, with an accumulated deficit of $136.0 million as of the same date[273]. - The company anticipates continued significant losses as it progresses with research and development of product candidates KYV-101 and KYV-201, and seeks regulatory approvals[273]. - The company will require substantial additional capital to finance its operations, and failure to raise such capital could lead to delays or reductions in research and drug development programs[278]. - As of December 31, 2023, the company had federal net operating loss (NOL) carryforwards of $48.8 million and state NOL carryforwards of $103.2 million[310]. - The company expects approximately $2.0 million of federal net operating losses and $1.9 million of California net operating losses to expire unused due to Section 382 limitations[310]. Regulatory and Compliance Risks - The company has identified material weaknesses in its internal control over financial reporting, which could adversely affect investor confidence and the value of its common stock[270]. - The company will incur significant increased costs due to compliance with public company regulations, which will divert management's attention from other business concerns[288]. - The company must design effective disclosure controls to ensure timely and accurate financial reporting under the Exchange Act[295]. - The company anticipates that compliance with Section 404 of the Sarbanes-Oxley Act will require substantial additional professional fees and internal costs[296]. - The FDA is investigating serious risks associated with T-cell malignancy related to certain therapies, which may impact the company's product candidates and regulatory approval processes[272]. Operational Challenges - The company has a limited operating history since its formation in 2018, which may affect the accuracy of predictions about its future success[273]. - The company relies on third-party manufacturers and suppliers for its product candidates, and any failure in these relationships could materially affect its business[271]. - The company relies on collaborators and CROs for clinical trial conduct, which may introduce additional delays and costs[327][330]. - The company is exposed to risks related to misconduct by employees, contractors, or partners, which could adversely affect its business and financial condition[307]. - The company faces significant product liability risks during clinical trials, which could adversely affect its business and financial condition[302]. Market and Competitive Landscape - The company faces competition from established pharmaceutical and biotechnology companies that have already developed approved therapies in its target indications[271]. - The product candidate KYV-101, aimed at treating B-cell-driven autoimmune diseases, will compete with established therapies like Rituxan and Ocrevus[343]. - The company must demonstrate that its product candidates provide better alternatives to existing therapies to achieve market penetration[345]. - The market acceptance of product candidates is uncertain, and failure to achieve adequate acceptance could limit revenue generation[299]. - The company focuses its limited resources on selected product candidates, which may lead to missed opportunities for more profitable candidates[340]. Clinical Development Risks - Clinical development is lengthy and expensive, with a high risk of failure for product candidates currently in preclinical or clinical development[325]. - The company has not successfully completed any large-scale or pivotal clinical trials, which may hinder its ability to obtain regulatory approvals and commercialize products[318]. - Delays in clinical trials can result from various factors, including participant withdrawals, regulatory disagreements, and insufficient patient enrollment[328][329]. - Any delays in clinical trials could adversely affect the company's ability to commercialize product candidates and generate revenue[331]. - Undesirable side effects observed in clinical trials could lead to recruitment difficulties, trial abandonment, or regulatory delays[353]. Intellectual Property Risks - The company is dependent on intellectual property licensed from third parties, and termination of these licenses could significantly impact its ability to commercialize product candidates[367]. - The patent position for biopharmaceutical companies is highly uncertain, with complex legal questions and recent litigation increasing uncertainties regarding the enforcement of patent rights[377]. - The company may face challenges in maintaining exclusive rights to co-owned patents, which could allow competitors to market similar products[389]. - The company may not be able to detect infringement against its owned or in-licensed patents, complicating enforcement actions[411]. - The uncertainty surrounding patent rights could dissuade potential collaborators from engaging with the company, affecting future partnerships[385]. Healthcare and Legislative Environment - The company is subject to extensive healthcare laws and regulations that could expose it to penalties and affect business operations[453]. - Recent U.S. legislative actions aim to increase transparency in drug pricing and reduce prescription drug costs under Medicare[450]. - The company anticipates pricing pressures on all product candidates due to managed healthcare trends and legislative changes[452]. - The ACA has significantly changed healthcare financing in the U.S., affecting the pharmaceutical industry through potential competition from lower-cost biosimilars[466]. - The Right to Try Act allows certain patients to access IND products that have completed Phase 1 clinical trials without enrolling in clinical trials[467].

Financial Performance - For the year ended December 31, 2023, Kyverna reported a net loss of $60.4 million, or a net loss per share of $89.61, compared to a net loss of $28.9 million, or a net loss per share of $63.43 for 2022[3] - Net cash used in operating activities was $52.4 million for 2023, an increase from $36.1 million in 2022[3] - Total operating expenses for 2023 were $62.4 million, up from $36.4 million in 2022, with research and development expenses at $49.9 million[13] - The company reported a total stockholders' deficit of $131.4 million as of December 31, 2023, compared to $74.0 million in 2022[15] Cash and Funding - As of December 31, 2023, Kyverna had $57.5 million in cash, cash equivalents, and available-for-sale marketable securities[4] - The company raised approximately $366.9 million in gross proceeds from its initial public offering completed in February 2024, expected to fund operations into 2026[5] Clinical Development - Kyverna advanced clinical development of KYV-101, treating 14 autoimmune patients cumulatively, including patients with multiple sclerosis, lupus nephritis, and myasthenia gravis[6] - The FDA granted Fast Track Designations for KYV-101 for multiple indications, including refractory myasthenia gravis and lupus nephritis[6] Assets and Manufacturing - Kyverna's total assets increased to $75.2 million in 2023 from $66.9 million in 2022[15] - The company is focused on advancing its manufacturing process, Ingenui-T, to improve patient experience and manufacturing efficiencies[6]