Kyverna Therapeutics(KYTX) - 2024 Q4 - Annual Results

Business Update and Financial Results Overview Kyverna reported full-year 2024 financial results and business highlights, emphasizing its leadership in CAR T-cell therapies for autoimmune diseases and a strong balance sheet - Kyverna reported its full-year 2024 financial results and business highlights, emphasizing its leadership in developing CAR T-cell therapies for autoimmune diseases[1][2] - The company is accelerating the clinical path for its lead candidate, KYV-101, in three priority indications: stiff person syndrome (SPS), myasthenia gravis (MG), and lupus nephritis (LN)[1][2] - A strong balance sheet is expected to extend the company's cash runway into 2027, funding operations through key clinical and regulatory milestones[1][12] Clinical Pipeline and Program Updates The company provided updates on its clinical pipeline, including progress for lead candidate KYV-101 in multiple indications and the development of next-generation KYV-102 KYV-101 Program The company's lead candidate, KYV-101, an autologous, fully human CD19 CAR T-cell therapy, is being prioritized for development in Stiff Person Syndrome, Myasthenia Gravis, and Lupus Nephritis, with significant clinical trial progress and regulatory alignment achieved Stiff Person Syndrome (SPS) The KYV-101 program for Stiff Person Syndrome is advancing with a registrational Phase 2 trial, targeting BLA filing in 2026, and has received multiple FDA designations - Kyverna has aligned with the FDA on a registrational Phase 2 trial design for KYSA-8 in SPS, which is currently 70% enrolled[1][6] - Key upcoming milestones for the SPS program include completing enrollment by mid-2025, reporting topline data in the first half of 2026, and targeting a Biologics License Application (BLA) filing in 2026[1][6] - The program has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the FDA[6] Myasthenia Gravis (MG) The KYV-101 program for Myasthenia Gravis has completed initial enrollment for its Phase 2 trial, with interim data expected in late 2025, and holds multiple FDA and EMA designations - The Phase 2 trial in MG, KYSA-6, has completed enrollment for its initial 6-patient cohort[5] - Interim data from this cohort is expected to be reported in the second half of 2025[5] - The program has received RMAT, Fast Track, and Orphan Drug designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency[7] Lupus Nephritis (LN) The KYV-101 program for Lupus Nephritis is advancing two Phase 1/2 trials, with Phase 1 data expected in late 2025, and has received Fast Track Designation from the FDA - Kyverna is advancing two Phase 1/2 trials (KYSA-1 and KYSA-3) and expects to report Phase 1 data from both in the second half of 2025[10] - The company has received Fast Track Designation from the FDA for this program[10] - Positive clinical data presented in November 2024 showed sustained efficacy and durability at over 6-month follow-up in patients with severe LN treated with KYV-101[10] Additional Indications Kyverna is exploring additional opportunities for KYV-101 through sponsored and investigator-initiated trials in other autoimmune diseases - Kyverna is exploring additional opportunities for KYV-101 through sponsored and investigator-initiated trials in diseases such as systemic sclerosis and multiple sclerosis[9] KYV-102 Program KYV-102 is a next-generation candidate utilizing the same CD19 CAR T as KYV-101 but incorporates a proprietary whole-blood rapid manufacturing process. This approach aims to improve the patient experience and broaden access to CAR T therapy by eliminating the need for apheresis - Kyverna plans to file an investigational new drug (IND) application for KYV-102 in the second half of 2025[9] Corporate Updates and Anticipated Milestones The company announced key leadership appointments and outlined anticipated clinical and regulatory milestones for its pipeline programs Corporate Updates The company strengthened its leadership by appointing a new CEO, Warner Biddle, and making several other key additions to the management team. The Board of Directors was also enhanced with the appointment of Christi Shaw and Mert Aktar, who bring extensive industry and manufacturing expertise - Strengthened management team with key hires including a new CEO, Chief Medical and Development Officer, and Chief Business Officer[11] - Appointed Christi Shaw and Mert Aktar to the Board of Directors, adding significant corporate strategy and cell therapy manufacturing expertise[11] Anticipated Milestones Kyverna has provided clear guidance on its upcoming clinical and regulatory milestones through 2026, focusing on completing enrollments, reporting trial data for its three priority indications, and filing its first BLA for KYV-101 Upcoming Program Milestones | Program | Milestone | Anticipated Timing | | :--- | :--- | :--- | | SPS | Complete Pivotal Phase 2 Enrollment | mid-2025 | | | Report Topline Pivotal Phase 2 Data | 1H 2026 | | | BLA filing | 2026 | | MG | Confirm Registrational Path with Regulators | 1H 2025 | | | Report Interim Phase 2 Data | 2H 2025 | | LN | Report Phase 1 Data | 2H 2025 | | Future Pipeline | File KYV-102 IND application | 2H 2025 | Financial Results Kyverna reported its full-year 2024 financial performance, including a significant increase in cash position and operating expenses, leading to a wider net loss Financial Highlights for Full Year 2024 For the year ended December 31, 2024, Kyverna reported a strong cash position of $286.0 million, providing a cash runway into 2027. The company's net loss increased to $127.5 million from $60.4 million in the prior year, driven by higher operating expenses Key Financial Metrics (Year Ended Dec 31) | Metric | 2024 | 2023 | | :--- | :--- | :--- | | Cash, Cash Equivalents, and Marketable Securities | $286.0 million | $57.5 million | | Net Loss | $127.5 million | $60.4 million | | Net Loss Per Common Share | $3.33 | $89.61 | | Net Cash Used in Operating Activities | $114.3 million | $52.4 million | - The company expects its cash, cash equivalents, and marketable securities of $286.0 million to provide a cash runway into 2027[12] Statements of Operations The company's operating expenses more than doubled in 2024, primarily due to a significant increase in research and development costs from $49.9 million to $112.5 million. This led to a wider loss from operations and a net loss of $127.5 million for the year Statements of Operations (in thousands) | Line Item | Year Ended Dec 31, 2024 | Year Ended Dec 31, 2023 | | :--- | :--- | :--- | | Research and development | $112,473 | $49,923 | | General and administrative | $30,131 | $12,483 | | Total operating expenses | $142,604 | $62,406 | | Loss from operations | ($142,604) | ($62,406) | | Total other income, net | $15,127 | $2,040 | | Net loss | ($127,477) | ($60,366) | Condensed Balance Sheets As of December 31, 2024, Kyverna's balance sheet showed significant growth, with total assets increasing to $304.6 million from $75.2 million in 2023. This was primarily driven by a substantial rise in cash and marketable securities. Stockholders' equity turned positive to $266.6 million from a deficit of $131.4 million, reflecting financing activities during the year Condensed Balance Sheets (in thousands) | Line Item | December 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $285,979 | $57,543 | | Total assets | $304,645 | $75,195 | | Total liabilities | $38,058 | $26,018 | | Stockholders' equity (deficit) | $266,587 | ($131,397) | About Kyverna and its Products This section provides an overview of Kyverna Therapeutics, its lead product candidates KYV-101 and KYV-102, and their therapeutic potential About KYV-101 KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate designed for tolerability with a potent CD28 co-stimulation domain. It is being investigated for its potential to achieve deep B cell depletion and long-term remission in various B-cell-driven autoimmune diseases - KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate designed for B-cell-driven autoimmune diseases[14] - It is currently being evaluated in a pivotal Phase 2 trial for stiff person syndrome, a Phase 2 trial for myasthenia gravis, and Phase 1/2 trials for lupus nephritis[15] About KYV-102 KYV-102 is a next-generation therapy that uses the same clinically validated CD19 CAR-T construct as KYV-101. It incorporates the proprietary Ingenui-T platform, which enables a rapid manufacturing process using whole blood, aiming to eliminate the need for apheresis and broaden patient access - KYV-102 leverages the same CAR-T construct as KYV-101 but incorporates the Ingenui-T platform, a proprietary, next-generation process[16] - The platform utilizes whole blood with a rapid manufacturing approach, aiming to eliminate the need for apheresis and reduce manufacturing turnaround time[16] About Kyverna Therapeutics Kyverna Therapeutics is a clinical-stage biopharmaceutical company focused on the curative potential of cell therapy for patients with autoimmune diseases. Its lead candidate, KYV-101, is advancing through multiple clinical trials, and its pipeline includes next-generation autologous and allogeneic CAR T-cell therapies - Kyverna is a clinical-stage biopharmaceutical company focused on developing cell therapies for autoimmune diseases[17] - The company's lead candidate, KYV-101, is in Phase 2 trials for stiff person syndrome and myasthenia gravis, and Phase 1/2 trials for lupus nephritis[17]