Kyverna Therapeutics Highlights Neuroimmunology CAR T Franchise and Registrational Phase 3 Trial Design in Myasthenia Gravis at Virtual KOL Event

Core Insights - Kyverna Therapeutics is advancing its CAR T-cell therapy, KYV-101, which has the potential to provide durable, drug-free, disease-free remission for patients with myasthenia gravis (MG) and stiff person syndrome (SPS) [3][24] - The company has designed an innovative Phase 3 trial for MG, aligning with FDA requirements, which is expected to facilitate a clear path to Biologics License Application (BLA) [4][6] - The virtual KOL event will showcase positive long-term data from compassionate use patients and insights from key opinion leaders (KOLs) regarding the potential of CD19 CAR T-cell therapy in autoimmune diseases [2][9] Company Overview - Kyverna Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing cell therapies for autoimmune diseases, with KYV-101 as its lead candidate [25] - The company is also conducting multi-center Phase 1/2 trials for lupus nephritis and exploring other autoimmune indications, including multiple sclerosis and rheumatoid arthritis [25] Clinical Trial Details - The KYSA-6 Phase 2/3 trial for MG is an open-label, randomized, controlled study aimed at demonstrating the superiority of KYV-101 over standard-of-care treatments [5][6] - The trial will enroll approximately 60 patients, randomized to receive either KYV-101 or continue with standard-of-care therapy, with co-primary endpoints focusing on changes in MG-ADL and QMG scores at 24 weeks [7][8] - The trial design allows for the assessment of KYV-101 as a standalone treatment, evaluating its potential for durable remission without concurrent immunosuppressive therapy [6][7] Disease Background - Myasthenia gravis is a neuromuscular autoimmune disease characterized by muscle weakness and fatigue, often leading to severe complications such as respiratory failure [22] - Stiff person syndrome is a rare autoimmune disease marked by muscle stiffness and spasms, resulting in significant mobility impairment and disability [23] Future Expectations - Kyverna expects to initiate patient enrollment in the Phase 3 portion of the KYSA-6 trial by the end of 2025 and plans to report interim data from the Phase 2 portion in the fourth quarter of 2025 [4][8]