Kyverna Therapeutics(US:KYTX) Globenewswire·2025-12-15 11:30Core Insights - Mivocabtagene autoleucel (miv-cel) has shown transformative results in treating stiff person syndrome (SPS), potentially becoming the first FDA-approved CAR T-cell therapy for autoimmune diseases [1][3][5] - The company plans to submit a Biologics License Application (BLA) for miv-cel in the first half of 2026 [1][5][8] Efficacy - Miv-cel demonstrated statistically significant improvements in all primary and secondary endpoints, reversing disability and eliminating the need for immunotherapies after a single dose [1][5][6] - The primary endpoint, the timed 25-foot walk (T25FW), showed a median improvement of 46% at Week 16 compared to baseline, with 81% of patients exceeding a 20% improvement [6][12] - Secondary endpoints also showed highly statistically significant benefits, with all p-values <0.0001, including improvements in the Modified Rankin Scale (mRS) and Hauser Ambulation Index (HAI) [6][12] Safety - Miv-cel was well-tolerated, with no high-grade cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) observed [1][14] - Grade 3/4 neutropenia was noted but was manageable, indicating a favorable safety profile [14] Clinical Trial Overview - The KYSA-8 trial is a single-arm registrational Phase 2 study involving 26 patients with SPS who had inadequate responses to non-approved treatments [4][11] - Patients received a single dose of miv-cel after lymphodepletion with cyclophosphamide and fludarabine, with follow-up for one year [12] Company Background - Kyverna Therapeutics is focused on developing cell therapies for autoimmune diseases, with miv-cel as its lead candidate [17] - The company has received Regenerative Medicine Advanced Therapy and Orphan Drug designations for miv-cel in the context of SPS [8][18]