Overview
- Kyverna reported topline results from the single‑arm, registrational Phase 2 KYSA‑8 trial in 26 stiff person syndrome patients after one dose of mivocabtagene autoleucel.
- At Week 16, the primary endpoint showed a median 46% improvement in the timed 25‑foot walk, with 81% of patients exceeding a clinically meaningful 20% improvement.
- Secondary measures also improved, and among 12 patients who used a walking aid at baseline, 67% no longer needed assistance at Week 16.
- Safety through the primary analysis showed no high‑grade cytokine release syndrome or immune effector cell‑associated neurotoxicity, and all patients remained off other immunotherapies at last follow‑up.
- Kyverna plans to submit a Biologics License Application in the first half of 2026, holds RMAT and Orphan designations for the program, will present the full dataset at a 2026 medical meeting, and saw its shares rise about 38% to $12.13 to a new 52‑week high.