Kyverna Therapeutics announced positive primary analysis results from its registrational trial of its CD19-targeting CAR-T cell therapy, miv-cel, in stiff person syndrome (SPS).
Miv-cel is an autologous, CD19-targeting CAR-T cell therapy with CD28 co-stimulation, designed for potency and tolerability.
In the phase 2 registrational trial, KYSA-8, a single dose of miv-cel delivered rapid, statistically significant and clinically meaningful improvements across all primary and secondary endpoints at 16 weeks, with the majority of patients regaining function, and all 26 patients discontinuing chronic immunotherapies. According to Kyverna, these outcomes have not been previously observed in SPS — a rare, progressive neurologic autoimmune disease characterized by muscle stiffness and painful muscle spasms, impacting mobility and gait.
Kyverna CEO Warner Biddle said the company is preparing its BLA submission for this initial indication. The company also believes the therapy has potential in myasthenia gravis, as well as other neurologic autoimmune diseases.
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