Kyverna Therapeutics reported positive primary phase II results from its registrational trial KYSA-8 of mivocabtagene autoleucel (miv-cel, KYV-101) in stiff-person syndrome. In an oral presentation at the American Academy of Neurology annual meeting, the company said the therapy produced rapid, statistically significant improvements at 16 weeks and helped most patients regain function. Kyverna characterized the study as notable because the majority of patients stopped chronic immunotherapies and showed clinically meaningful endpoint gains without new safety concerns. The company is preparing regulatory next steps, including plans to submit a BLA to the FDA in the first half of this year. The results further validate the broader strategy of using CAR T in non-oncology settings where sustained disease control depends on overcoming persistent immune dysregulation rather than direct tumor targeting.
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