What's Happening?
Kyverna Therapeutics has reported significant improvements in mobility for patients with stiff person syndrome (SPS) using its investigational CAR T therapy, mivocabtagene autoleucel (miv-cel). In a pivotal study, patients experienced a 46% median improvement in mobility,
as measured by the timed 25-foot walk (T25FW), after 16 weeks of treatment. The therapy also met all secondary endpoints, including improvements in ambulation, sensitivity, and disability. Notably, all patients were able to discontinue immunotherapies without needing rescue therapy. The company plans to submit a biologics license application to the FDA in the first half of 2026, potentially making miv-cel the first FDA-approved CAR T therapy for an autoimmune disease.
Why It's Important?
The development of miv-cel represents a significant advancement in the treatment of stiff person syndrome, a rare neurological disorder affecting fewer than 5,000 people in the U.S. The therapy's ability to improve mobility and reduce the need for chronic treatment could greatly enhance the quality of life for patients. Additionally, the success of miv-cel could pave the way for further CAR T therapies targeting autoimmune diseases, potentially transforming treatment paradigms and reducing healthcare burdens associated with long-term management of such conditions.
What's Next?
Kyverna plans to file for FDA approval in 2026, which, if granted, would mark a milestone in autoimmune disease treatment. The approval process will be closely watched by stakeholders in the biotech and healthcare sectors, as it could influence future research and investment in CAR T therapies. The outcome may also prompt discussions among healthcare providers about integrating such therapies into standard care practices for autoimmune diseases.
