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Miv-cel Shows Durable Results in Stiff Person Syndrome and Myasthenia Gravis
Friday, 30 October, 2026
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses one-year data showing sustained benefits of KYV-101 (mivocabtagene autoleucel ; Miv-cel) in stiff person syndrome (SPS), along with longer-term follow-up data in generalized myasthenia gravis (gMG).SPS and gMG are rare neurological and neuromuscular disorders:SPS: SPS is a rare, progressive neurological autoimmune disease that causes muscle stiffness and painful spasms that can affect walking and mobility. Up to 80% of patients lose their mobility over time, and there are currently no FDA-approved treatments. gMG: gMG is a rare autoimmune neuromuscular disease characterized by muscle weakness and fatigue. As the disease progresses, it can interfere with daily activities, such as speaking, chewing, swallowing, and breathing. Positive one-year topline data from the KYSA-8 clinical trial (NCT06588491) were announced on September 24, 2026. KYSA-8 was a registrational, phase two, open-label, single-arm, multicenter study of miv-cel.Miv-cel is a fully human, autologous, CD19-targeting CAR T-cell therapy with CD28 co-stimulation. It’s designed to achieve deep B-cell depletion, reset the immune system, and deliver durable drug-free, disease-free remission in autoimmune diseases with a single dose. A total of 26 adult patients who had an inadequate response to at least one immunotherapy treatment participated in the study. Each participant received a single dose of miv-cel. Among patients who achieved a clinically meaningful improvement in the timed 25-foot walk at primary analysis, 95% maintained that benefit at 12 months. Of the 12 patients who needed a walking aid before treatment, 67% still did not require assistance at the 12-month follow-up. Furthermore, 92% of patients remained off chronic immunotherapies for SPS. Also announced were longer-term follow-up data for its KYSA-6 trial (NCT06193889). KYSA-6 is a phase 2/3 trial evaluating miv-cel, administered as a single dose, in patients with gMG. A total of seven adult patients participated in the study. Follow-up now extends to one year and beyond (up to 1.5 years) in five patients, with one patient at 9 months and another at 6 months. All seven participants achieved clinically meaningful improvements in both the Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores at 24 weeks, while all five participants with at least one year of follow-up maintained clinically meaningful improvements. Additionally, six of the seven patients remained off immunosuppressant therapies at their latest follow-up. Miv-cel continued to be well-tolerated in both studies. In the KYSA-8 trial, no high-grade cases of cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) and no cases of immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) were reported through the 12-month follow-up.In the KYSA-6 trial, no high-grade CRS, no ICANS, and no cases of IEC-HS were reported during the available follow-up.For more information, visit: https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-therapeutics-reports-positive-one-year-dataTo learn more about stiff person syndrome, myasthenia gravis, and other rare neurological and neuromuscular disorders, visit: https://checkrare.com/diseases/neurology-nervous-system-diseases/Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.
