Kyverna Therapeutics disclosed that its Phase 2 registrational trial (KYSA‑8) of mivocabtagene autoleucel (miv‑cel) in stiff person syndrome (SPS) met its primary endpoint. The single‑arm study enrolled 26 patients who received a single dose of 1 × 10⁸ CAR‑T cells and achieved a 46 % median improvement in the Timed 25‑Foot Walk at 16 weeks, with 81 % of patients reaching the predefined clinically meaningful threshold.
Safety data were favorable, with no high‑grade cytokine release syndrome or neurotoxicity observed. In addition, 67 % of patients who required a walking aid at baseline no longer needed assistance, and all 26 patients discontinued chronic immunotherapies by week 16.
The results position miv‑cel as the first CAR‑T therapy for an autoimmune disease and could secure a multi‑year exclusivity window in a market that, while rare for SPS, sits within a broader $80 billion autoimmune disease landscape. The trial’s success also supports Kyverna’s strategy to advance miv‑cel across other B‑cell‑mediated indications such as generalized myasthenia gravis, multiple sclerosis, and rheumatoid arthritis.
Kyverna reported a net loss of $161.3 million for 2025 and holds $279.3 million in cash as of December 31 2025, providing a runway that extends into 2028. The company’s financial position is expected to support the upcoming BLA filing and the commercial launch of miv‑cel in SPS.
Warner Biddle, CEO, said, “The results from our KYSA‑8 registrational trial mark a defining moment for Kyverna, and more importantly, for patients living with stiff person syndrome. We see compelling evidence that a one‑time therapy can reset the immune system, reverse the course of disease, and free patients from lifelong treatment burden.” Naji Gehchan, Chief Medical & Development Officer, added, “The findings from our primary analysis of miv‑cel highlight its differentiated therapeutic profile, with the potential to deliver durable disease‑free remission with just a single dose, without need for any immunotherapies for SPS.”
Kyverna plans to file a Biologics License Application for SPS in the first half of 2026 and will continue to advance miv‑cel in its broader pipeline, leveraging its Regenerative Medicine Advanced Therapy and orphan drug designations to accelerate regulatory review.
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