As two Big Pharma players in the autoimmune CAR-T space face unexpected safety setbacks, Kyverna Therapeutics is reporting long-term clinical data that showcase sustained efficacy alongside an unblemished safety profile for its potential first-in-class candidate, miv-cel.

A single dose of miv-cel (mivocabtagene autoleucel) showed durable clinical benefit out to 12 months in a registrational trial for stiff person syndrome (SPS) and up to a year and a half in the phase 2 portion of a study for generalized myasthenia gravis (gMG), according to data released Thursday.

With zero cases of high-grade cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome (ICANS) or immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS), Kyverna plans to add the updated one-year data to its FDA rolling submission for miv-cel in SPS, keeping the biotech on track to complete its application in the fourth quarter.

The latest results showcase durable remission of miv-cel that “allows these patients to have a drug-free, disease-free remission and do so with a well-tolerated safety profile,” Kyverna CEO Warner Biddle told Fierce in an interview.

In the Kysa-8 trial, 26 patients with SPS who received miv-cel showed a median 49% improvement on the timed 25-foot walk test (T25FW) after 12 months versus baseline. The sustained efficacy marked a slight improvement from the 46% recorded at week 16, which already translated into a statistically significant win on the trial’s primary endpoint.

All but one patient who had previously achieved a clinically meaningful improvement— defined as at least a 20% reduction from baseline—at the 16-week primary analysis sustained that level of benefit at at the one-year mark.

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