Kyverna Therapeutics reported that its lead CAR‑T therapy, miv‑cel (formerly KYV‑101), met the primary endpoint in the single‑arm Phase 2 KYSA‑8 trial for stiff person syndrome (SPS). The study enrolled 26 patients and demonstrated a median 46 % improvement in the timed 25‑foot walk test, with 81 % of participants achieving a clinically meaningful 20 % gain. All secondary endpoints—including the Modified Rankin Scale, Distribution‑of‑Stiffness Index, Hauser Ambulation Index, and Heightened Sensitivity Scale—reached statistical significance (p < 0.0001).
The safety profile was favorable. No high‑grade cytokine release syndrome or immune‑effector cell‑associated neurotoxicity was observed. Grade 3/4 neutropenia occurred in a minority of patients, was manageable, and resolved without lasting sequelae. Importantly, every patient was able to discontinue chronic immunotherapies, indicating durable clinical benefit from a single infusion.
CEO Warner Biddle emphasized the clinical impact, stating, “These data represent a transformative breakthrough for patients with a disease that has no approved therapies.” Chief Medical Officer Naji Gehchan added, “miv‑cel’s ability to reverse progressive disability in SPS underscores the potential of CAR‑T technology beyond oncology.” The results support Kyverna’s plan to file a Biologics License Application with the FDA for SPS in the first half of 2026, positioning the company as a pioneer in the emerging neuro‑autoimmune cell‑therapy market.
Financially, Kyverna remains well‑capitalized. As of September 30 2025, the company held $171.1 million in cash and equivalents. A $25 million draw from a $150 million loan facility in November 2025 extends the cash runway into 2027, providing sufficient liquidity for the BLA filing, ongoing Phase 3 development in generalized myasthenia gravis, and pre‑launch activities. The strong trial outcome also enhances Kyverna’s valuation prospects by validating its platform and expanding the addressable market for miv‑cel beyond SPS.
The announcement is expected to reinforce investor confidence in Kyverna’s strategic trajectory, as the data confirm the therapeutic promise of its CAR‑T platform and open a new therapeutic avenue for a rare autoimmune disease with significant unmet need.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.