Kyverna Therapeutics (KYTX) H.C. Wainwright 28th Annual Global Investment Conference summary

Strategic focus and pipeline developments

• Advancing cell therapies for autoimmune diseases, with a unique CAR construct and established manufacturing process, focusing on neuroimmunology and Stiff Person Syndrome (SPS).

• BLA filing for SPS on track for Q4, aiming to be the first approved therapy for SPS and first cell therapy in autoimmune diseases globally.

• Promising data in generalized myasthenia gravis (gMG) and progressive MS, with plans to expand miv-cel to more patients.

• Over 100 patients dosed with miv-cel, showing no high-grade CRS, ICANS, or IEC-HS events, highlighting a strong safety profile.

• Manufacturing uses traditional, validated processes, avoiding rapid manufacturing to ensure product consistency and safety.

Clinical results and patient impact

• SPS affects 6,000 US patients, with 80% progressing to severe disability; miv-cel shows transformative results, including reversal of disability.

• Two-thirds of patients needing walking devices at baseline no longer required them after 16 weeks of miv-cel therapy.

• One-time miv-cel therapy enables patients to discontinue chronic therapies, improving mobility and quality of life.

• 16-week data showed a 46% reduction in the timed 25-foot walk test, sustained over time; one-year durability data expected soon.

• Natural history study of SPS patients over 10 years showed progressive worsening, contrasting sharply with miv-cel outcomes.

Regulatory and commercialization outlook

• Rolling BLA submission granted after positive pre-BLA meeting; CMC module submitted, clinical module pending one-year follow-up data.

• Natural history data contextualizes clinical results and supports regulatory discussions; FDA interested in using this as supporting evidence.

• High demand anticipated at launch, with 2,000–2,500 refractory SPS patients immediately addressable and a total US population of 6,000.

• Commercialization model targets concentrated patient populations in academic centers for capital efficiency.

• Pricing strategy aims for a significant premium over current CAR T therapies ($500,000–$600,000), justified by high value and cost offsets.