Kyverna Therapeutics (KYTX) Investor presentation summary

Transformative therapy overview

• Miv-cel is a single-dose, fully human autologous CD19 CAR T therapy designed to reset the immune system and deliver durable, drug-free, disease-free remission in neurologic autoimmune diseases.

• Demonstrated deep depletion of B cells, CNS penetration, and upregulation of regulatory T cells, supporting broad immune reset and durable remission.

• Over 100 patients have been dosed across multiple indications with no high-grade CRS, ICANS, or IEC-HS reported.

• Validated, scalable manufacturing process with >98% success rate and dual-source U.S. manufacturing strategy.

Clinical efficacy and safety in stiff person syndrome (SPS)

• SPS is a progressive, debilitating autoimmune disease with no FDA-approved therapies, causing severe mobility loss and high economic burden.

• In the KYSA-8 registrational trial (N=26), miv-cel met its primary endpoint with a 46% median reduction in T25FW at 16 weeks, sustained at 49% at 1 year.

• 92% of patients remained free of immunomodulatory or immunosuppressant therapies for SPS at 1 year; 67% of those needing walking aids at baseline no longer required them.

• Statistically significant improvements were observed in all primary and secondary endpoints, including disability and ambulation scores, through 1 year.

• Safety profile remained consistent with no high-grade CRS, ICANS, or IEC-HS; all serious adverse events resolved.

Market opportunity and launch strategy

• SPS market includes ~6,000 diagnosed U.S. patients, with 2,000–2,500 immediately addressable; chronic disease progression supports long-term market capture.

• Premium pricing anticipated ($500–$600K WAC), targeting ~10 high-value academic centers for launch.

• Strong early adoption interest: 85% of surveyed SPS treaters would use miv-cel for moderate-to-severe patients at launch.

• Commercial agreements and payer dynamics expected to enable efficient, scalable launch and biologics-like margins.