Kyverna Therapeutics (KYTX) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Company overview and pipeline progress

• Lead therapy miv-cel is designed for autoimmune diseases, offering improved potency and safety over existing CAR-T and traditional therapies.

• Stiff Person Syndrome (SPS) is the first target, with no current FDA-approved therapies and high unmet need.

• Demonstrated unprecedented clinical responses, including disease reversal, in SPS patients.

• Building a neuroimmunology franchise, with future focus on myasthenia gravis and progressive MS.

Clinical data and regulatory strategy

• KYSA-8 pivotal study in SPS showed significant improvements across all endpoints, including reversal of disability.

• Two-thirds of patients using walking aids were able to discontinue them after 16 weeks.

• FDA has aligned with the single-arm study design and core submission components; rolling BLA submission expected to complete in Q4.

• Natural history data confirm SPS patients do not spontaneously improve, supporting the transformative effect of miv-cel.

Patient, physician, and payer perspectives

• Physicians and patients value the comprehensive impact: improved mobility, reversal of disability, and freedom from chronic therapies.

• Durable effects observed, with some patients off background therapies for up to two years.

• Payers are engaged and view the one-time therapy as cost-saving compared to chronic, ineffective treatments like IVIG.