Kyverna Therapeutics (KYTX) Investor presentation summary

Clinical progress and efficacy

• Achieved transformative clinical efficacy in stiff person syndrome (SPS) and myasthenia gravis (MG), with statistically significant improvements in all primary and secondary endpoints for SPS and 100% clinically meaningful response in MG interim Phase 2 results.

• Miv-cel demonstrated robust, sustained mobility improvements in SPS, with 81% of patients achieving clinically meaningful improvement and 67% of those previously needing walking aids no longer requiring assistance.

• No high-grade cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) observed in trials or compassionate use, supporting a favorable safety profile.

• Miv-cel's single-dose approach led to 100% elimination of off-label immunotherapies in SPS and drug-free remission in MG up to 24 weeks.

• Early data in multiple sclerosis, rheumatoid arthritis, and lupus nephritis show promising efficacy and safety, expanding the potential of miv-cel in autoimmune diseases.

Regulatory and commercial strategy

• Established clear regulatory path with FDA-aligned registrational trial designs, RMAT and ODD designations, and BLA filing for SPS planned in 1H 2026.

• Positioned to be first-to-market in SPS, followed by MG, leveraging first-mover advantage to build scalable neuroimmunology franchise and commercial infrastructure.

• SPS launch targets ~10 centers, with expansion to 50–75 centers for MG at peak, focusing on high-volume treaters and centers with CAR T experience.

• Commercialization strategy includes activating clinical sites, payer engagement, patient advocacy, and ensuring manufacturing readiness.

• Addressable market for SPS estimated at 2,000–2,500 patients at launch, with potential to expand to 5,500; MG addressable market up to 40,000 patients.

Product differentiation and innovation

• Miv-cel is a fully human autologous CD19 CAR T with CD28 costimulation, designed for potency and tolerability, achieving deep and broad B-cell depletion.

• Demonstrates superior depth and duration of response compared to chronic antibody, mRNA CAR T, and T-cell engager therapies, with a single-dose, drug-free approach.

• KYV-102, a next-generation CAR T, aims to further improve patient access, reduce costs, and eliminate the need for apheresis or intensive lymphodepletion.

• Compassionate use and investigator-initiated trials provide a robust safety database and inform future indication expansion.

• Proprietary manufacturing and CMC strategies support scalability and cost efficiency.