Kyverna Therapeutics (KYTX) Wells Fargo 21st Annual Healthcare Conference summary

Key program updates and clinical milestones

• Pivotal data for miv-cel in stiff person syndrome (SPS) showed unprecedented reversal of disability and drug-free remission with a one-time treatment.

• Rolling BLA submission for SPS is underway, with all modules except the clinical module filed; full submission expected in Q4, aiming for launch readiness in 2027.

• One-year data readout for SPS and additional natural history analyses will be included in the BLA clinical package, with results expected imminently.

• Phase III trial for myasthenia gravis (MG) is ongoing, leveraging strong phase II results showing transformative reductions in MG-ADL and QMG scores.

• Progressive MS program received RMAT designation based on early data showing stabilization and improvement in EDSS, with a clinical development plan expected in early 2027.

Regulatory and market access strategy

• RMAT designation and priority review are being pursued for SPS, supported by high unmet need, transformative results, and strong safety profile.

• Ongoing productive dialogue with the FDA, with consistent review committees and supportive feedback on the development plan.

• Launch strategy for SPS targets 10 specialized CAR T centers, focusing on concentrated patient populations and efficient commercialization.

• Payer discussions highlight the value of a one-time therapy, with anticipated premium pricing over current CAR T therapies due to significant cost offsets.

Manufacturing and safety innovations

• Miv-cel features a unique CD28 costimulatory domain and fully human CAR T design, contributing to strong efficacy and safety.

• No high-grade CRS, ICANS, or IEC-HS observed in over 100 treated patients; traditional, validated manufacturing process yields 98% success rate.

• Manufacturing partnerships with ElevateBio and Minaris Advanced Therapies ensure scalability for SPS, MG, and future indications.

• Ongoing innovation in manufacturing aims to reduce turnaround time and improve patient access, including potential use of whole blood.