Kyverna Therapeutics (KYTX) Corporate presentation summary

Strategic vision and market opportunity

• Positioned as a leader in autoimmune CAR T therapies, aiming for the first approved CAR T-cell therapy for autoimmune diseases with a differentiated, best-in-class profile and durable efficacy in indications like SPS and gMG.

• Focused neuroimmunology franchise strategy leverages first-mover advantage in SPS to expand into gMG and other indications, supported by a scalable commercial and medical infrastructure.

• Pipeline-in-a-product approach with clinical data supporting expansion into broader autoimmune indications, including MS, RA, and lupus nephritis.

• Commercial opportunity in SPS with premium pricing potential and a concentrated patient base for efficient market entry.

• Well-funded through key milestones, including SPS BLA filing, commercialization, and Phase 3 gMG trial.

Clinical development and efficacy

• Miv-cel (KYV-101) is a fully human autologous CD19 CAR T with over 100 patients dosed across multiple indications, showing deep and broad B-cell depletion and durable efficacy beyond 24 months in SPS and gMG.

• Registrational SPS trial (KYSA-8) demonstrated statistically significant improvements in mobility and disability scores, with 81% achieving clinically meaningful improvement and 67% of previously aid-dependent patients regaining independent walking.

• Safety profile is favorable, with no high-grade CRS or ICANS observed; most adverse events were manageable and resolved within 28 days.

• In gMG, interim Phase 2 results showed 100% clinically meaningful response, rapid and sustained reductions in MG-ADL and QMG scores, and all patients remained drug-free up to 24 weeks post-treatment.

• Compassionate use data in 40 patients across neuroimmunology and rheumatologic diseases reinforce safety and inform future indications.

Regulatory and commercialization strategy

• Clear regulatory path with FDA-aligned registrational trial designs, RMAT and ODD designations, and anticipated SPS BLA filing in 1H 2026.

• Commercialization strategy includes leveraging clinical sites, activating new centers, optimizing patient and center experience, and ensuring manufacturing readiness.

• Dual-source U.S. manufacturing strategy supports scalable growth, with >98% manufacturing success rate and biologics-like margins.

• SPS launch targets ~10 centers, expanding to 50-75 centers for gMG, focusing on high-volume treaters and centers with CAR T experience.

• Favorable patient demographics and high-cost burden support premium pricing and rapid adoption.