Kyverna Therapeutics (KYTX) investor relations material

Key achievements and clinical progress

• Reported transformative pivotal data for miv-cel in stiff person syndrome, showing reversal of disease course and significant improvement in clinical symptoms, with a 46% reduction in the 25-foot walk test at 16 weeks, more than double the clinically meaningful threshold.

• Preparing for BLA submission in the first half of the year, aiming for potential launch by year-end as the first autologous CAR T in autoimmune disease.

• Manufacturing success rates are high, with 95-100% success in clinical trials, and two suppliers in place to support launch and scale-up.

• Commercial launch strategy targets 10 key academic centers, covering the majority of refractory SPS patients, enabling a capital-efficient rollout.

• Pipeline expansion includes myasthenia gravis (MG) with promising phase II data, phase III enrollment underway, and early positive data in progressive MS.

Differentiation and value proposition

• miv-cel is a next-generation, fully human CD19 CAR T with a CD28 costimulatory domain, offering a 10-fold reduction in high-grade CRS and ICANS, enabling outpatient administration.

• Demonstrates deep, durable B-cell depletion and potential for long-term remission with a single treatment, reducing or eliminating need for chronic therapies.

• Health economic benefits include potential to replace costly chronic treatments like IVIG and reduce overall burden on patients and caregivers.

• Positive payer feedback supports potential for premium pricing above current CAR T therapies.

Regulatory and commercial outlook

• Strong, consistent engagement with FDA, leveraging RMAT and orphan drug designations, with confidence in regulatory pathway for single-arm pivotal study.

• SPS addressable population estimated at 6,000 in the US, with 2,000-2,500 refractory patients as initial target; MG market estimated at 80,000, with 40,000 CAR T-eligible and 12,000-13,000 refractory patients.

• Efficient sales force planned for initial launch, with expansion aligned to additional indications and centers.

• Academic centers are increasing capacity for autoimmune CAR T, providing first-mover advantage and facilitating outpatient treatment.