Kyverna Therapeutics (KYTX) Investor presentation summary

Strategic vision and market opportunity

• Positioned to deliver curative CAR T therapy for neuroimmunology diseases, with a focus on stiff person syndrome (SPS) and generalized myasthenia gravis (gMG).

• First-mover advantage in SPS, establishing commercial foundation and enabling efficient expansion into gMG and other indications.

• Significant market opportunity: ~6,000 SPS and ~80,000 gMG diagnosed patients in the U.S., with high unmet need and premium pricing potential.

• Focused launch strategy targets high-value centers, leveraging commercial synergies and scalable infrastructure.

• Strong financial position with $236M cash and additional loan facility, supporting commercial launch and clinical development through 2028.

Clinical innovation and product differentiation

• Mivocabtagene autoleucel (miv-cel) is a fully human autologous CD19 CAR T with CD28 costimulation, dosed in over 100 patients across multiple indications.

• Demonstrates deep and broad B-cell depletion, supporting immune reset and durable remission with a single dose.

• No high-grade cytokine release syndrome (CRS) or neurotoxicity (ICANS) observed; well-tolerated safety profile confirmed in both clinical trials and compassionate use.

• Outperforms antibodies, mRNA CAR T, and T-cell engagers by providing complete B-cell depletion and durable, drug-free remission.

• Next-generation KYV-102 aims to further improve patient access and reduce costs with a rapid, whole-blood process.

Clinical trial results and efficacy

• SPS registrational trial (KYSA-8) met primary and secondary endpoints: 46% median improvement in walking ability (T25FW) at 16 weeks, with 81% achieving clinically meaningful improvement.

• 100% of SPS patients remained free of immunomodulatory therapies at last follow-up; significant improvements in disability, mobility, stiffness, and hypersensitivity.

• gMG trial (KYSA-6) showed rapid, robust, and sustained improvements in MG-ADL and QMG scores, with all patients achieving clinically meaningful responses and 100% drug-free status at 24 weeks.

• Miv-cel reduced autoantibody levels while preserving humoral immunity and induced broad immune reset, including increased naïve B cells and regulatory T cells.

• Phase 2/3 gMG trial underway, with global expansion and crossover design to further validate efficacy and durability.