Kyverna Therapeutics (KYTX) Status update summary

Clinical trial results and efficacy

• Miv-cel demonstrated rapid, statistically significant, and clinically meaningful improvements across all primary and secondary endpoints in SPS, with 81% of patients achieving or exceeding a 20% improvement in the Timed 25-Foot Walk at week 16 and nearly a third walking at healthy adult speed.

• Two-thirds of patients who required walking aids at baseline no longer needed assistance at week 16, and improvements were sustained through 24 weeks.

• All patients discontinued chronic immunotherapies, with significant improvements in disability, stiffness, hypersensitivity, and ambulation indices; SF-36 scores at week 16 were comparable to healthy adults.

• In gMG, 100% of patients achieved clinically meaningful, rapid, and robust responses, with the majority remaining symptom-free and off immunotherapies at last follow-up.

• Durable efficacy was observed, with some patients maintaining benefits beyond 24 months after a single dose.

Safety and immune reset

• Miv-cel showed a consistent, well-tolerated safety profile with no high-grade CRS or ICANS; Grade 3/4 neutropenia was manageable and resolved.

• Serious treatment-related adverse events were rare and resolved fully.

• Robust CAR T-cell expansion resulted in deep B-cell depletion, significant reduction in autoantibody titers, and increased regulatory T-cells, with evidence of immune reset.

• Patients maintained humoral immunity to common vaccinations despite B-cell depletion.

Market opportunity and launch strategy

• Approximately 6,000 SPS patients are diagnosed in the US, with 2,000–2,500 immediately addressable due to inadequate response to current therapies.

• Initial launch will target 10 high-volume centers, covering roughly half of the immediately addressable SPS population, with plans to expand to additional centers for SPS and gMG.

• Physician feedback indicates strong early adoption potential, with 85% of surveyed treaters willing to use miv-cel in moderate to severe SPS patients at launch.

• Centers selected for launch have CAR T experience, strong referral networks, and favorable reimbursement dynamics.