Kyverna Therapeutics (KYTX) investor relations material

Strategic positioning and innovation

• Holds a leadership position in autologous CAR T therapies for autoimmune diseases, leveraging a unique CAR T construct with proven durability and safety across multiple indications.

• Focuses on Stiff Person Syndrome (SPS) as the lead indication, with plans to expand into Myasthenia Gravis (MG), progressive MS, and Rheumatoid Arthritis.

• Manufacturing advancements enable rapid, cost-effective, and scalable production, supporting commercial launch ambitions.

• Dual-source manufacturing with high success rates and biologics-like margins positions for efficient market entry.

• Strong leadership team assembled with expertise in cell therapy, rare diseases, and neuroimmunology.

Clinical data and patient outcomes

• Over 100 patients treated with no high-grade CRS or ICANS, showing durable responses beyond 24 months in SPS and MG.

• miv-cel demonstrates deep B cell depletion, broad immune reset, and upregulation of regulatory T cells, indicating a fundamentally different mechanism from traditional therapies.

• SPS pivotal study (KYSA-8) showed a 46% reduction in the timed 25-foot walk at 16 weeks, with two-thirds of patients eliminating walking aids.

• Secondary endpoints in SPS, including mobility and stiffness indices, showed statistically and clinically meaningful improvements.

• Phase II MG study showed significant reductions in MG-ADL and QMG scores, with 100% response rate and 57% achieving minimal symptom expression.

Regulatory and commercial milestones

• Achieved FDA alignment on CMC and single-arm trial design for SPS, with rolling BLA submission underway and completion targeted for Q4.

• Plans to launch initially at 10 centers of excellence, targeting concentrated patient populations and leveraging high physician interest.

• Over 90% of SPS treaters view the clinical profile as compelling, with 85% ready to use at launch for moderate to severe patients.

• SPS market includes 6,000 diagnosed U.S. patients, with 2,000–2,500 refractory to current therapies as initial targets.

• MG market opportunity includes 80,000 diagnosed U.S. patients, with 12,000 refractory to biologics as initial focus.